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Noted This Week - 2017 Archive

Archive of cancer-related news briefs, by week, for 2017


December 2017

December 29, 2017–January 4, 2018

  • According to a report from the American Cancer Society, the cancer mortality rate decreased by 26% between 1991 and 2015, which translates into approximately 2.3 million fewer deaths, a change that can largely be attributed to declines in mortality for lung, breast, and prostate cancers. The overall mortality rate, however, varied by race: In 2015, it was 14% higher for blacks than it was for whites, a disparity that was especially pronounced in people under age 65.
  • Alcohol, in the form of ethanol, damages DNA in stem cells in mice, resulting in a rearrangement of chromosomes and irreversible changes to DNA sequences, according to a study published in Nature. The study also found that mice lacking the aldehyde dehydrogenase enzyme ALDH2 that normally breaks down acetaldehyde, a by-product of ethanol, incurred four times as much DNA damage as control mice.
  • Biotech company Arvinas is teaming up with Pfizer in a deal potentially worth $830 million for the discovery and development of small-molecule protein degradation therapies. Their efforts will focus on developing proteolysis-targeting chimeras, or PROTACs, therapies designed to break down the cellular proteins that cause disease. So far, Arvinas has been advancing PROTACs to treat cancer, but small molecules could have applications in other fields, such as neuroscience.
  • Lung cancer screening based on individual risk may save more lives than screening based on current U.S. Preventive Services Task Force recommendations, according to an Annals of Internal Medicine study. Current guidelines recommend annual screening with low-dose CT for anyone between the ages of 55 and 80 who currently smokes or quit in the last 15 years and has at least a 30-pack-year smoking history. However, these guidelines may miss some people at high risk for lung cancer: The study found that screening based on individual risk calculated using the Lung Cancer Risk Assessment Tool could have prevented more than 5,000 additional deaths in 2015 than current screening criteria.
  • The FDA granted Priority Review to brentuximab vedotin (Adcetris; Seattle Genetics) in combination with chemotherapy for first-line treatment of patients with advanced classic Hodgkin lymphoma. The decision was based on data from the phase III ECHELON-1 trial, which determined that patients who received brentuximab vedotin plus AVD (doxorubicin, vinblastine, and dacarbazine) chemotherapy had a 23% reduction in the risk of disease progression, death, or need for additional cancer treatments compared with those who received the ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine) chemotherapy regimen.

Earlier This Year:

 ::  January  ::  February  ::  March  ::  April  ::  May  ::  June  ::  July  ::  August  ::  September  ::  October  ::  November


Noted This Week Archive:

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  • The FDA granted Breakthrough Therapy designation to ribociclib (Kisqali; Novartis) for the initial treatment of premenopausal or perimenopausal women with HR-positive/HER2-negative advanced or metastatic breast cancer in combination with tamoxifen or an aromatase inhibitor. The decision was based on positive results in the phase III MONALEESA-7 clinical trial of 672 women in which those who received the ribociclib combination had significantly longer progression-free survival compared with those treated with endocrine therapy alone (a median of 23.8 vs. 13 months, respectively). Ribociclib is approved in the United States and Europe for use in postmenopausal women with this form of breast cancer.
  • Two Science studies independently discovered a genetic mechanism in cancer cells that affects how the cells respond to checkpoint inhibitors. One study focused on patients with advanced kidney cancer treated with checkpoint inhibitors, whereas the other examined the mechanism for immunotherapy resistance in mouse melanoma cells. Both teams found that changes in the genes that code for SWI/SNF, a group of proteins called a chromatin remodeling complex, affected resistance to checkpoint inhibitors, providing a possible explanation for the varied responses to the drugs.

December 15–28, 2017

  • San Diego, CA–based Ignyta will merge with Roche in a deal valued at $1.7 billion. Ignyta’s lead drug candidate is entrectinib, under development to treat non–small cell lung cancer that harbors ROS1 fusions, as well as a variety of solid tumors with NTRK fusions. The move will expand Roche’s portfolio of oncology drugs.
  • According to a report published in The New England Journal of Medicine, the higher a tumor’s mutational burden, the more likely it is to respond to checkpoint inhibitors. Researchers analyzed 27 different cancer types and found a strong correlation between high mutation burden and checkpoint inhibitor response for most of them; one outlier was Merkel cell carcinoma, which tends to have a moderate number of mutations yet responds well to the drugs. The findings, researchers say, could help guide clinical trials of checkpoint inhibitors for diseases in which they haven’t yet been tested.
  • The Institute for Clinical and Economic Review released its Draft Evidence Report assessing the comparative clinical effectiveness and value of two chimeric antigen receptor T-cell therapies—tisagenlecleucel (Kymriah; Novartis) and axicabtagene ciloleucel (Yescarta; Kite/Gilead)—for the treatment of certain B-cell cancers. The independent nonprofit research organization’s report concludes that the therapies “provide gains in quality-adjusted and overall survival over alternative chemotherapies” and that they “seem to be priced in alignment with clinical benefits over a lifetime.”
  • In a phase III study, lenalidomide (Revlimid; Celgene) in combination with rituximab (Rituxan; Genentech) did not prove superior to standard treatment with rituximab plus chemotherapy in patients with previously untreated follicular lymphoma. Lenalidomide is approved to treat multiple myeloma and certain patients with myelodysplastic syndrome.
  • Two companies announced that the FDA issued special designations to two products: The PD-L1 inhibitor avelumab (Bavencio; EMD Serono/Pfizer), in combination with axitinib (Inlyta; Pfizer) was granted Breakthrough Therapy designation as a first-line treatment for advanced renal cell carcinoma (RCC). The androgen receptor inhibitor apalutamide (Janssen) received Priority Review designation for the treatment of men with nonmetastatic castration-resistant prostate cancer who have a rapidly increasing prostate-specific antigen level despite receiving continuous androgen deprivation therapy.
  • The FDA also updated the label for nilotinib (Tasigna; Novartis) to include information on discontinuing the drug in patients with chronic phase Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML) who have achieved a sustained molecular response (MR4.5) to it after at least 3 years of treatment. Criteria for monitoring patients who discontinue nilotinib and guidance on restarting treatment if necessary are also spelled out.
  • In addition, the FDA approved/cleared a host of drugs and a radiotherapy device the week before Christmas. Among the announcements:
    • Based on data from the phase III CheckMate 238 trial, nivolumab (Opdivo; Bristol-Myers Squibb) was approved for adjuvant treatment of patients with melanoma with lymph node involvement or metastatic disease following surgery. A PD-1 inhibitor, nivolumab significantly improved recurrence-free survival compared with the company’s CTLA-4 inhibitor, ipilimumab (Yervoy).
    • The agency expanded the indication for cabozantinib (Cabometyx; Exelixis) to include patients with previously untreated RCC. The tyrosine kinase inhibitor had already been approved for patients with advanced RCC who previously received antiangiogenic therapy.
    • The agency also expanded the indication for bosutinib (Bosulif; Pfizer) to include patients with newly diagnosed chronic phase, Ph+ CML. This tyrosine kinase inhibitor was originally approved in 2012 for patients with chronic, accelerated, or blast phase Ph+ CML with resistance or intolerance to prior therapy.
    • Pertuzumab (Perjeta; Genentech/Roche) was approved, in combination with trastuzumab (Herceptin; Genentech/Roche) and chemotherapy, for the adjuvant treatment of HER2-positive early breast cancer at high risk of recurrence. Earlier accelerated approval of pertuzumab for neoadjuvant use was converted to full approval.
    • The GammaPod (Xcision Medical Systems) was cleared for use in noninvasive stereotactic delivery of radiation to discrete areas of the breast in conjunction with breast conserving treatment. The GammaPod has not been shown to be as effective as whole breast radiation therapy—and is not designed to replace it—although the new system minimizes the radiation dose to surrounding healthy tissues.

December 8–14, 2017

  • Jerusalem, Israel–based Teva Pharmaceuticals announced that it will eliminate more than 25% of its workforce over the next 2 years to reduce costs, simplify its organizational structure, and increase profitability. In addition to the layoffs, the company will close manufacturing plants, R&D facilities, and offices around the world, including in the United States. Teva is developing CT-P103, a biosimilar to Rituxan (rituximab; Genentech), and CT-P63, a biosimilar to Herceptin (trastuzumab; Genentech); also, it makes bendamustine hydrochloride (Bendeka) for chronic lymphocytic leukemia and arsenic trioxide (Trisenox) for acute promyelocytic leukemia.
  • More than half of 108 patients with aggressive non–Hodgkin lymphoma who received a single infusion of the CAR T-cell therapy axicabtagene ciloleucel (Yescarta; Kite Pharm) were still alive at least a year later, researchers reported concurrently in The New England Journal of Medicine and at the 2017 American Society of Hematology Annual Meeting in Atlanta, GA, on December 10. In addition, after a median follow-up period of 15.4 months, 42% of patients remained in remission, according to the latest analysis of data from the ZUMA-1 trial. Approved by the FDA in late October, axicabtagene ciloleucel targets the CD19 protein on lymphoma cells.
  • Cancer Research UK announced the signing of a 5-year drug-discovery collaboration between its subsidiary, Cancer Research Technology (CRT), and Celgene to discover, develop, and commercialize new anticancer treatments. The collaboration is centered on mRNA translation. Under the terms of the agreement, Celgene will pay an up-front fee, the amount of which was not disclosed, to CRT and have the option to secure U.S. rights to projects resulting from the collaboration, subject to the payment of additional option fees.
  • According to a study in JAMA Oncology, researchers found a lack of congruence in the genetic mutations uncovered by liquid biopsy tests from two separate commercial providers, suggesting that patients with the same mutations could be prescribed different treatments depending upon which company performs the liquid biopsy. Researchers compared Guardant360 (Guardant Health), which sequenced at least part of the coding sequences of 73 genes, and PlasmaSELECT (Personal Genome Diagnostics), which sequenced coding sequences of 64 genes. Even when the companies were analyzing DNA from the same blood drawn, their results rarely matched: When comparing results within the overlapping genetic sequences, the results from both companies completely matched in only 7.5% of cases.
  • Merck announced that pembrolizumab (Keytruda) did not prolong overall survival in patients with advanced gastric or gastroesophageal junction adenocarcinoma compared with paclitaxel when used as a second-line treatment for PD-L1–positive tumors. Additionally, the KEYNOTE-061 study found no statistically significant improvement in progression-free survival. In September, the FDA approved pembrolizumab as a third-line treatment for patients whose tumors express PD-L1 as determined by an FDA-approved test.

December 1–7, 2017

  • The FDA approved trastuzumab-dkst (Ogivri; Mylan) as a biosimilar to Genentech’s Herceptin (trastuzumab) for the treatment of patients with breast cancer or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors are HER2+. Trastuzumab-dkst is the first biosimilar approved for these diseases and the second approved to treat cancer. Biosimilars must be highly similar to an approved biological product and have no clinically meaningful differences in safety and effectiveness.
  • At the San Antonio Breast Cancer Symposium (SABCS) in Texas, researchers reported that, for postmenopausal women with HR-positive breast cancer, taking the aromatase inhibitor anastrozole for 2 years after an initial 5 years of adjuvant endocrine therapy is as effective as taking it for 5 additional years. Between 2004 and 2010, 3,484 patients were randomly assigned to receive either 2 years or 5 years of extended adjuvant therapy; as of June 2016, 78% of women in each group were alive without disease recurrence. However, longer treatment led to more bone fractures, suggesting that the shorter treatment duration may provide sufficient benefits and reduce the risk of harmful side effects.
  • Also at the SABCS, researchers reported that 9 weeks of adjuvant trastuzumab and chemotherapy was not as effective as 12 months of treatment in terms of disease-free survival for women with early-stage HER2-positive breast cancer after a median follow up of 5.2 years (88% and 90.5%, respectively). The finding supports the current practice of extended trastuzumab treatment. However, the study found no substantial difference in the secondary endpoints of distant disease-free survival and overall survival between the 9-week and 12-month treatment arms.
  • Clovis Oncology reported that the FDA has granted fast-tracked review of rucaparib to treat a broader range of women with ovarian cancer. The company is seeking approval of the use of the drug as a maintenance treatment in patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are sensitive to platinum-containing therapies—regardless of their genetic profile. Rucaparib was approved in 2016 for third-line treatment of BRCA-mutated ovarian cancer.
  • Researchers reported on their use of CRISPR/Cas9 screening of MYCN-amplified neuroblastoma to discover a preferential dependency on genes encoding PRC2 components, including EZH2. In addition, they found that “EZH2 represses neuronal differentiation in neuroblastoma in a PCR2-dependent manner” and that “MYCN-amplified and high-risk primary tumors from patients with neuroblastoma exhibited strong repression of EZH2-regulated genes.… Together, these observations demonstrate that MYCN upregulates EZH2, leading to inactivation of a tumor suppressor program in neuroblastoma, and support testing EZH2 inhibitors in patients with MYCN-amplified neuroblastoma.”
  • Bristol-Myers Squibb announced that the FDA lifted partial clinical holds on two trials of nivolumab (Opdivo), following amendments to study protocols, for the treatment of relapsed/refractory multiple myeloma: the phase I CheckMate-039 study of the tolerability of nivolumab combined with daratumumab; and the phase II CA204142 study of elotuzumab in combination with pomalidomide and low-dose dexamethasone, and in combination with nivolumab. A third multiple myeloma trial, CheckMate-062, remains on a partial clinical hold.

November 2017

November 22–30, 2017

  • The FDA approved Foundation Medicine’s FoundationOne CDx (F1CDx), the first breakthrough, next-generation sequencing–based in vitro diagnostic test, which can detect genetic mutations in 324 genes and two genomic signatures in any solid tumor type—and the Centers for Medicare & Medicaid Services proposed covering the cost of the test for eligible beneficiaries. Previously approved companion diagnostics match one test to one drug; based on individual test results, F1CDx can identify which patients with any of five tumor types may benefit from 15 different FDA-approved targeted treatment options. The test can also help determine patients’ candidacy for cancer clinical trials.
  • Tobacco companies began airing “corrective statements” to warn the public about the addictive nature of cigarettes—and the companies’ role in making cigarettes addictive—as well as the many health problems smoking can cause, including several cancers. A federal court in 2006 mandated the new advertisements, which will appear in newspapers and on television, the companies’ websites, and cigarette packaging for at least a year. The ruling stems from a 1999 lawsuit brought by the U.S. government that sought to recover billions of dollars in health care costs for tobacco-related illnesses.
  • According to a study published in JAMA Oncology, 11% of people ages 20 to 64—and 25% of adults age 65 and over—diagnosed with cancer between 2009 and 2013 had cancer earlier in their lives, meaning that they are often excluded from clinical trials. As the number of cancer survivors continues to grow, more and more patients could be prevented from participating in cancer research; investigators say that these patients may respond differently to some anticancer treatments than patients with a first cancer, and suggest that enrollment criteria should be broadened.
  • Merck KGaA and Pfizer announced that the phase III JAVELIN Gastric 300 trial did not meet its primary endpoint of superior overall survival with single-agent avelumab (Bavencio; EMD Serono) compared with physician’s choice of chemotherapy. The trial investigated avelumab as a third-line treatment for patients with inoperable, recurrent, or metastatic gastric or gastroesophageal junction adenocarcinoma regardless of PD-L1 expression.
  • Research published in Annals of Internal Medicine indicates that biennial biopsies may be an acceptable alternative to annual biopsies for men pursuing active surveillance for low-risk prostate cancer. The consequences of the different biopsy schedules were similar across four cohorts of men with a Gleason score between 2 and 6 with T1 or T2 prostate cancer. Given the invasiveness and potential morbidity of annual biopsies, less-frequent testing may be well justified, the analysis suggests.
  • Kyowa Hakko Kirin announced that the FDA granted Priority Review designation to mogamulizumab, a monoclonal antibody directed against CCR4, for the treatment of cutaneous T-cell lymphoma. The agency also granted Fast Track designation to IMO-2125 (Idera Pharmaceuticals), a toll-like receptor 9 agonist, in combination with ipilimumab for the treatment of anti–PD-1 refractory metastatic melanoma.

November 17–21, 2017

  • More than 45% of cancer deaths can be attributed to modifiable risk factors—smoking, excess weight, alcohol intake, poor diet, physical inactivity, ultraviolet radiation exposure, and infection with Helicobacter pylori or one of five cancer-associated viruses, such as human papillomavirus—according to a study published in CA: A Cancer Journal for Clinicians. Further, 42% of all incident cancers could be traced to these risk factors. The findings “underscore the vast potential for reducing cancer morbidity and mortality through … known preventive measures,” the researchers wrote.
  • The tax bill passed in the U.S. House of Representatives would repeal the Orphan Drug Tax Credit program, which credits companies for half of the costs associated with research and testing drugs for diseases affecting fewer than 200,000 people. The Hill reported that the move would save the federal government about $54 billion over the next decade, revenue that would compensate for tax cuts elsewhere in the budget. A tax bill under debate in the U.S. Senate would not end the program; rather, it would cut the credit from 50% to 27.5%.
  • Roche’s Genentech unit filed suit in federal court in Wilmington, DE, to block Pfizer from selling a biosimilar version of trastuzumab (Herceptin) in the United States. The suit alleges that PF-05280014, for which Pfizer is seeking FDA approval, would infringe upon 40 patents, some of which are set to expire in 2019, Bloomberg reported. Trastuzumab, which was approved by the FDA in 1998, treats HER2-positive breast cancer.
  • A study published in the Annals of Internal Medicine concludes that testing for novel protein biomarkers in stool finds significantly more colorectal cancers and advanced adenomas compared with testing for hemoglobin alone. By using a combination of four protein biomarkers, investigators found that they could detect almost twice as many colorectal cancers and five times as many advanced adenomas, precursors to cancer, than by testing for hemoglobin alone. Because the test uses the same technology as the current standard stool-based test, researchers say that it could easily be integrated into population-wide screening programs.
  • The European Medicines Agency approved the use of abiraterone with standard hormone therapy as a first-line treatment for advanced prostate cancer, defined as those cancers that are high-risk or have already metastasized. Abiraterone was previously approved for the treatment of advanced prostate cancer before chemotherapy. The decision was based on data from the international phase III LATITUDE trial, which showed that the combination had a clear benefit when used earlier.

November 10–16, 2017

  • MSK-IMPACT became the first tumor-profiling laboratory-developed test to earn FDA approval. Created by researchers at Memorial Sloan Kettering Cancer Center in New York, NY, the 468-gene panel uses next-generation sequencing (NGS) to rapidly detect mutations and other key genomic changes in patients' tumors. The FDA also unveiled a regulatory pathway to streamline the review of additional NGS-based tumor-profiling assays, and announced the New York State Department of Health's accreditation as a third-party reviewer of in vitro diagnostics, including tests similar to IMPACT.
  • The Centers for Disease Control and Prevention mapped out cancer trends and statistics on a state-by-state basis. Among various findings, the data for 2014—the most recent year available—showed that Utah had the lowest cancer death rate (127.8 deaths per 100,000 people) and New Mexico had the lowest incidence of new cancers (369.9 cases per 100,000 people). Meanwhile, Kentucky had both the highest cancer death rate and incidence of new cancers, at 199.1 deaths and 513.7 cases per 100,000 people, respectively.
  • Bayer paid Loxo Oncology $400 million up front to jointly develop larotrectinib and LOXO-195. The deal also includes $650 million for the Stamford, CT–based biotech in regulatory and commercial milestones. Larotrectinib, which inhibits the TRK receptor tyrosine kinase family, is being developed for patients whose tumors harbor NTRK fusions; LOXO-195 is a second-generation drug designed to address acquired resistance, through TRK point mutations, to larotrectinib.
  • San Francisco, CA–based Nektar Therapeutics reported positive data from its phase I/II study of NKTR-214, an investigational immunotherapy designed to expand and activate cytotoxic T cells and natural killer cells directly within the tumor microenvironment. NKTR-214 was evaluated alongside nivolumab (Opdivo; Bristol-Myers Squibb) in patients with melanoma, renal cell carcinoma, and non–small cell lung cancer; the last cohort had PD-L1–negative tumors. The objective response rates were 63%, 46%, and 75%, respectively.
  • Three organizations teamed up to improve access to cancer care in Sub-Saharan Africa, starting with Ethiopia, Uganda, and Tanzania. Funded by Novartis, the American Society for Clinical Pathology will focus on building healthcare capacity for IHC analysis in hospital laboratories, from better diagnostic equipment to well-trained staff. Meanwhile, the American Cancer Society will implement a program aimed at promoting the safe handling and administration of chemotherapy.

November 3–9, 2017

  • Most patients with colorectal cancer are not being screened for a common genetic mutation, according to a study published in JAMA Oncology that examined compliance with DNA mismatch repair (MMR) deficiency testing. Researchers found that only 28% of adult patients who had colorectal cancer surgery had MMR-deficiency testing on their tumors, 43% of whom ranged in age from 18 to 49. Testing is particularly important for patients in this age group because they have a higher incidence of Lynch syndrome, which can cause subsequent cancers.
  • Cellectis announced that the FDA released a clinical hold on phase I trials of UCART 123 in acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). The company agreed to implement revisions to the trial protocols, such as a dose reduction of the gene-edited T-cell investigational drug that targets CD123. The CD123 antigen is expressed on the surface of leukemic cells in AML, as well as on leukemic and other tumoral cells in BPDCN.
  • According to a statement from the American Society of Clinical Oncology (ASCO), alcohol use is linked with an increased risk of several malignancies, including breast, colon, esophagus, and head and neck cancers. The organization said that 5% to 6% of new cancers and cancer deaths globally can be directly attributed to alcohol—regardless of whether its use is light, moderate, or heavy. Researchers said that the finding was particularly troubling because, based on a survey conducted earlier this year, 70% of Americans do not consider alcohol consumption a risk factor for cancer.
  • The FDA approved alectinib (Alecensa; Roche) for as a first-line treatment for patients with ALK-positive non–small cell lung cancer (NSCLC). The approval was based on results from the phase III ALEX study, which demonstrated that alectinib significantly improved progression-free survival compared with standard crizotinib (Xalkori; Pfizer) treatment. The agency also converted the drug’s initial accelerated approval in 2015 to full approval for the treatment of people with ALK-positive, metastatic NSCLC who experienced disease progression on, or intolerance to, crizotinib.
  • Merck announced that its letermovir (Prevymis) received FDA approval for the prevention of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant. In the pivotal phase III trial supporting the approval, significantly fewer patients who received letermovir, which inhibits viral replications, developed CMV infection compared with a placebo group—38% vs. 61%, respectively. All-cause mortality after 24 weeks was also lower among patients who received letermovir compared with a placebo—12% vs. 17%, respectively.
  • The FDA announced its intent to allow genetic health risk (GHR) tests to be exempted from premarket review, under certain conditions, to speed patients’ access to innovative new devices. If and when the rule is finalized, manufacturers of these types of tests would have to undergo a one-time review to ensure that they meet the FDA’s requirements, after which they could commercialize new GHRs without additional agency review. In its notice, the FDA also outlined requirements to ensure the tests’ accuracy, reliability, and clinical relevance.

October 27–November 2, 2017

  • The FDA granted accelerated approval to acalabrutinib (Calquence; AstraZeneca) for adults with mantle cell lymphoma who have received at least one prior therapy. Acalabrutinib, which blocks Bruton tyrosine kinase (BTK), is a second-generation drug designed to be more potent and selective than the first-in-class BTK inhibitor ibrutinib (Imbruvica; Janssen). The agency based its decision on data from a single-arm study of 124 patients, in which 81% had a complete or partial response to treatment.
  • A team of experts outlined future cancer research priorities in the United States, which were published in The Lancet Oncology. The group delved into and expanded the National Cancer Moonshot Blue Ribbon Panel’s original recommendations, issued in 2016. For instance, they proposed constructing a Premalignant Cancer Atlas, noting that “the rate-limiting step in developing and implementing precision-based prevention approaches has been our limited understanding of precancer biology…in contrast with the extensive knowledge of advanced disease.”
  • The FDA warned four companies about marketing marijuana-derived products with unsubstantiated therapeutic claims. Greenroads Health, Natural Alchemist, That’s Natural! Marketing and Consulting, and Stanley Brothers Social Enterprises LLC were accused of illegally selling products that claim to prevent, diagnose, treat, or cure cancer without any supporting evidence. These products contain cannabidiol, which is not FDA-approved in any drug for any indication.
  • According to Reuters, two members of the United States House of Representatives questioned the operation of the International Agency for Cancer Research (IARC), part of the World Health Organization. Specifically, Lamar Smith (R-TX) and Andrew Biggs (R-AZ) expressed concern “about the scientific integrity” of the IARC’s monograph program, which assesses the potential carcinogenic effects of various substances. Calling for the IARC to make its scientific review process public, they also asked the agency to testify about its work.
  • Less than 5% of approximately 9 million Americans at high risk of lung cancer have been screened, the American Lung Association reported. According to the association’s fourth annual Lung Health Barometer, 84% of this high-risk population were unfamiliar with the low-dose CT scan—which can save lives through early detection—largely because it was never recommended by their doctors. Only 15% were aware that lung cancer screening is covered by Medicare and most health care plans at no cost.

October 2017

October 20–26, 2017

  • The majority of Americans are unaware of key cancer risk factors, according to a national survey carried out by the American Society of Clinical Oncology. Less than a third knew that obesity and alcohol consumption increase cancer risk; meanwhile, cell phones and caffeine were incorrectly identified as risk factors by 14% and 8% of those polled, respectively. The survey also found that 1 in 4 Americans facing cancer, either themselves or a family member, often forgo treatment or physician visits, due to the high costs involved.
  • Stand Up To Cancer (SU2C) launched four “Cancer Interception” teams aimed at detecting lung cancer and pancreatic cancer as early as possible. The groups will receive a total of $16.6 million in funding from SU2C, the Lustgarten Foundation for Pancreatic Cancer Research, LUNGevity, and the American Lung Association. Their research efforts include testing new preoperative treatments to allow more patients with pancreatic cancer to achieve complete tumor resection, and developing a blood-based lung cancer interception assay that can be used in conjunction with low-dose CT scans.
  • New genetic variants that contribute to breast cancer risk were identified by the OncoArray Consortium, a global collaboration of 550 researchers. Analyzing data from 275,000 women, the group pinpointed 65 new loci associated with overall breast cancer risk. An additional 10 variants correlated specifically with the risk of ER-negative disease, which doesn’t respond to endocrine therapy. The findings were published in Nature and in Nature Genetics, respectively.
  • According to the Boston Globe, Massachusetts General Hospital (MGH) became the first in the United States to start using a new mammography system called Pristina. Designed by General Electric Co., the system’s hand-held remote allows patients, rather than medical professionals, to control the pressure of breast tissue compression, an uncomfortable but necessary part of screening. Pristina recently earned FDA approval, and MGH plans to study how it works for patients.
  • The FDA launched an educational campaign about biosimilars, posting fact sheets and graphics to a new website aimed at helping health care professionals gain a better understanding of these important products and the approval process they undergo, according to an agency blog post. The FDA also pointed prescribers and patients to its Purple Book, which catalogs drugs for which approved biosimilars are available.
  • Wilmington, DE–based Incyte paid MacroGenics $150 million up front for global rights to MGA012, an investigational PD-1 inhibitor. The deal with the Rockville, MD–based biotech also includes $750 million in development, regulatory, and commercial milestones. MGA012 is currently undergoing phase I evaluation as a monotherapy for four solid tumor types.

October 13–19, 2017

  • Norman Sharpless, MD, was sworn in as the NCI’s 15th director. Previously, he headed the University of North Carolina’s Lineberger Comprehensive Cancer Center in Chapel Hill. Sharpless succeeds Harold Varmus, MD, who stepped down in 2015; Douglas Lowy, MD, who had served as acting director since then, resumes his role as the institute’s deputy director.
  • The FDA approved axicabtagene ciloleucel (Yescarta; Gilead/Kite) for adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Axicabtagene ciloleucel, a CD19-targeting chimeric antigen receptor (CAR) T-cell therapy, is the second in this class of agents to earn approval within the last 2 months, and the first such therapy for DLBCL. The FDA based its decision on a phase II trial in which 72% of 101 patients given a single infusion of CAR T cells responded to treatment, including a complete remission rate of 51%.
  • San Diego, CA–based Ignyta announced positive results from its phase II trial evaluating entrectinib in patients with advanced or metastatic non–small cell lung cancer whose tumors harbor ROS1 fusions. Among 32 patients, 78% had durable responses to entrectinib, and the median progression-free survival was 29.6 months; the investigational tyrosine kinase inhibitor was also active against brain metastases.
  • The FDA granted Priority Review to olaparib (Lynparza; AstraZeneca) to treat patients with germline BRCA1/2-mutant, HER2-negative metastatic breast cancer. If approved, this will be the first indication for a PARP inhibitor beyond ovarian cancer. The agency’s decision was based on a phase III trial, in which patients who received olaparib had a 42% reduction in their risk of disease progression or death, compared with those given standard chemotherapy.
  • Eli Lilly signed a $1.8 billion deal with CureVac to jointly develop up to five mRNA cancer vaccines. Lilly will pay the German biotech $100 million up front to jump-start this collaboration, which involves using CureVac’s proprietary RNActive technology to design mRNA-delivering vaccines that direct the immune system to target tumor-specific neoantigens.
  • The first screening recommendations for children at risk for retinoblastoma were issued by the American Association of Ophthalmic Oncologists and Pathologists. According to the report, at-risk children—defined as having a family history of retinoblastoma in a parent, sibling, or first- or second-degree relative—should be screened from birth up to the age of 7 years. After that, only known RB1 mutation carriers should have examinations every 1 to 2 years indefinitely.

October 6–12, 2017

  • The NIH launched its Partnership for Accelerating Cancer Therapies (PACT), a collaboration with 11 major biopharmaceutical companies, including AbbVie, Bristol-Myers Squibb, and GlaxoSmithKline. PACT’s first aim is to identify, develop, and validate biomarkers of response and resistance to immunotherapy. The 5-year alliance, part of the Cancer Moonshot initiative, will receive up to $215 million in funding from its members.
  • Stand Up To Cancer (SU2C) awarded 10 SU2C Catalyst clinical trial grants totaling more than $25 million. Researchers from more than 30 institutions will collaborate with nine pharmaceutical companies to explore new uses for various therapeutics—both approved and investigational agents, used singly or in combination with other drugs. The trials will involve patients with a variety of cancers, including breast, lung, ovarian, pancreatic, hypermutant pediatric, and urothelial cancers, as well as melanoma, multiple myeloma, and sarcoma.
  • Seven cancer therapies were among 19 drugs flagged by the FDA for potential signals of serious risks or new safety information. These drugs, which appeared on the agency’s latest watch list—posted quarterly and derived from the FDA Adverse Event Reporting System—included imatinib (Gleevec; Novartis) and pembrolizumab (Keytruda; Merck), whose labels were revised to warn about renal toxicity and Stevens-Johnson syndrome, respectively.
  • The FDA cleared Magnetom Terra (Siemens), a seven tesla (7T) MRI device, for clinical use. This is the first 7T system to receive clearance; it more than doubles the static magnetic field strength available for use in the United States, and will enable “better visualization of smaller structures and subtle pathologies that may improve disease diagnosis,” the agency said. Magnetom Terra is intended for patients who weigh more than 66 pounds, and is limited to examinations of the head, arms, and legs.
  • Eli Lilly’s CDK4/6 inhibitor abemaciclib (Verzenio) was granted Priority Review by the FDA as initial treatment for patients with advanced or metastatic ER+, HER2- breast cancer. The agency’s decision, based on positive data from the MONARCH3 study, comes on the heels of abemaciclib’s recent indication, as monotherapy or combined with fulvestrant, to treat disease that has progressed despite endocrine therapy.
  • Eli Lilly reported that a phase III study evaluating abemaciclib as monotherapy for KRAS-mutant advanced non–small cell lung cancer did not meet its primary endpoint of overall survival. However, an analysis of the trial’s secondary endpoints—progression-free survival and objective response rate—showed evidence of antitumor activity in the abemaciclib arm, which was “encouraging,” the company said. Data from this study will be submitted for presentation at a medical meeting in 2018.

September 29–October 5, 2017

  • Being overweight or obese is associated with 40% of all cancers diagnosed in the United States, the Centers for Disease Control and Prevention (CDC) reported. Two in three U.S. adults weigh more than recommended, the CDC noted, and more than half of Americans aren’t aware that excessive weight increases the risk of at least 13 cancer types, including meningioma, esophageal adenocarcinoma, multiple myeloma, and colorectal cancer.
  • Breast cancer death rates dropped by 39% between 1989 and 2015, according to the American Cancer Society’s (ACS) latest analysis. This decline was attributed to improved treatments and to early detection by mammography. Even so, the breast cancer death rate remained higher among African American women compared with Caucasian women, the ACS said, reflecting not only social and economic disparities, but biological complexities as well.
  • According to a study in the BMJ, most oncology drug indications recently approved by the European Medicines Agency lacked clear evidence of meaningful clinical benefit. Of 68 indications approved between 2009 and 2013, evidence of prolonged survival was lacking for 44 at the time of market authorization, the researchers reported. With a median of 5.4 years’ follow-up, only three of the 44 were subsequently found to extend life, and five were associated with improved quality of life.
  • The NIH announced plans to fund 12 specialized research centers aimed at improving minority health and reducing health disparities. Each Center of Excellence (COE) will carry out innovative multidisciplinary studies that “directly and demonstrably impact” at least one “health disparity population”—for instance, underserved rural populations, ethnic minorities, and socially disadvantaged groups, all of whom experience a disproportionate burden of preventable diseases, including cancer. The 12 COEs will share approximately $82 million over 5 years.
  • The American Society of Clinical Oncology and Friends of Cancer Research called for broadening eligibility criteria in clinical trials. Working with the FDA, both groups identified five areas where current criteria were most likely to restrict patient participation in a trial, but least likely to affect safety: minimum age requirements, HIV/AIDS status, brain metastases, organ dysfunction, and prior/concurrent malignancies. “To maximize generalizability of results, trial enrollment criteria should strive for inclusiveness,” they wrote.
  • Endocyte paid German drugmaker ABX GmbH $12 million up front for the rights to Lu-PSMA-617, a radiopharmaceutical that delivers lutetium selectively to PSMA-expressing tumor cells. The West Lafayette, IN–based company described Lu-PSMA-617 as “phase III ready,” with the potential to be “the first-in-class radioligand therapeutic to address both bone and soft tissue disease” in patients with metastatic castration-resistant prostate cancer. A phase III trial is planned for early 2018.

September 2017

September 22–28, 2017

  • The FDA launched a public dashboard to enable better access to reports of adverse drug reactions. The interactive web-based tool is designed so consumers can easily query the FDA’s Adverse Event Reporting System (FAERS) using various criteria, including biological product, patient age, specific time frame, and type of adverse event. The agency hopes this increased transparency through FAERS spurs the submission of more detailed safety reports from health care professionals and the pharmaceutical industry.
  • According to MedCityNews, FDA Commissioner Scott Gottlieb, MD, intends to have Congress develop legislation around laboratory-developed tests (LDT). LDTs are “no longer a mom-and-pop industry” where the agency “can continue to just exercise enforcement discretion,” he said. The news outlet noted that this means the FDA is moving away from its plan to phase in LDT regulation—albeit without input from Congress—over the next decade, which it first announced in 2014 but has since delayed finalizing amid uncertainty following the 2016 general election.
  • The Susan G. Komen Foundation announced its first crowdfunding initiative, slated to begin in October. It plans to highlight four researchers whose work focuses on metastatic breast cancer and encourage donations to their specific grants. This will give the public an opportunity to directly affect breast cancer research, Komen said.
  • The Journal of the National Comprehensive Cancer Network (NCCN) reported that 29% of breast cancer clinical trials did not have a control arm meeting the current standard of care. The researchers analyzed 210 phase III studies and found that the control arm was less likely to be consistent with NCCN guidelines—mainly in terms of recommended treatment regimen, as well as dosing and/or schedule—in trials involving women with early-stage breast cancer, trials recruiting in four or more countries, or studies not recruiting in the United States.
  • Next spring, the NIH will widen enrollment in its “All of Us” project, a key part of the Precision Medicine Initiative, according to the Associated Press. For now, the project is in an invitation-only pilot phase; approximately 50 sites have already recruited more than 2,500 participants to provide blood samples for genomic analyses, as well as environmental and socioeconomic data. By opening this study to any U.S. adults interested in joining, the NIH hopes to recruit a highly diverse population, especially underrepresented minorities.
  • Two new indications for pembrolizumab (Keytruda; Merck) and nivolumab (Opdivo; Bristol-Myers Squibb) were granted by the FDA. Pembrolizumab received accelerated approval to treat recurrent advanced or metastatic gastric or gastroesophageal junction adenocarcinoma expressing PD-L1. Nivolumab received accelerated approval for hepatocellular carcinoma previously treated with sorafenib (Nexavar; Bayer).

September 15–21, 2017

  • The NIH proposed updating its Genomic Data Sharing Policy—specifically, easing researchers’ access to genomic summary results (GSR), or “aggregated statistics from all participants in a genomic research study or set of studies,” according to an agency blog post. This update would allow GSR from most studies to be provided via a public, rapid-access model, reserving the current controlled-access model—in which requests from researchers are subject to committee review and approval—for sensitive studies. The NIH is accepting public comments on this plan until October 20.
  • The FDA approved Dako PD-L1 IHC 28-8 pharmDx (Agilent Technologies) as a complementary diagnostic for two more indications: urothelial carcinoma, and squamous cell carcinoma of the head and neck. The PD-L1 assay can help physicians determine which patients are most likely to benefit from nivolumab (Opdivo; Bristol-Myers Squibb). It was previously greenlighted for nonsquamous non–small cell lung cancer and melanoma.
  • Japanese drugmaker Taiho Oncology agreed to pay Arcus Biosciences $35 million for access to the latter’s immunotherapy portfolio. Taiho will also offer another $275 million for each drug it elects to license over the deal’s 5-year term. Arcus, based in Hayward, CA, is actively pursuing targets along the ATP–adenosine pathway, including CD73 and the A2A/A2B receptors; the idea is that blocking this pathway could disrupt a mechanism that otherwise suppresses antitumor immunity.
  • In a study published in Science Translational Medicine, researchers described how modified poliovirus unleashes the immune system against cancer. The virus binds directly to and kills tumor cells that express its receptor, CD155; it also infects antigen-presenting cells, including macrophages, thereby triggering T cells into tumor-attacking mode. Modified poliovirus was designated a Breakthrough Therapy by the FDA last year, based on promising data from early clinical trials in patients with recurrent glioblastoma.
  • Novartis and The Max Foundation announced a new collaboration to continue providing free access to imatinib (Gleevec) to patients in lower-income countries. Called CMLPath to Care, the new program evolved from GIPAP, created by the two organizations 15 years ago, which has enabled roughly 75,000 patients with chronic myeloid leukemia, gastrointestinal tumors, and other rare cancers to receive imatinib at no cost. The Max Foundation will now assume from Novartis the responsibility of managing the entire medicine supply chain and interactions with local stakeholders in nearly 70 countries.
  • As it works on appropriations for fiscal year 2018, Congress was actively reminded of the importance of NIH funding. At the fifth annual Rally for Medical Research Hill Day, organized by the American Association for Cancer Research (AACR), leaders from nearly 400 medical and scientific organizations met with House and Senate members to advocate for robust, sustained, and predictable budget increases for the NIH. The AACR issued a similar call to action in its recently released 2017 Cancer Progress Report.

September 8–14, 2017

  • The FDA greenlighted its first biosimilar for cancer, Amgen’s bevacizumab-awwb (Mvasi). The agency’s decision was based on extensive analytic data demonstrating Mvasi’s high similarity to Genentech’s bevacizumab (Avastin). Approved indications for this biosimilar include metastatic colorectal cancer, metastatic renal cell carcinoma, glioblastoma, and cervical cancer.
  • Follicular lymphoma got a new therapy, with the FDA granting accelerated approval to copanlisib (Aliqopa; Bayer), which inhibits PI3Kα and PI3Kδ. Patients with this type of indolent non–Hodgkin lymphoma often experience disease recurrence even after multiple treatments and have few therapeutic options. In a single-arm trial of 104 patients who had relapsed after at least two prior therapies, 59% had a complete or partial response to copanlisib for a median of 12.2 months.
  • JAMA Internal Medicine published a paper in which researchers pegged the cost of developing a single cancer drug in the United States at $648 million; median revenue for the 10 drugs considered in the analysis was calculated to be $1,658.4 million after a median of 4 years on the market. An earlier estimate from the Tufts Center for the Study of Drug Development put the cost of bringing a single drug to market at $2.7 billion, adjusted to 2017 dollars.
  • French biotech Onxeo reported disappointing phase III results with Livatag, its nanoparticle formulation of doxorubicin, in patients with liver cancer whose disease had progressed on sorafenib (Nexavar; Bayer). There were no significant differences in efficacy between patients randomly assigned to receive Livatag versus physician’s choice of therapy.
  • The U.S. Preventive Services Task Force (USPSTF) issued a draft recommendation for cervical cancer screening, proposing that women in their 20s have a Pap test every 3 years. For women ages 30 to 65, the group suggests a Pap test every 3 years or testing for high-risk strains of human papillomavirus every 5 years. The USPSTF previously recommended that women ages 21 to 65 receive a Pap test every 3 years or, for women ages 30 to 65, a Pap test and HPV testing every 5 years.
  • Bavarian Nordic discontinued its global phase III trial of Prostvac in patients with metastatic castration-resistant prostate cancer, based on an independent data monitoring committee’s futility analysis. The Danish biotech had been evaluating whether its poxvirus-based vaccine, alone or combined with the cytokine GM-CSF, could prolong overall survival in men with asymptomatic or minimally symptomatic disease.
  • Bristol-Myers Squibb (BMS) agreed to pay Halozyme $105 million for access to the latter’s ENHANZE technology. BMS will use this platform to develop versions of its drugs, including nivolumab (Opdivo), that can be administered subcutaneously rather than intravenously. ENHANZE is based on a proprietary enzyme that temporarily degrades hyaluronan, thereby aiding the dispersion and absorption of injected therapeutics, and potentially speeding drug delivery.

September 1–7, 2017

  • Strong efficacy data prompted early termination of a phase III trial evaluating two checkpoint inhibitors—Bristol-Myers Squibb’s (BMS) nivolumab (Opdivo) and ipilimumab (Yervoy)—in untreated advanced or metastatic renal cell carcinoma. Overall survival was significantly improved among patients randomly assigned to receive this combination, compared with those given sunitinib (Sutent; Pfizer), a multi-tyrosine kinase inhibitor. Full results will be presented at the European Society for Medical Oncology 2017 Congress, which starts on September 8, the company said.
  • Three BMS trials evaluating nivolumab-based combinations in multiple myeloma were placed on partial clinical hold by the FDA. The move comes a week after the agency issued a safety alert on the use of pembrolizumab (Keytruda; Merck) for the disease, in combination with dexamethasone and the immunomodulatory agents lenalidomide or pomalidomide. For now, the FDA has determined that the risks of such combination therapies for multiple myeloma outweigh the potential benefit, BMS said; no new patients will be enrolled in these trials.
  • The FDA also placed two phase I trials of UCART123 (Cellectis) on clinical hold after a treatment-related death was reported. The patient, who had blastic plasmacytoid dendritic cell neoplasm, experienced severe cytokine release syndrome and capillary leak syndrome, adverse events that suggest this “universal” chimeric antigen receptor (CAR) T-cell therapy—created by gene-editing cells from a healthy, unmatched donor, rather than from each individual patient—“may be affected by safety issues both general to CAR-Ts and specific to its targeting of CD123,” according to FierceBiotech.
  • Eli Lilly announced plans to cut about 3,500 jobs globally, a move expected to yield annual savings of $500 million starting in 2018. The cuts translate to a workforce reduction of roughly 8% and will mainly come from a voluntary early-retirement program offered in the United States, the company said; several facilities in Iowa, New Jersey, and China will also be closed.
  • GlaxoSmithKline licensed the rights to Adaptimmune’s investigational T-cell therapy targeting the NY-ESO peptide. Adaptimmune uses its proprietary technology, dubbed SPEAR, to engineer T-cell receptors (TCR) with “enhanced affinity,” thereby strengthening natural T cell–driven antitumor responses in patients. GlaxoSmithKline will pay the Oxfordshire, England–based biotech up to $63 million for NY-ESO TCR, which is being evaluated in six tumor types, including synovial sarcoma and multiple myeloma.
  • Sanofi/Regeneron’s cemiplimab earned Breakthrough Therapy status from the FDA for adults with locally advanced, metastatic, or inoperable cutaneous squamous cell carcinoma (CSCC). This will expedite the PD-1 inhibitor’s development for CSCC, the second deadliest skin cancer after melanoma. Earlier this year, the companies reported promising preliminary data from 26 patients, with an overall response rate to cemiplimab of 46%.

August 2017

August 25–31, 2017

  • Novartis’s tisagenlecleucel (Kymriah) became the first CAR T-cell therapy to earn FDA approval for patients up to 25 years of age with relapsed/refractory acute lymphoblastic leukemia. The company is establishing a network of certified treatment centers throughout the United States, where staff will be fully trained on the use of this one-time treatment and appropriate patient care. Additional filings for tisagenlecleucel are also planned, including for adults with diffuse large B-cell lymphoma.
  • Staking its place in the emerging field of cell therapy, Gilead Sciences inked a deal to buy Santa Monica, CA–based Kite Pharma for $11.9 billion. This will give Gilead access to Kite’s robust CAR T-cell therapy pipeline. Its lead candidate, axicabtagene ciloleucel, is expected to be approved later this year, which would make it the first such treatment for patients with relapsed/refractory non–Hodgkin lymphoma.
  • The FDA issued a safety alert on the use of pembrolizumab (Keytruda; Merck) for multiple myeloma, in combination with dexamethasone and the immunomodulatory agents lenalidomide or pomalidomide. Pembrolizumab is not approved for this disease; the agency’s move was based on interim data analyses from two phase III trials—currently on clinical hold—in which the relative risk of death more than doubled among patients in the treatment arm, compared with the control group.
  • Irish drugmaker Jazz Pharmaceuticals paid ImmunoGen $75 million for the rights to two early-stage antibody–drug conjugates (ADC) being developed by the Waltham, MA–based biotech for hematologic malignancies. A phase I trial of CD33-targeting IMGN779 for acute myeloid leukemia is already under way; IMGN632, which targets CD123, is expected to enter clinical testing later this year.
  • Five Prime Therapeutics terminated its immuno-oncology collaboration with InhibRx, valued at more than $460 million when it was signed 2 years ago. The two biotechs—in San Francisco, CA, and La Jolla, CA, respectively—had been working together on antibodies targeting GITR that would both inhibit immunosuppressive T cells and spur tumor-attacking effector T cells into action.
  • The FDA took action against StemImmune, Inc., in San Diego, CA, and the California Stem Cell Treatment Centers, halting their treatment of cancer patients with a potentially dangerous stem cell product. Five vials of the live Vaccinia virus—intended only for people at high risk of smallpox—were seized; the virus had been used to create this unapproved therapy, which was injected directly into patients’ tumors.
  • The FDA designated DS-8201 (Daiichi Sankyo) a Breakthrough Therapy for patients with HER2+ locally advanced or metastatic breast cancer previously treated with trastuzumab (Herceptin; Roche) and pertuzumab (Perjeta; Roche), whose disease has progressed on ado-trastuzumab emtansine (Kadcyla; Roche). DS-8201, an ADC targeting HER2, contains a novel topoisomerase I inhibitor, DXd, as its cytotoxic payload. Preliminary clinical evidence suggests it could considerably benefit a patient population that currently lacks other options, the company said.

August 18–24, 2017

  • Vaccination rates for human papillomavirus (HPV) were found to be significantly lower among childhood cancer survivors compared with the general population—24% versus 40%. Lack of a physician’s proactive recommendation, reported by 72% of 982 childhood cancer survivors surveyed, was the most significant barrier to vaccination. Childhood cancer survivors are particularly susceptible to HPV infection, due to cancer treatment often suppressing their immune defenses, so they stand to benefit from vaccination even more than their peers.
  • Eight Senate Democrats urged FDA Commissioner Scott Gottlieb, MD, to pull menthol cigarettes from the market, using the authority provided by the Tobacco Control Act. In a letter, they noted “substantial scientific data” showing that menthol cigarettes “frequently serve as a starter product for youth, are associated with increased nicotine dependence in young smokers, and make it more difficult to quit smoking.”
  • Fewer than 1 in 5 women with a family history of breast or ovarian cancer have been tested for BRCA1/2 mutations, an analysis of pooled data from the 2005, 2010, and 2015 National Health Interview Surveys found. Up to 15% of these cancers can be attributed to heritable mutations—which, if identified, provide critical knowledge for treatment and preventive care—so large national efforts are warranted to increase genetic testing rates, the researchers said.
  • Long-term use of vitamin B6 and B12 supplements correlated with a 30% to 40% higher risk of lung cancer among men, but not women, according to a report in the Journal of Clinical Oncology. The increase was tied to individual supplement sources, not multivitamins, and was particularly pronounced among smokers. “This sex- and source-specific association provides further evidence that vitamin B supplements are not chemopreventive for lung cancer and may be harmful,” the researchers concluded.
  • CRISPR/Cas9–based research got a $3.2 million funding boost from the National Human Genome Research Institute. The University of California, Berkeley, received $2.1 million to set up the Center for Genome Editing and Recording, where technologies will be developed to interrogate DNA sequences at a scale and level of accuracy not presently feasible. Meanwhile, another $1.1 million went to the Broad Institute in Cambridge, MA, which will be used to engineer new and improved RNA-targeting Cas enzymes, among other tools to better dissect gene expression dynamics.
  • The FDA designated mogamulizumab (Kyowa Hakko Kirin) a Breakthrough Therapy for patients with mycosis fungoides and Sézary syndrome. This will expedite mogamulizumab’s development for the two most common subtypes of cutaneous T-cell lymphoma. The CCR4-targeting antibody is being evaluated against FDA-approved vorinostat (Zolinza; Merck), a histone deacetylase inhibitor, in a global phase III trial.

August 11–17, 2017

  • The FDA approved inotuzumab ozogamicin (Besponsa; Pfizer) for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The approval was based on data from the phase III INO-VATE ALL study. Inotuzumab ozogamicin is an antibody–drug conjugate composed of a humanized IgG4 anti-CD22 antibody linked to the cytotoxic agent CalichDMH; upon binding to B cell–specific CD22 receptors, the cell internalizes the drug, which causes double-strand DNA breaks and apoptosis.
  • Also, the FDA OK’d the PARP inhibitor olaparib (Lynparza; AstraZeneca) in tablet form for maintenance treatment of patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who experience a complete or partial response to platinum-based chemotherapy. In 2014, the agency approved olaparib capsules for the treatment of patients with germline BRCA-mutated advanced ovarian cancer who had received three or more lines of chemotherapy; because the capsules are being phased out, the new olaparib tablets have been approved for this indication. However, the tablets and capsules are not interchangeable, the FDA says.
  • The PD-1 inhibitor pembrolizumab (Keytruda; Merck) demonstrated a favorable safety profile and promising, durable clinical activity in patients with advanced small cell lung cancer (SCLC) with high PD-L1 levels who had previously been treated with platinum-based chemotherapy. Out of 24 evaluable patients, the overall response rate was 33%, with one complete response. Based on the strength of the findings, additional trials of pembrolizumab in patients with SCLC have been launched.
  • Bristol-Myers Squibb announced that its PD-1 inhibitor, nivolumab (Opdivo), combined with its CTLA4 inhibitor ipilimumab (Yervoy), did not significantly improve progression-free survival compared with standard-of-care sunitinib (Sutent; Pfizer) in patients with previously untreated advanced or metastatic renal cell carcinoma. However, the company said that the combination did yield a higher objective response rate (41.6% vs. 26.5%) and a longer median duration of response (not reached vs. 18.17 months) than sunitinib.
  • Patients with advanced non–small cell lung cancer with high PD-L1 expression and no targetable mutations should receive pembrolizumab as first-line therapy, according to updated treatment guidelines from the American Society of Clinical Oncology; other checkpoint inhibitors, either alone or in combination with another drug, are not recommended. However, some recommendations have not changed. For example, patients with EGFR mutations or ALK or ROS1 rearrangements should still initially receive targeted therapy.
  • Rigorous confirmatory studies to validate the FDA’s accelerated approval of drugs are lacking, according to research published in the Journal of the American Medical Association that reviewed the 22 drugs that earned accelerated approval in 24 indications from 2009 to 2013. For the 10 accelerated approvals (all cancer drugs) that subsequently met FDA requirements, confirmatory studies tested surrogate measures. Although overall survival is considered to be “the most dependable end point in clinical trials of cancer drugs,” the study authors wrote, “overall survival was among the prespecified primary end points in only 5% of required confirmatory studies.”
  • The ROS1ders (pronounced “ross wonders”), a group of patients with ROS1 rearrangements, has teamed up with the Addario Lung Cancer Medical Institute and Champions Oncology to aid in the creation of a collection of PDX mouse models and cell lines using their tumor tissue. Genomic sequencing data and patient medical histories will be linked to the models. ROS1 rearrangements are found in a wide variety of cancer types but are relatively uncommon, occurring in 1% to 3% of lung, gastric, and ovarian cancers, as well as melanoma, cholangiocarcinoma, glioblastoma, and other cancers.

August 4–10, 2017

  • Anti-CTLA4 and anti–PD-1 immunotherapies expand distinct immune infiltrates against cancer, according to a study published in Cell, uncovering the cellular mechanisms used by each type of checkpoint inhibitor and explaining why combining these agents works better than using them as monotherapy. The researchers noted that anti–PD-1 therapy “predominantly induces the expansion of specific tumor-infiltrating exhausted-like CD8 T cell subsets. In contrast, anti-CTLA-4 induces the expansion of an ICOS+ Th1-like CD4 effector population in addition to engaging specific subsets of exhausted-like CD8 T cells.” The research boosts understanding of the basic science behind the drugs, which could lead to more effective use in the clinic.
  • The Centers for Disease Control and Prevention (CDC) launched an online database that includes detailed, deidentified information on several million cancer cases reported between 2001 and 2014. Available at no charge, the data can be analyzed by researchers and the general public to better understand cancer, inform prevention efforts, and evaluate progress in cancer control. This tool, dubbed the United States Cancer Statistics Database, combines data from the CDC’s National Program of Cancer Registries and the NCI’s Surveillance, Epidemiology, and End Results Program.
  • The Discovery Channel aired the first in a three-part series called “First in Human,” which provides a look inside the NIH Clinical Center in Bethesda, MD, and follows four patients who volunteered to take part in clinical trials. Noting that one survey found that 16% of all patients—and just 3% of patients with cancer—have taken part in a trial, NIH Director Francis Collins, MD, PhD, wrote, “I hope we can begin to build momentum to encourage more Americans to take part in clinical trials.” The remaining episodes in the series will air at 9 pm ET on August 17 and 24.
  • The FDA announced a new public education campaign aimed at discouraging the use of electronic cigarettes (e-cigarettes) and other electronic nicotine delivery systems by kids. The campaign, which will kick off this fall, will include messaging to teens, a growing number of whom are using e-cigarettes, about the health risks associated with these products. The endeavor is part of the agency’s new comprehensive plan for tobacco and nicotine regulation, as well as its ongoing educational efforts.
  • The FDA designated the novel immunotherapy vaccine SurVaxM (MimiVax LLC) as an Orphan Drug for the treatment of glioblastoma. Also known as DRU-2017-5947, the vaccine targets survivin, a cell-survival protein present in most cancers, suggesting that it could be used against other cancers. A phase II trial of SurVaxM in addition to standard treatment in patients with newly diagnosed glioblastoma is currently under way.

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July 2017

July 28–August 3, 2017

  • The Senate overwhelmingly passed a bill that reauthorizes the FDA to collect fees from drug companies and medical device manufacturers for the next 5 years, money that accounts for more than a quarter of the agency’s budget, according to The Hill. The funds are used to help speed up the review of new agents and devices. Just one senator opposed the measure, which was already approved by the House; the legislation will now be sent to President Donald Trump for his signature.
  • Stamford, CT–based Loxo Oncology purchased Redx Pharma’s BTK inhibitor program for $40 million. The sale includes the investigational agent LOXO-305 (formerly RXC005), which was designed to reversibly bind BTK and preserve activity in the presence of the C481S acquired resistance mutation.
  • Bristol-Myers Squibb announced that the FDA granted accelerated approval to nivolumab (Opdivo) for the treatment of certain patients with metastatic colorectal cancer—specifically, patients age 12 and older with mismatch repair–deficient and microsatellite instability–high disease that has progressed following fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. The approval was based on data from the CheckMate 142 trial, which assessed responses to nivolumab in 53 patients who had previously received these chemotherapies. The overall response rate was 28%, with responses lasting at least 6 months for 67% of patients. Trials of nivolumab have not been conducted in pediatric patients; efficacy in this population was extrapolated from results in adults.
  • The FDA approved two treatments for acute myeloid leukemia (AML). First, the agency signed off on enasidenib (Idhifa; Celgene), an isocitrate dehydrogenase-2 (IDH2) inhibitor, for the treatment of adults with relapsed or refractory AML with an IDH2 mutation, as detected by the RealTime IDH2 Assay (Abbott Laboratories), a companion diagnostic that was also approved. Also green-lighted: the liposome-encapsulated combination of daunorubicin and cytarabine (Vyxeos; Jazz Pharmaceuticals) for the treatment of adults with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes, two types of AML with a poor prognosis. The single formulation may help some patients live longer than if they had received the two chemotherapies separately.
  • In addition, the agency granted Breakthrough Therapy Designation to three drugs: venetoclax (Venclexta; Roche), a first-in-class BCL2-specific oral inhibitor, for patients with previously untreated AML who are elderly and not eligible for chemotherapy; alacabrutinib, a Bruton tyrosine kinase (BTK) inhibitor in development for multiple B-cell cancers, for patients with mantle cell lymphoma; and durvalumab (Imfinzi; AstraZeneca), a PD-L1 inhibitor, as monotherapy for the treatment of locally advanced, inoperable non–small cell lung cancer that has not progressed following platinum-based chemotherapy.
  • The Norwalk, CT–based Multiple Myeloma Research Foundation (MMRF) announced that it will invest $5 million over the next 3 years to launch the MMRF Answer Fund, which seeks to find better ways to identify and treat patients at high risk for the disease. To that end, the MMRF issued a request for applications to fund innovative projects aimed at identifying the molecular drivers that contribute to early disease progression; projects will rely on the organization’s CoMMpass study, which is tracking more than 1,100 patients over 8 years. Letters of intent are due by August 14.
  • The NCI and the Children’s Oncology Group announced that enrollment has opened for the Pediatric MATCH trial, which will explore whether targeted therapies can be effective in children and adolescents with solid tumors that harbor specific mutations and have progressed during or after standard therapy. The trial will begin with six treatment arms, each testing an investigational drug aimed at a distinct molecular abnormality. A similar trial for adults, NCI-MATCH, opened in August 2015.

July 21–27, 2017

  • A nationwide survey of 1,000 adults commissioned by Research!America found that 86% of Americans say that health care providers should discuss clinical trials with patients diagnosed with a disease as part of their standard care. In addition, 75% of respondents said that taking part in clinical trials can improve health and advance science, but only 18% said that they or a family member has participated in one; more than half said that they haven’t participated due to a lack of awareness and information.
  • Stock in AstraZeneca dropped by nearly 16% after the company reported that the combination of durvalumab (Imfinzi) and tremelimumab did not improve progression-free survival (PFS) compared with standard of care—platinum-based chemotherapy—in patients newly diagnosed with non–small cell lung cancer (NSCLC) whose tumors express PD-L1 on 25% or more of their cancer cells. A PD-L1 inhibitor, durvalumab blocks the interaction of PD-L1 with PD-1 and CD80 on T cells. The company plans to assess two additional endpoints: overall survival (OS) with durvalumab alone and OS with the combination.
  • The news wasn’t all bad for AstraZeneca, however. In a phase III trial, the EGFR inhibitor osimertinib (Tagrisso) demonstrated a statistically significant and clinically meaningful improvement in PFS as a first-line treatment for patients newly diagnosed with locally advanced or metastatic EGFR+ NSCLC compared with erlotinib or gefitinib, standard first-line treatments. Data from the trial will be presented at an upcoming medical meeting, the company said.
  • In addition, AstraZeneca and Merck announced a strategic collaboration to develop and commercialize AstraZeneca’s PARP inhibitor olaparib (Lynparza) as a monotherapy and in combination with other medicines. The companies will independently develop and commercialize olaparib in combination with their respective PD-L1 and PD-1 inhibitors, durvalumab and pembrolizumab (Keytruda). Lastly, they will jointly develop and commercialize AstraZeneca’s selumetinib, a MEK inhibitor, for multiple indications, including thyroid cancer.
  • Maine Governor Paul LePage vetoed legislation that would have raised the minimum age to purchase tobacco in the state to 21. Lawmakers aim to hold an override vote next month. Meanwhile, New Jersey Governor Chris Christie signed a bill that limits the sale of tobacco products in that state to people who are 21 and older, making it the third state to enact such a law. Hawaii and California are the other two.

July 14–20, 2017

  • The FDA approved neratinib (Nerlynx; Puma) for the extended adjuvant treatment of early HER2+ breast cancer following treatment with adjuvant therapy with trastuzumab (Herceptin; Genentech/Roche). The decision was based on a multicenter, randomized, double-blind placebo-controlled trial including 2,840 women who had finished taking trastuzumab within the previous 2 years. After 2 years of follow up, invasive disease-free survival was 94.2% in patients treated with neratinib compared with 91.9% in those who received placebo.
  • The U.S. Court of Appeals for the Federal Circuit ruled that a patent on bortezomib (Velcade; Takeda) is valid until 2022, preventing the launch of lower-cost generic versions by Teva Pharmaceuticals and other competitors. A lower court had ruled in favor of the generic drug makers in 2015, saying that the compound covered by the patent was “the inherent result of an obvious process.” The appeals court overruled that decision, validating the patent on the multiple myeloma drug, which generates annual sales of more than $1 billion.
  • Astellas Pharma announced that the FDA granted Orphan Drug designation to gilteritinib for the treatment of acute myeloid leukemia (AML). Gilteritinib is a receptor tyrosine kinase inhibitor of FLT3 and AXL. The agency can assign the designation to drugs and biologics for the treatment, diagnosis, or prevention of diseases or disorders that affect fewer than 200,000 people in the United States.
  • The Leukemia & Lymphoma Society announced the expansion of its Beat AML Master trial, a groundbreaking precision medicine study launched last year to test investigational treatments for the disease. Currently, six leading cancer centers are participating in the trial, with four more expected to join by the end of the summer. Four major pharmaceutical companies are providing the investigational therapies—which are prescribed to patients based on their genetic markers—and three more companies may soon contribute agents for study.
  • The American Society of Clinical Oncology (ASCO) issued a position statement regarding rising drug prices for the treatment of cancer, noting that there should be a “real and consistent relationship between the benefits of a particular drug to patients and its cost.” In the statement, ASCO suggested that the FDA consider meaningful clinical outcomes when assessing new and supplemental drug applications, rather than small benefits that achieve statistical significance in clinical trials. In addition, the organization suggested that Medicare test an approach that would encourage health-care providers to use higher-value drugs and pharmaceutical companies to develop higher-value treatments.
  • Based on its draft recommendation, the U.S. Preventive Services Task Force will continue to recommend against screening for ovarian cancer in asymptomatic women, as it did in 2012. The committee that developed the draft recommendation found that screening does not reduce ovarian cancer deaths, and that it yields many false-positive results, which can lead to unnecessary, potentially harmful surgery. Women at high risk of the disease, such as those with BRCA1 and BRCA2 mutations, Lynch syndrome, Li-Fraumeni syndrome, and Peutz-Jeghers syndrome, and those with a family history of ovarian cancer should be screened.

July 7–13, 2017

  • The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously to recommend approval of the investigational chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (CTL019; Novartis) for the treatment of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia. If approved, tisagenlecleucel would be the first CAR T-cell therapy OK’d for use by the FDA. The agency isn’t required to follow the recommendation of its advisory panel, but it usually does so.
  • ODAC also voted unanimously to recommend that the FDA approve two biosimilar drugs: Mylan’s MYL-1401O, a biosimilar version of Roche’s Herceptin (trastuzumab), and Amgen’s ABP 215, a biosimilar of Roche’s Avastin (bevacizumab). The committee found no “clinically meaningful” differences between the biosimilars and their reference products.
  • In a funding proposal for fiscal year (FY) 2018 unveiled by the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, the NIH could receive a budget increase of $1.1 billion over FY2017. That sum includes the $496 million designated for the NIH Innovation Fund through the 21st Century Cures Act—$300 million of which would support the Beau Biden Cancer Moonshot—as well as an $80 million increase for the NCI. Those sums, if approved by Congress, would bring the NIH budget to $35.2 billion and the NCI budget to $5.771 billion.
  • According to a report in The New England Journal of Medicine, after nearly 20 years of follow up, men with prostate cancer who immediately had surgery lived no longer than those who pursued active surveillance—and they experienced more adverse effects, such as urinary and sexual dysfunction. Among the study population as a whole, neither overall survival (OS) nor prostate cancer–specific survival differed significantly between the groups. However, the subgroup of men with intermediate-risk disease who underwent surgery had a 14.5% improvement in OS, which was statistically significant; in men with low-risk disease, that difference was less than 1%.
  • The House Appropriations Committee approved legislation that would roll back FDA oversight of electronic cigarettes, also called e-cigarettes. The law would prevent the FDA from requiring safety reviews of e-cigarette products and some cigars already on the market. Supporters of FDA oversight say that the products contain toxic chemicals and are marketed to children; those in favor of loosening FDA authority argue that e-cigarettes are safer than traditional cigarettes and should be a readily available tool to help current smokers kick the habit.
  • Amgen announced that the proteasome inhibitor carfilzomib (Kyprolis) prolonged survival in patients with relapsed multiple myeloma. In the phase III ASPIRE trial, which included 792 patients, those who received carfilzomib plus lenalidomide and dexamethasone had a median OS of 48.3 months compared with 40.4 months for those who received lenalidomide and dexamethasone alone. The drug regimen was previously approved based on improvements in progression-free survival, the trial’s primary endpoint.

June 30–July 6, 2017

  • The FDA placed a clinical hold on three studies of pembrolizumab (Keytruda; Merck) that have been assessing the anti–PD-1 therapy in combination with pomalidomide or lenalidomide for the treatment of multiple myeloma. The move came after a data monitoring committee observed more deaths in patients who received pembrolizumab than in the control arms. The clinical hold does not apply to other studies involving the drug.
  • A report published in Annals of Oncology found that more than half of all cancer deaths in China can be attributed to modifiable risk factors such as smoking, alcohol use, poor nutrition, being overweight, physical inactivity, and infections. Cancer is the leading cause of death in China, with about 2.8 million fatalities every year. The researchers say that the analysis may underestimate the total because they weren’t able to consider every lifestyle factor, notably indoor air pollution due to the use of coal for cooking and heating.
  • The FDA cleared the expanded use of a cooling cap to reduce hair loss during chemotherapy. Dignitana originally received marketing authorization for its DigniCap Cooling System for use in patients with breast cancer in 2015; it can now be used in patients with other types of solid tumors. The cooling is intended to constrict blood vessels in the scalp, reducing the amount of chemotherapy that reaches hair follicle cells and slowing their division.
  • The British artificial intelligence company Exscientia announced that it will collaborate with GlaxoSmithKline to discover novel, selective small molecules for up to 10 disease-related targets across multiple therapeutic areas. By delivering preclinical drug candidates and meeting certain milestones, Exscientia would earn $43 million. The company will harness the power of supercomputers and machine learning to predict which compounds are most likely to be effective drugs, which could speed drug development while keeping costs down.
  • The cancer gap between rural and urban communities in the United States continues to grow, according to a report released by the Centers for Disease Control and Prevention (CDC). Using data from the CDC’s National Program of Cancer Registries and the NCI’s Surveillance, Epidemiology, and End Results program, researchers found that nonmetropolitan rural areas had lower average annual age-adjusted cancer incidence rates for all cancers combined, but higher death rates than in metropolitan areas. To narrow the gaps, researchers say that health care providers in rural areas should encourage patients make healthy lifestyle choices, such as quitting smoking; receive vaccines that can prevent cancer; and undergo routine cancer screening.

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June 2017

June 23–29, 2017

  • Former Vice President Joe Biden and his wife, Dr. Jill Biden, celebrated the launch of the Biden Cancer Initiative, which aims to accelerate progress in cancer prevention, detection, diagnosis, research, and treatment, and to reduce disparities in treatment outcomes. According to its website, the initiative “will work closely with patients and patient organizations, cancer researchers, cancer hospitals and community health centers, research universities, governments, and the private and philanthropic sectors to identify and address critical issues,” such as encouraging collaborative research across disciplines and improving access to healthcare.
  • The FDA announced a plan to reduce the backlog of about 200 requests for Orphan Drug Designation that are pending review within 90 days. The agency will increase staff to consider applications and employ a new, streamlined process to “increase consistency and efficiency of its reviews.” The designation, which provides tax credits and 7 years of marketing exclusivity to approved products, can be granted to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases—those that affect fewer than 200,000 people in the United States.
  • Illumina received the nod from the FDA to market its Extended RAS Panel, a next-generation sequencing kit, to help identify patients with metastatic colorectal cancer eligible to receive the EGFR inhibitor panitumumab (Vectibix; Amgen). The panel detects 56 variants in KRAS and NRAS that contraindicate anti-EGFR therapy. Panitumumab is approved for use with FOLFOX for first-line treatment of wild-type RAS metastatic colorectal cancer.
  • The FDA announced that it authorized the marketing of ClearLLab Reagents to aid in the detection of several leukemias and lymphomas, including chronic leukemia, acute leukemia, non-Hodgkin lymphoma, myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms. The authorization was supported by a study conducted at four independent clinical sites that used the test to analyze 279 samples and compared the results to alternative detection methods. ClearLLab Reagents matched the study sites’ diagnosis in 93.4% of cases and correctly detected cancer in 84.2% of cases.
  • Novartis announced that the European Commission approved expanding the use of ceritinib (Zykadia) to include first-line treatment of patients with advanced ALK+ non–small cell lung cancer. The decision was based on the phase III ASCEND-4 trial, which demonstrated a 45% reduction in the risk of disease progression with ceritinib compared with standard-of-care chemotherapy. In addition, patients treated with ceritinib had a median progression-free survival of 16.6 months compared with 8.1 months for those who received chemotherapy.
  • In a notice filed with the Securities and Exchange Commission, Coherus Biosciences reported that it eliminated 51 jobs—30% of its workforce—after the FDA said it could not approve the company’s current application to market pegfilgrastim, a Neulasta (Amgen) biosimilar. Earlier this month, Coherus said the FDA requested a reanalysis of a subset of samples with a revised immunogenicity assay, as well as information related to manufacturing processes. Pegfilgrastim is used to reduce the chance of infection in some patients with cancer receiving myelosuppressive therapy.
  • ARMO BioSciences announced promising efficacy data from a phase I/Ib clinical trial of AM0010 (pegilodecakin; pegylated IL10) in combination with FOLFOX chemotherapy in patients with advanced pancreatic cancer. Median overall survival was 10.2 months for patients who received the combination, compared with 3.8 months for those receiving AM0010 alone; 1-year survival rates were 47% and 22%, respectively. The findings were presented at the European Society of Medical Oncology 19th World Congress on Gastrointestinal Cancer in Barcelona, Spain.
  • During a Senate Appropriations Subcommittee hearing on the NIH budget, both Democrats and Republicans criticized the Trump administration’s proposal to cap facilities and administration costs at 10%. Senator Lamar Alexander (R-TN) deemed the proposal to limit these indirect costs “harebrained,” and called it “thoroughly awful,” noting that it would result in job losses. Senator Patty Murray (D-WA) worried that, in addition to hurting her state’s Fred Hutchinson Cancer Research Center in Seattle, Washington, capping these expenses would also harm the NIH intramural program.

June 16–22, 2017

  • Seattle Genetics announced the discontinuation of its phase III CASCADE trial of vadastuximab talirine (SGN-CD33A) in patients with acute myeloid leukemia after an independent data monitoring committee found a higher rate of fatal infections and deaths in the experimental arm compared with the control arm. In addition, the company suspended patient enrollment and treatment in all other trials of the drug. Subsequently, the FDA put a clinical hold on the drug, noting that no clinical trials could resume without the agency’s go-ahead.
  • It was a busy week for the FDA. The agency approved the use of daratumumab (Darzalex; Janssen) with pomalidomide (Pomalyst; Celgene) and dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received at least two prior therapies, including lenalidomide (Revlimid; Celgene) and a proteasome inhibitor. The approval was based on data from a phase I study of the three-drug combination in 103 patients in which the overall response rate was 59%.
  • The FDA also granted regular approval to Novartis’s dabrafenib (Tafinlar) and trametinib (Mekinist), given together, for patients with metastatic non–small cell lung cancer (NSCLC) with a BRAF V600E mutation. The agency also approved the Oncomine DX Target Test (Thermo Fisher Scientific), a next-generation sequencing test, to detect multiple gene mutations for NSCLC in a single test from a single tissue specimen. The test detects the presence of BRAF, ROS1, and EGFR gene mutations and can be used to select patients with the BRAF V600E mutation for treatment with the drug combination.
  • In addition, the agency approved the combination of rituximab and hyaluronidase human (Rituxan Hycela; Genentech) for the treatment of adults with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), and chronic lymphocytic leukemia (CLL). The approval provides a subcutaneous route of administration, which takes about 5 to 7 minutes, compared with an intravenous infusion that can last for several hours.
  • Speaking of rituximab, the European Commission approved Rixathon (Sandoz/Novartis), a rituximab biosimilar, for use in Europe. The drug, an anti-CD20 chimeric monoclonal antibody, is approved for use in the same indications as the reference medicine, MabThera—non–Hodgkin lymphoma (FL and DLBCL), CLL, and immunologic diseases such as rheumatoid arthritis. European patents on rituximab expired in 2013.
  • A New York district court judge awarded scientific publisher Elsevier $15 million in damages for copyright infringement by Sci-Hub, the Library of Genesis project, and similar sites, Nature reported. The judge had ruled in October 2015 that the sites were in violation of copyright law and issued a preliminary injunction against the sites’ operators, who nonetheless continued to offer free access to tens of thousands of medical and scientific papers. Experts say that Elsevier is unlikely to receive any money from Sci-Hub’s founder, Alexandra Elbakyan, who lives in Russia, and that the judgment is unlikely to prompt sites that pirate copyrighted materials to shut down.

June 9–15, 2017

  • President Donald Trump appointed Norman Sharpless, MD, to serve as the NCI’s director. He will succeed Douglas Lowy, MD, who has served as the agency’s acting director since 2015. Sharpless currently heads the University of North Carolina’s Lineberger Comprehensive Cancer Center in Chapel Hill.
  • In a 9-0 ruling, the U.S. Supreme Court agreed that biosimilar manufacturers needn’t wait for the FDA’s OK before giving 6 months’ notice to the maker of a brand-name therapeutic that a competing product will be launched. The decision means that nearly identical versions of biologic drugs could reach the market sooner, which would save patients money. The case pitted Novartis/Sandoz, which makes the biosimilar filgrastim-sndz (Zarxio), against Amgen, which makes filgrastim (Neupogen).
  • The FDA held off on approving Coherus BioSciences’ CHS-1701, a biosimilar for pegfilgrastim (Neulasta; Amgen). The company was asked to reanalyze certain data and provide more manufacturing information, and the Redwood City, CA–based biosimilar developer is working to address the FDA’s concerns. Pegfilgrastim is a PEGylated form of GCSF, a bone marrow stimulant that helps patients ward off infection during chemotherapy.
  • Austin, TX–based XBiotech terminated its phase III evaluation of Xilonix in advanced colorectal cancer, after a second interim analysis found insufficient data to meet efficacy endpoints or the threshold for study continuation. A novel agent that targets and neutralizes IL1α, Xilonix is one of XBiotech’s first-in-class “True Human” antibodies, derived without modification from individuals with natural immunity to certain diseases.
  • Epizyme reported positive interim results from its phase II trial of tazemetostat in patients with relapsed/refractory follicular lymphoma or diffuse large B-cell lymphoma, and activating EZH2 mutations. The objective response rates (ORR) to the first-in-class oral EZH2 inhibitor were 92% and 29%, respectively. The Cambridge, MA, company said that it will work with the FDA to determine next its next steps.
  • Seattle Genetics and Bristol-Myers Squibb announced positive interim phase I/II data from a study of brentuximab vedotin (Adcetris) combined with nivolumab (Opdivo) in relapsed/refractory classic Hodgkin lymphoma. Among 59 evaluable patients, the ORR was 85%, including 37 complete responses. A pivotal phase III trial comparing the combination to brentuximab vedotin alone will start later this year.
  • Dragonfly Therapeutics, a startup in Cambridge, MA, inked its first partnership—a $33 million deal with Celgene that gives the company the option of jointly developing up to four blood cancer drugs. Dragonfly is developing antibodies called TriNKETs that link natural killer (NK) cells to tumor antigens; the idea is that this will unleash the NK cells’ own tumor-fighting properties as well as alert other immune cells to the presence of cancer.

June 2–8, 2017

  • Francis Collins, MD, PhD, will continue to serve as director of the NIH. Collins had been asked to stay in the job temporarily following the inauguration of President Donald Trump. On June 6, Trump officially nominated Collins for the job, which he has held since 2009, and his appointment is all but certain to be approved by the U.S. Senate.
  • The NIH launched the Next Generation Researchers Initiative, a revised approach to support early- and mid-career scientists. Previously, the agency had announced plans to implement a Grant Support Index to limit the amount of NIH funding a particular investigator could receive, which prompted concerns in the scientific community. The new initiative will allocate $210 million annually for 5 years to fund researchers with less than 10 years’ experience as a principal investigator who are about to lose all NIH funding; who are seeking support for their next RO1 grant; or who just missed funding for their first competitive renewal.
  • Researchers announced that the phase II NCI-MATCH trial reached its goal of sequencing tumor samples from 6,000 patients, nearly 2 years earlier than anticipated. The precision medicine trial seeks to determine the effectiveness of treatment directed by genomic profiling in patients with solid tumors, lymphomas, or myelomas that have progressed following standard treatments. The NCI capped its funding for NCI-MATCH at 6,000 patients, but the trial will continue through collaborations with commercial and academic laboratories.
  • At its 2017 annual meeting in Chicago, the American Society of Clinical Oncology (ASCO) announced that eight new cancer centers in 36 locations have signed on to its TAPUR study, bringing the total number of study sites to 101 in 20 states. To increase enrollment in the trial, the age of eligibility has been lowered from 18 to 12 years. The study is evaluating 15 molecularly targeted therapeutic options for advanced cancers and collecting data on clinical outcomes to learn about additional uses of drugs beyond those indications already approved by the FDA.
  • During his opening remarks at the ASCO meeting, the organization’s president, Daniel Hayes, MD, said, “We owe it to our patients to not just assume the strategy of precision medicine is appropriate, but to prove it, and trials such as TAPUR and Lung-MAP are a step in that direction.” Noting that cancer mortality rates in the United States are geographically disparate—due to differences in treatment, not incidence or epidemiology—Hayes also emphasized the need to standardize oncology care. “Where you live should not dictate whether you live,” he said.
  • Carl June, MD, of the University of Pennsylvania in Philadelphia, observed that the number of chimeric antigen receptor (CAR) T-cell therapy trials “has increased exponentially—183 ongoing as of April, whereas back in 2010, there were only three. For the first time, too, there are more trials in China than in the United States.” June, who received the 2017 David A. Karnofsky award, ASCO’s highest scientific honor, for his pioneering research in CAR T cells, also called for the field’s myriad disciplines to collaborate to find ways to manufacture high-quality cells at low cost.
  • Roche announced that it is returning the rights to the IDO inhibitor navoximod (GDC-0919) to NewLink Genetics after disappointing data on the drug’s effectiveness in combination with the PD-L1 inhibitor atezolizumab (Tecentriq; Roche) were announced at the ASCO meeting. In a phase Ib study, the response rate was about 10%—and most of those were partial responses. Roche and NewLink had agreed to a deal in 2014 that would have been worth nearly $1 billion if the drug hit certain development milestones.
  • According to the results of two trials presented at the 2017 ASCO Annual Meeting, adding abiraterone to androgen-deprivation therapy (ADT) increases overall survival (OS) in men with locally advanced or hormone-sensitive metastatic prostate cancer—a treatment that is also well tolerated. In the STAMPEDE trial, the regimen improved OS by 37% compared with radiotherapy plus ADT, the standard of care. The LATITUDE trial compared ADT plus placebo to ADT plus abiraterone and prednisone; the OS rate at 3 years was 49% in the ADT plus placebo group, versus 66% with ADT plus abiraterone and prednisone.
  • The CDK4/6 inhibitor abemaciclib combined with fulvestrant reduced the risk of disease progression by 45% in the phase III MONARCH 2 study, which involved 669 women with HR+, HER2- advanced breast cancer resistant to endocrine therapy. The results, which were presented at the 2017 ASCO Annual Meeting, were published concurrently in the Journal of Clinical Oncology.
  • “We have made a heroic attempt thus far to understand which cancers we will die with and which cancers we will die of,” author and oncologist Siddhartha Mukherjee, MD, DPhil, told attendees at the ASCO meeting. However, that remains “a black box still to be opened,” with numerous challenges—overdiagnosis and overtreatment of cancer, what therapies should be given and when, who is going to pay for therapy, and how to set up clinical trials. Even so, Mukherjee said that he’s “nothing less than enthusiastic about this new era of targeted therapy.”

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May 2017

May 26–June 1, 2017

  • Eli Lilly announced that a randomized phase III study of ramucirumab (Cyramza) plus docetaxel in bladder cancer met its primary endpoint. Compared with docetaxel alone, adding the VEGFR2 antagonist significantly extended progression-free survival (PFS) in patients with locally advanced or metastatic disease that had progressed on platinum chemotherapy. Ramucirumab is the first antiangiogenic agent to improve PFS in a phase III trial in this disease; detailed efficacy data will be reported at a future medical meeting, and final overall survival results are expected in mid-2018.
  • The FDA approved ceritinib (Zykadia; Novartis) for untreated ALK-positive metastatic non–small cell lung cancer. The decision was based on data from a phase III trial in which the median PFS among patients randomly assigned to receive the ALK inhibitor was 16.6 months, more than double that of patients given platinum-pemetrexed doublet chemotherapy (8.1 months). The agency also approved Roche’s Ventana ALK (D5F3) CDx Assay as a companion diagnostic to identify patients eligible for treatment with ceritinib.
  • Grail and Cirina teamed up to develop and commercialize liquid biopsies for early cancer detection. The two startups, respectively based in Menlo Park, CA, and Hong Kong, share an interest in exploiting the science of circulating DNA to noninvasively detect deadly diseases at their earliest stages. This business merger is expected to help Grail expand its presence in Asian markets.
  • Oregon Health & Science University’s (OHSU) Knight Cancer Institute in Portland was awarded $9.2 million from the NCI to serve as a research center for the agency’s Cancer Systems Biology Consortium (CSBC). Launched in late 2015, the CSBC studies cancer initiation, progression, and metastasis; current members include Stanford University in Palo Alto, CA, and Memorial Sloan Kettering Cancer Center (MSKCC) in New York, NY. OHSU will analyze core cell lines, patient-derived cultures, and primary tumors to develop better treatment strategies for drug-resistant triple-negative breast cancer.
  • MSKCC researchers established a prospective clinical sequencing initiative to reveal the mutational landscape of metastatic cancer by molecularly profiling more than 10,000 patients. Their comprehensive assay, MSK-IMPACT, helped pinpoint clinically relevant somatic mutations, novel noncoding alterations, and mutational signatures shared by common and rare tumor types; patients were enrolled on genomically matched trials at a rate of 11%. Study data have been made publicly available for further analyses.

May 19–May 25, 2017

  • The White House released a detailed fiscal year (FY) 2018 budget blueprint that included a total proposed funding level of $26.9 billion for the NIH—approximately $7 billion below that of FY 2017—and a commensurate 20% (about $1.2 billion) funding decrease to the NCI. If passed, this would reduce the total number of NIH-funded research grants by 1,946 in FY 2018, including 1,648 fewer new grants.
  • Merck’s pembrolizumab (Keytruda) received accelerated approval for adult and pediatric patients with unresectable or metastatic tumors that are microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR). This is the first time the FDA has greenlighted a drug based not on tumor type, but on a common biomarker. The agency based its decision on data from five studies that enrolled 149 patients with MSI-H or dMMR cancers, primarily gastrointestinal in origin; 39.6% had a complete or partial response to pembrolizumab, and for 78% of these patients, the response lasted 6 months or longer.
  • According to a report from the American Cancer Society, about one in five cancers diagnosed in the United States is a rare cancer, defined as an incidence of fewer than six cases per 100,000 individuals per year. Using data from the North American Association of Central Cancer Registries and the Surveillance, Epidemiology, and End Results program, the researchers found that the occurrence of rare cancers is especially prevalent (71%) among children and adolescents. However, their 5-year relative survival, at 82%, is substantially higher than that of adults (46%) with a rare cancer diagnosis who are 65 years of age or older.
  • The University of Nebraska’s Fred and Pamela Buffett Cancer Center in Omaha celebrated the completion of a new $323 million building. Key features include a 10-story, 98-laboratory research tower, as well as a sanctuary for patients, their families, and staff, featuring art installations by glass artist Dale Chihuly. Former U.S. Vice President Joe Biden was the keynote speaker at the May 23 ribbon-cutting ceremony; the new cancer center will open to patients in early June.
  • Cel-Sci’s phase III trial of Multikine (Leukocyte Interleukin, Injection) in head and neck cancer was placed on full clinical hold after the FDA identified new deficiencies in the Vienna, VA–based company’s application to lift the partial clinical hold imposed in September 2016. No new patients may be enrolled in the study, nor may previously enrolled patients continue to receive this investigational therapy—a defined mixture of cytokines designed to stimulate both active and passive immunity. In a letter to shareholders, Cel-Sci noted that full clinical hold “does not result in termination of the…trial and all protocol-specified data collection will continue as described.”
  • The FDA granted Priority Review to Bristol-Myers Squibb’s nivolumab (Opdivo) and pembrolizumab for gastric cancer and liver cancer, respectively. Phase II study data supporting these applications are slated for presentation at the American Society of Clinical Oncology’s Annual Meeting in Chicago, IL, in early June. The FDA is expected to issue formal decisions on both PD-1 inhibitors during the last week of September.

May 12–May 18, 2017

  • The NCI granted Comprehensive Cancer Center status to Emory University’s Winship Cancer Institute in Atlanta, GA. This is the 48th center to earn the agency’s highest distinction, awarded to institutions that not only bridge basic and clinical research, but also successfully connect these programs to their local populations.
  • A report from the American Cancer Society on cancer prevention and early detection estimates that 20% of cancers could be prevented. The associated suffering and death could be avoided by “more systematic efforts to reduce tobacco use and obesity, improve diet, and increase physical activity and the use of established screening tests,” the authors write. For example, the report notes that approximately 190,500 people in the United States will die this year due to cigarette smoking alone.
  • Stand Up To Cancer announced a second SU2C Catalyst collaboration with Genentech to support clinical trials focused on new uses for 14 company drugs, including atezolizumab (Tecentriq) and emactuzumab, an investigational CSF1R-targeting antibody. The collaboration is expected to fund one or more proposals—each in the range of $1 million to $3 million—from the scientific community. Project ideas must be submitted by June 27 at http://proposalcentral.altum.com.
  • Merck’s pembrolizumab (Keytruda) received regular FDA approval for patients with locally advanced or metastatic urothelial carcinoma whose disease has progressed on platinum-based chemotherapy. The go-ahead was based on a randomized phase III trial showing improvements in overall survival and objective response rate (ORR) among patients given the PD-1 inhibitor, compared with those given chemotherapy. Pembrolizumab is the fourth anti–PD-1 therapy to be OKed for bladder cancer within the last year, but the first to receive full, not accelerated, approval for this patient population.
  • The FDA granted Priority Review to copanlisib (Bayer) for relapsed/refractory follicular lymphoma. In a phase II trial, the ORR among 104 patients given the PI3Kα and PI3Kδ inhibitor was 58.7%, including a complete response rate of 14.4%; the median duration of response was over 1 year. A formal decision from the agency is expected within 6 months.
  • In the United States, the number of women, particularly young women, living with distant metastatic breast cancer (MBC) is growing, likely due to improvements in treatment, according to a study published in Cancer Epidemiology, Biomarkers & Prevention. Using data from the NCI’s Surveillance, Epidemiology, and End Results Program, the researchers estimate that between 1992 and 1994, and 2005 and 2012, the 5-year relative survival of women diagnosed with MBC between ages 15 and 49 doubled from 18% to 36%; the median relative survival time increased from 22.3 months to 38.7 months.
  • The FDA designated entrectinib (Ignyta) a Breakthrough Therapy for adult and pediatric patients with TRK-positive locally advanced or metastatic solid tumors. This will expedite entrectinib’s development for a group of cancers for which there is currently no approved treatment, the company said. The tyrosine kinase inhibitor, designed to target NTRK1/2/3, ROS1, and ALK gene fusions, is currently being evaluated in a global phase II basket trial.

May 5–May 11

  • Roche announced that a phase III study of atezolizumab (Tecentriq) in advanced/metastatic urothelial carcinoma did not meet its primary endpoint of overall survival. Patients randomly assigned to receive the PD-L1 inhibitor did no better than those given chemotherapy. Atezolizumab got the FDA’s nod for bladder cancer last year, but full approval hinged on results from this pivotal study and is now “in serious doubt,” an analyst told Reuters.
  • The U.S. Senate confirmed Scott Gottlieb as commissioner of the FDA by a vote of 57 to 42. One of his biggest jobs, The New York Times noted, will be implementing the provisions of the 21st Century Cures Act, which includes a directive for the FDA to speed up drug approvals. Although Gottlieb has promised to divest himself from the drug companies to which he has close ties, critics remain concerned about his ability to lead the agency in an unbiased way.
  • The FDA greenlighted pembrolizumab (Keytruda; Merck), combined with pemetrexed and carboplatin, for the first-line treatment of metastatic nonsquamous non–small cell lung cancer (NSCLC), irrespective of PD-L1 expression. In a phase II trial, adding the PD-1 inhibitor nearly doubled patients’ objective response rate, compared with those given just pemetrexed plus carboplatin. Pembrolizumab is the only immune checkpoint blocker approved as both monotherapy (for patients with high PD-L1 expression), and now combination therapy, for untreated NSCLC.
  • The U.S. Preventive Services Task Force recommended against screening for thyroid cancer in asymptomatic adults. The federal oversight group found inadequate evidence that population-based or targeted screening could decrease mortality rates. They noted that “screening that results in the identification of indolent thyroid cancers, and treatment of those overdiagnosed cancers, may increase the risk of patient harms.”
  • According to a report in JAMA, nearly one third of 222 drugs approved by the FDA from 2001 through 2010 were affected by postmarket safety events—withdrawal, boxed warnings, or agency announcements about new risks—a median of 4.2 years later. These safety issues were significantly more frequent among biologics, psychiatric drugs, and agents granted accelerated approval. Eleven cancer therapies were among the 71 affected drugs, including cetuximab (Erbitux; Eli Lilly), lenalidomide (Revlimid; Celgene), and sunitinib (Sutent; Pfizer).
  • The UK’s Wellcome Trust Sanger Institute launched COSMIC-3D, a database integrating Sanger’s COSMIC (Catalogue of Somatic Mutations in Cancer) with protein sequence data from the European Bioinformatics Institute’s UniProt and structural information from the Worldwide Protein Data Bank. Project leaders, describing COSMIC-3D as a “human structural proteome of oncology,” hope it sheds light on how mutations affect the shape of protein binding sites and protein–protein interactions; this could then improve structure-based design of cancer therapies.

April 28–May 4, 2017

  • Congress approved a $1.1 trillion spending bill to fund the government through September, which includes a $2 billion boost for the NIH, bringing the agency’s total funding for fiscal year (FY) 2017 to $34.1 billion. The NCI will be allocated $5.7 billion from this amount, an increase of $475.8 million over FY 2016. President Donald Trump is expected to sign the measure to avert a government shutdown.
  • AstraZeneca’s durvalumab (Imfinzi) received the FDA’s nod to treat locally advanced or metastatic urothelial carcinoma that has progressed on platinum chemotherapy. The agency based its decision on a single-arm phase II trial of the PD-L1 inhibitor, in which the objective response rate to durvalumab was 26.3% among 95 patients with a high PD-L1 score, as measured by Roche’s Ventana PD-L1 (SP263) Assay—simultaneously greenlighted by the FDA as a complementary diagnostic—and 4.1% among 73 patients with a low or negative score.
  • Aeterna Zentaris announced disappointing results from its phase III trial of zoptarelin doxorubicin for women with locally advanced, recurrent, or metastatic endometrial cancer. The therapy, which comprises doxorubicin linked to a small peptide agonist to the LHRH receptor, performed no better than doxorubicin alone in terms of overall or progression-free survival. The Charleston, SC–headquartered company stated that it will not evaluate zoptarelin doxorubicin for any other indications.
  • The International Association for the Study of Lung Cancer (IASLC) released an Atlas of PD-L1 Immunohistochemistry Testing in Lung Cancer. The 132-page publication is the first to collectively discuss the specifics of each of the five PD-L1 IHC assays currently available—three from Agilent Technologies (28-8 pharmDx; 22C3 pharmDx; 73-10) and two from Roche (SP263; SP142). The IASLC aims for their atlas to be a resource that helps clinicians and patients better understand current biomarker-based treatment options.
  • The World Health Organization (WHO) announced a pilot project to prequalify biosimilar cancer drugs, a step toward expanding the availability of these therapies in low-income countries. In September, manufacturers will be invited to submit prequalification applications for biosimilar versions of rituximab (Rituxan; Genentech) and trastuzumab (Herceptin; Roche). Both are on the WHO’s Essential Medicines list; biosimilars that make the cut—comparable to the original in terms of quality, safety, and efficacy—will be eligible for procurement by United Nations agencies.
  • Northwestern University opened the Ronald and JoAnne Willens Center for Nano Oncology. The Evanston, IL, center, launched with a $10 million gift from the Willens family, will support interdisciplinary research aimed at tackling particularly aggressive cancers with nanotechnology—for instance, exploring the utility of nanostructures like spherical nucleic acids in delivering drugs to the brain.

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April 2017

April 21–27, 2017

  • Bayer announced that the FDA OK’d the expanded use of regorafenib (Stivarga) to treat patients with hepatocellular carcinoma who have previously received another one of its drugs, sorafenib (Nexavar). A kinase inhibitor that blocks enzymes in the VEGF pathway, regorafenib is the first treatment for liver cancer to be greenlighted by the agency in nearly a decade. The drug is also approved to treat colorectal cancer and gastrointestinal stromal tumors that no longer respond to other therapies.
  • On April 22, tens of thousands of people in more than 600 cities around the globe marched to champion robust funding for scientific research. Organized by the nonpartisan March for Science, the group calls for supporting “science that upholds the common good and for political leaders and policy makers to enact evidence-based policies in the public interest.” Following the march, the group called for a “Week of Action,” promoting different activities each day, such as visiting a science museum and registering to vote.
  • A phase III trial of Eli Lilly’s abemaciclib plus an aromatase inhibitor demonstrated statistically significant improvement in progression-free survival in women with HR-positive, HER2-negative advanced breast cancer—as well as improvement in overall response rate—compared with an aromatase inhibitor alone. The company made the announcement following a preplanned interim analysis of the data, which will be presented at a medical conference later this year. A CDK4/6 inhibitor, abemaciclib is also under investigation for the treatment of lung and pancreatic cancers.
  • The FDA granted Breakthrough Therapy Designation to Pfizer’s lorlatinib, a next-general ALK/ROS1 tyrosine kinase inhibitor, for the treatment of patients with ALK-positive metastatic non–small cell lung cancer previously treated with another ALK inhibitor.
  • Redwood City, CA–based OncoMed Pharmaceuticals will cut about half of its staff, leaving just 64 employees, after its two most advanced drug candidates failed in separate phase II clinical trials earlier this month. First, its stem cell cancer drug demcizumab, a monoclonal antibody targeting Delta-like Ligand 4 in the Notch signaling pathway, failed to work better than current treatments for patients with newly diagnosed metastatic pancreatic cancer. Similarly, tarextumab, which targets Notch2/3, proved no better than a placebo in patients with small cell lung cancer.
  • After the U.S. Preventive Services Task Force recommended against routine PSA testing in 2012, screening rates dropped from 37.8% in 2010 to 30.8% in 2013 among men age 50 and older. However, based on data from the 2015 National Health Interview Survey, that decline has leveled off, as nearly one in three men age 50 and over is receiving routine PSA testing, according to a report from the American Cancer Society. The report’s authors suggest multiple factors could be at play, such as the fact that other public health organizations still support PSA testing, albeit with shared decision-making between physicians and patients, and that physicians interested in dropping PSA testing have already done so.
  • “The Immortal Life of Henrietta Lacks” premiered on HBO. Based on the book of the same name by Rebecca Skloot, the movie explores the use of Lacks’ cervical cancer cells for research without her knowledge. Countless studies employing so-called HeLa cells, which have been bought and sold by the billions since they were harvested in 1951, have led to the development of drugs to treat leukemia and human papillomavirus, among other conditions.

April 14–20, 2017

  • Genentech’s PD-L1 inhibitor atezolizumab (Tecentriq) received the FDA’s nod as first-line therapy for patients with locally advanced or metastatic urothelial carcinoma who aren’t eligible for platinum chemotherapy. This is the first immune checkpoint blocker to gain approval as initial treatment for bladder cancer. The FDA based its decision on data from a phase II study in which the objective response rate to atezolizumab among 119 patients was 23.5%, including a complete response rate of 6.7%.
  • AbbVie announced disappointing results from two phase III studies evaluating veliparib, an investigational PARP inhibitor, in combination with carboplatin and paclitaxel, in non–small cell lung cancer (NSCLC) and triple-negative breast cancer (TNBC). Neither trial met its primary endpoint—overall survival for NSCLC; pathologic complete response (pCR) for TNBC.
  • The FDA designated CTL019 as a Breakthrough Therapy for diffuse large B-cell lymphoma (DLBCL). Results of a phase II study supporting the designation will be presented at an upcoming meeting, according to Novartis. Business news outlet TheStreet reported that investors are keen to compare the data on this investigational chimeric antigen receptor (CAR) T-cell therapy to that of KTE-C19 (Kite Pharma), another CD19-targeting CAR T-cell therapy under review as a treatment for DLBCL.
  • TheStreet also noted that the FDA’s decision to spend only 5 hours reviewing neratinib for breast cancer may be a bad omen for Puma Biotechnology. In recent years, the agency “has often used half-day sessions of the Oncologic Drugs Advisory Committee to administer public floggings to flawed cancer drugs”—for instance, Spectrum’s apaziquone for bladder cancer last September. The phase III study data for neratinib, a pan-HER inhibitor, indicate only a modest improvement in disease-free survival at the cost of high rates of severe diarrhea.
  • A study in JAMA Surgery suggests that patients with early-stage breast cancer who have a pCR to neoadjuvant chemotherapy may be able to avoid surgery. In an analysis of 527 patients, those who achieved a pCR were also more than seven times less likely to have residual nodal disease. The investigators plan to test whether radiation would be just as effective as surgery for this subset in an upcoming phase II trial.
  • As part of the 21st Century Cures Act, the University of Kentucky (UK) in Lexington was awarded $11.2 million from the NIH to study cancer and obesity. The 5-year grant will help fund UK’s new Center for Cancer and Metabolism. University leaders hailed the funding announcement, noting that Kentucky has one of the nation’s highest cancer mortality rates and is also in the top 10 for obesity, so research into these major health issues is sorely needed.

April 7–13, 2017

  • The U.S. Preventive Services Task Force issued a draft recommendation in support of an individualized approach to prostate cancer screening, with a decision based upon a discussion with a health-care provider about the risks and potential benefits of screening, for men ages 55 to 69. In 2012, the group recommended against routine screening for all men, even though the American Urological Association and other organizations supported it for men who discussed the pros and cons with a clinician. The public can comment on the draft recommendation until May 8.
  • According to a report by the International Agency for Research on Cancer (IARC), published in Lancet Oncology, the number of childhood cancers worldwide rose by 13% between the 1980s and the period spanning 2001 to 2010. That conclusion was based upon numbers recorded in 153 cancer registries in 62 countries, mainly in North American and Europe. IARC attributes part of the increase to improved detection efforts.
  • GenomeWeb reported that the University of California (UC) filed an appeal related to a patent dispute with the Broad Institute over CRISPR/Cas9 technology with the U.S. Court of Appeals for the Federal Circuit. In February, the Patent Trials and Appeals Board sided with the Broad Institute, saying that the institute’s patents specified use in eukaryotic cells and thus didn’t overlap with patent applications from the university. However, the UC claims that its researchers were the first to engineer CRISPR/Cas9 for use in all environments.
  • The FDA granted Orphan Drug Designation to two experimental therapies: ganitumab (NantCell) and tasquinimod (Active Biotech). A monoclonal antibody directed against IGF1R, ganitumab is being tested in a phase III trial for the treatment of Ewing sarcoma. Tasquinimod is an immunomodulatory, antimetastatic, and antiangiogenic compound under investigation for the treatment of multiple myeloma.
  • Research from a team of scientists at École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland, and a second group from EPFL and Katholieke Universiteit Leuven in Belgium, published back to back in Science Translational Medicine, shows that the combined use of angiogenesis inhibitors and PD-1 checkpoint blockers shrank tumors and improved survival in mice with different types of cancer, including breast and pancreatic malignancies. Anti–PD-1 drugs work in only about 20% to 40% of patients with certain types of cancer, such as melanoma, so researchers have been testing therapeutic combinations in an effort enhance cytotoxic T-cell activity.
  • Although it was shown to improve survival in patients with certain head and neck cancers in a phase III trial, the PD-1 inhibitor nivolumab (Opdivo) will not be recommended to treat these patients in the UK. The decision was based on a cost/benefit analysis by the National Institute for Health and Care Excellence (NICE), prompting calls from clinicians and the public for NICE to work with the drug’s maker, Bristol-Myers Squibb, to agree on an acceptable price.

April 1–6, 2017
This Week: Special Content from the American Association for Cancer Research’s (AACR) Annual Meeting 2017 and other news

 

  • Reflecting on much of the research presented at the American Association for Cancer Research (AACR) Annual Meeting in Washington, DC, Louis Weiner, MD, director of the Georgetown Lombardi Cancer Center in Washington, DC, and moderator of an April 4 press conference on immunotherapeutics, said that long-held dogma has changed. “It wasn’t so long ago, maybe even only a decade ago, that people thought that immunotherapy was kind of like a stupid pet trick when it came to the treatment of cancer,” he noted. “That certainly has been disproven.”
  • During a plenary address at the AACR meeting, Jennifer Grandis, MD, of the Helen Diller Comprehensive Cancer Center at the University of California, San Francisco, outlined the benefits of “window-of-opportunity” trials, in which patients are treated with a novel drug that has exhibited strong preclinical activity prior to tumor resection. Researchers can then use tumor samples to gain insight into the pharmacodynamics of the drug, resistance mechanisms, and biomarkers of response. These trials, she said, “expand the scope of clinical research programs” and can speed improvements in cancer treatment.
  • During a science policy session, researchers from the United States and the UK presented differing views of electronic cigarettes, or e-cigarettes, as a smoking cessation tool. Ben Toll, PhD, of the Medical University of South Carolina in Charleston, noted that American guidelines call for oncologists to recommend only FDA-approved cessation tools, such as nicotine patches and gum, because they have proven effective. However, Lion Shahab, PhD, of University College London said that British doctors aren’t quite as leery of the devices because there’s little evidence that they’ll cause harm while potentially conveying a small benefit to users.
  • Several organizations have begun to collaborate on the Human Cancer Model Initiative, which was described by Mathew Garnett, PhD, of the UK’s Wellcome Trust Sanger Institute, during a Stand Up To Cancer scientific session. The participating institutions—Cancer Research UK, Hubrecht Organoid Technology, the NCI, the Wellcome Trust Sanger Institute, Cold Spring Harbor Laboratory in New York, and the Broad Institute in Cambridge, MA—aim to generate 1,000 highly annotated tumor-derived models and make them available to researchers through the American Type Culture Collection.
  • On Monday, April 3, former Vice President Joe Biden gave an inspirational and at times moving speech about the Beau Biden Cancer Moonshot, named for his son, rattling off several of the collaborative projects that the Moonshot has launched, including the engagement of dozens of federal agencies with a stake in cancer research, dedication to greater sharing of deidentified patient records, efforts to boost enrollment in clinical trials, and a push to standardize electronic medical records. However, the work has only just begun. “I’d like to tell you we solved all of the problems we identified and that you know so well,” Biden said. “In the time we’ve had, we got a running start, but we’re nowhere near finishing the race.”
  • Biden also sought to reassure attendees at the AACR meeting that congressional support for the NIH remains high, even though President Donald Trump proposed what Biden called “draconian cuts” of nearly 20% to the agency’s budget for the 2018 fiscal year. “Here’s the good news—I don’t think that there’s a chance that the American people or the United States Congress, virtually the same Congress that passed the 21st Century Cures Act just several months ago, will support or pass this budget.”
  • During a panel discussion that followed Biden’s speech, Representative Kevin Yoder of Kansas, a Republican, echoed Biden’s sentiments. “Regardless of who is in the White House, this continues to be a priority for us,” Yoder said. “This isn’t just a line item in the budget … but something that really is fundamental to who we are as American people.” He continued by saying that cancer doesn’t affect just one political party or one region of the country: “It knows no particular region. It affects every street, every cul-de-sac in America and therefore it affects every member of Congress and their constituents.”
  • The future of immuno-oncology may involve “using anticancer probiotics to normalize an individual’s cancer–immune set point [inherent immunologic status],” said Laurence Zitvogel, MD, of Institut Gustave Roussy in France, during the April 3 plenary session. She noted that in a study of 175 patients given anti–PD-1 therapy, outcomes were poorer among those who also received antibiotics, which induced dysbiosis, or an imbalance in gut flora, determined through quantitative metagenomic analyses of patients’ stool composition. However, in mice bearing human non–small cell lung cancer, fecal microbiota transplantation restored the efficacy of PD-1 blockade; in particular, two microbial species, Akkermansia muciniphila and Enterococcus hirae, had important immunomodulatory effects.
  • Reviewing the field of chimeric antigen receptor (CAR) T-cell therapy during the same plenary session, Crystal Mackall, MD, of Stanford University in California, described antigen loss—a ready route for tumor cells to evade engineered T cells that target single tumor antigens—as a “canary in the coal mine” in forewarning resistance/relapse. Better CARs are on the horizon, she said, including a bispecific iteration that simultaneously goes after CD19 and CD22, both highly expressed in B-cell malignancies. “You will witness, in the near future, the delivery of increasingly complex, intelligent cellular therapeutics”—for instance, remote-controlled, exhaustion-resistant CAR T cells to diminish toxicity and improve efficacy—“to help ensure that genetically quiet cancers can still benefit from the immunotherapy revolution,” she concluded.
  • Attendees of the April 2 Opening Ceremony at the AACR’s meeting stood together to call upon Congress to maintain—if not increase—NIH funding. After hearing an impassioned plea from AACR President Nancy Davidson, MD, that scientific funding must remain a national priority, the audience of thousands raised and waved signs bearing slogans such as “Cancer Research Saves Lives,” “Support Medical Research,” and “Invest in Life-Saving Cancer Research.”
  • For patients lacking tumor tissue, a liquid biopsy can detect the emergence of new mutations, such as the EGFR T790M mutation in patients with non–small cell lung cancer. However, due to a variety of concerns, such as reproducibility and variation among assays, testing tissue samples remains the gold standard, said Phil Stephens, PhD, of Foundation Medicine in Cambridge, MA, during a panel discussion on liquid biopsies. Yet even if tissue is available, argued Howard Scher, MD, of Memorial Sloan Kettering Cancer Center in New York, NY, liquid biopsies may make more sense in some cases, such as in metastatic prostate cancer, where extracting tissue from bone can be painful and difficult for patients.
  • Following up on interim results reported last spring, Italian researchers presented final data on patients with HER2-positive metastatic colorectal cancer enrolled in Cohort A of the phase II HERACLES trial, which tested the combination of trastuzumab (Herceptin; Genentech) and lapatinib (Tykerb; GlaxoSmithKline). The objective response rate (ORR) was 30% (10 of 33 patients), including two complete responses; thirteen patients had stable disease. The ORR was even higher—50%—in patients with tumors with highly amplified HER2.
  • In a position statement, the American Society of Clinical Oncology raised serious concerns about recently proposed federal “right-to-try” (RTT) legislation, as well as RTT state laws that have been enacted. Although the organization supports increasing access to investigational treatments for patients with cancer, it asserted that most RTT laws are “not an effective mechanism for improving access to investigational drugs for terminally ill patients and may cause unintended harms.” The statement notes that RTT laws do not require or compel drug manufacturers to provide investigational products or offer new rights or protections for patients.
  • Roche announced that alectinib (Alecensa) significantly improved progression-free survival in people with ALK-positive advanced non–small cell lung cancer whose disease had progressed following treatment with a platinum-based chemotherapy and crizotinib (Xalkori; Pfizer). The company said that peer-reviewed data from this phase III study will be published later this year.
  • The Roche CINtec Histology test received marketing clearance from the FDA. The test is the only clinically validated p16 biomarker test that, when used in conjunction with hematoxylin and eosin staining, helps pathologists determine which women should receive treatment for precancerous cervical lesions.

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March 2017

March 31, 2017

  • President Donald Trump called for a cut of more than $1.2 billion in NIH funding for the current fiscal year, which ends on September 30—a reduction that would be in addition to the $5.8 billion cut to the agency’s budget for fiscal year 2018 that he proposed earlier this month. Trump also suggested paring back FDA funding for this year by $40 million and chopping $314 million out of the budget for the Centers for Disease Control and Prevention, measures that could increase allocations for defense spending and the border wall. However, Congress has the final say on the nation’s spending.
  • Niraparib (Zejula; Tesaro) received FDA approval as maintenance therapy for women with recurrent ovarian, fallopian tube, or primary peritoneal cancers. The decision was based on phase III data indicating that niraparib reduced the risk of disease progression or death regardless of BRCA mutation status. This is the third PARP inhibitor to get the agency’s nod, but the first for which a companion diagnostic isn’t necessary. Although the FDA greenlighted Myriad Genetics’ BRACAnalysis CDx, using it to identify patients most likely to benefit from the drug is not required.
  • The FDA granted Priority Review to Novartis’s CTL019 for pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL). CTL019, an investigational chimeric antigen receptor T-cell therapy that targets CD19, is the first in this immunotherapeutic class to be considered for approval. The decision was based on a phase II study in which 41 of 50 patients achieved complete remission within 3 months of treatment.
  • Another therapy targeting CD19, Amgen’s blinatumomab (Blincyto), was granted the FDA’s Priority Review for label expansion: treating all forms of relapsed/refractory B-cell precursor ALL, not just Philadelphia chromosome–negative disease, for which the drug is already approved. Blinatumomab is a bispecific monoclonal antibody that binds CD3 on T cells and CD19 on tumor cells, thereby linking both and unleashing tumor-directed T-cell cytotoxicity.
  • Danish biotech Genmab and Janssen Pharmaceuticals pulled the plug on their joint evaluation of Janssen’s CD38-targeting monoclonal antibody daratumumab (Darzalex) in non-Hodgkin lymphoma. In two cohorts of the phase II study—patients with relapsed/refractory follicular lymphoma and diffuse large B-cell lymphoma—the targeted objective response rates (ORR) of 50% and 30%, respectively, were not reached. The ORR in the third cohort, comprised of patients with mantle cell lymphoma, was not evaluable due to slow recruitment.
  • The eighth edition of the American Joint Committee on Cancer’s Cancer Staging Manual will introduce key changes, effective January 2018.Medscape reports that “prognostic” staging, incorporating genomic profiles and molecular markers, will be the primary stage recorded in cancer registries in the United States. The current approach involves “anatomic” staging, which considers three elements: tumor size (T), lymph nodes (N), and distant metastases (M). Under the new system, T2N0 or T1N1 HER2+ breast cancer, for instance, will be considered stage 1 disease; currently, any lymph node involvement (N1) would disqualify this classification.

March 24, 2017

  • The FDA approved the PD-L1 inhibitor avelumab (Bavencio; EMD Serono) for the treatment of metastatic Merkel cell carcinoma in patients age 12 or older; it’s the first FDA-approved therapy for the disease. The approval was based on data from a single-arm trial that included 88 patients who had been previously treated with at least one chemotherapy regimen. Of those patients, 33% experienced complete or partial shrinkage of their tumors, and the response lasted for more than 6 months in 86% of them.
  • Despite dramatic progress in recent years—including new approaches to cancer diagnosis and treatment—several hurdles threaten to slow advances in cancer care in the United States, according to a report from the American Society of Clinical Oncology published in the Journal of Oncology Practice and presented during a briefing on Capitol Hill. Access and affordability challenges, increased administrative challenges for medical practices, and questions about consistent funding for the NCI and FDA pose barriers to high-value, high-quality cancer care, the report states.
  • Regeneron Pharmaceuticals and GlaxoSmithKline will collaborate with UK Biobank to sequence samples collected from 500,000 volunteer participants over the past 10 years. The companies aim to sequence genetic material in 50,000 samples by the end of the year; sequencing of all of the samples in the UK Biobank is expected to take 3 to 5 years. During the initial phase of the project, the companies will have exclusive access to the data for 9 months, after which it will be made openly available to the broader scientific community.
  • Introduced in the U.S. House of Representatives and Senate, bipartisan legislation aims to support the growing demand for palliative care. Called the Palliative Care Hospice Education and Training Act, the legislation would expand federal research on palliative care, support training for nurses and other health care professionals, and establish a national public education effort related to palliative care.
  • Waltham, MA–based Cerulean Pharma will merge with Daré Bioscience of San Diego, CA, which focuses on products for women’s reproductive health. Separately, failing Cerulean sold its two lead cancer drug candidates, CRLX101 and CRLX301, to BlueLink Pharmaceuticals for $1.5 million. CRLX101 failed a phase II trial in kidney cancer last summer and a mid-stage study in lung cancer in 2013.
  • In updated guidance, the UK’s National Institute for Health and Care Excellence said that women should be offered anastrozole if they have a family history of breast cancer to reduce their risk of developing the disease. Based on recent data, researchers concluded that if 1,000 postmenopausal women at high or moderate risk of breast cancer took anastrozole for 5 years, 35 cases of breast cancer would be prevented, compared with 21 if they took tamoxifen. Premenopausal women at high risk should continue to be offered tamoxifen, the guideline says.
  • According to STAT, members of the Chilean congress and patient advocacy groups asked the country’s health minister to issue compulsory licenses for enzalutamide (Xtandi; Astellas/Medivation), a prostate cancer drug, as well as hepatitis C therapies marketed by Gilead Sciences. If implemented, the move could allow more patients to access the drugs.

March 17, 2017

  • President Donald Trump released his proposed budget for fiscal year (FY) 2018, calling for an increase of $54 billion in defense spending. To pay for that without adding to the federal deficit, he suggests cutting allocations to a number of agencies and programs, including the NIH, which would see its budget slashed by $5.8 billion, about 20% of its current funding. Although no specifics were provided, the proposal also calls for “a major reorganization of NIH’s Institutes and Centers to help focus resources on the highest priority research and training activities.”
  • President Trump nominated Scott Gottlieb, MD, to lead the FDA. Currently, Gottlieb is a health policy analyst and fellow at the conservative American Enterprise Institute, where he has studied the FDA and the Centers for Medicare and Medicaid Services. Previously, he served as deputy commissioner for medical and scientific affairs at the FDA.
  • The FDA gave its stamp of approval to ribociclib (Kisqali; Novartis) as a first-line treatment for HR-positive, HER2-negative metastatic breast cancer in combination with an aromatase inhibitor such as letrozole. A CDK4/6 inhibitor, ribociclib was approved based on interim data from a phase III trial showing a 44% reduction in the risk of disease progression or death after 18 months of treatment. A subsequent analysis determined that the progression-free survival for ribociclib plus letrozole was 25.3 months compared with 16 months for letrozole alone.
  • The FDA also approved the use of pembrolizumab (Keytruda; Merck) to treat children and adults with refractory classical Hodgkin lymphoma, as well as patients whose disease has returned after three or more prior therapies. The decision was based upon a multicenter, nonrandomized, open-label trial involving 210 patients, which yielded an overall response rate of 69%. The anti–PD-1 immunotherapy is already indicated for certain melanomas, non–small cell lung cancers, and head and neck squamous cell carcinomas.
  • Under new rules issued by the Accreditation Council for Graduate Medical Education (ACGME), work hours for all medical residents will be capped at 80 hours a week, with shifts limited to 24 hours plus an additional 4 hours “to manage necessary care transitions.” The change, which goes into effect on July 1, will improve patient safety by reducing the number of times that one physician hands off patient care to another, according to the ACGME. The maximum shift duration for first-year residents had been set at 16 hours in 2011.
  • The U.S. Preventive Services Task Force posted its Draft Research Plan on Risk Assessment, Genetic Counseling, and Genetic Testing for BRCA-Related Cancer. The plan is available for review and public comment until April 12. The plan guides a systematic review of evidence, which will be drawn upon to evaluate the benefits and harms of these clinical services and develop specific recommendations for their use.

March 10, 2017

  • Personalized medicine can improve patients’ health and reduce medical expenses, yet multiple challenges are slowing broad implementation of the practice, which involves matching patients to therapies based on the molecular characteristics of their tumors, according to a report from The Personalized Medicine Coalition. Titled “The Personalized Medicine Report: Opportunity, Challenges, and the Future,” the report says that the lack of standardization in the review of laboratory-developed tests, questions about regulatory oversight of next-generation sequencing, reimbursement concerns, and the need to increase awareness among patients and healthcare providers about the value of personalized medicine have stymied its adoption.
  • According to an audit, a project involving IBM’s Watson and The University of Texas MD Anderson Cancer Center in Houston has made little progress in using the technology to transform clinical care after almost 5 years of work and an investment of $62 million by the institution. The audit, according to The Wall Street Journal, found that “technology challenges at MD Anderson … made it hard to integrate artificial-intelligence software into complicated health-care settings,” and that the pilot program didn’t work with the hospital’s electronic health records. The audit didn’t assess Watson’s ability to learn from scientific data and publications and then recommend appropriate treatment options for patients.
  • The FDA lifted a clinical hold on studies of the leukemia drug vadastuximab talirine (Seattle Genetics) after an analysis of data collected on about 300 patients and some changes to study protocols. The hold was implemented in December after four clinical-trial participants died.
  • Using data from the Surveillance, Epidemiology, and End Results (SEER) database, researchers have found that deaths from childhood cancer may be nearly four times more common than previously thought. Writing in the Journal of Clinical Oncology, they point to a clinical trial involving children with acute myeloid leukemia in which 1.6% of patients died. In contrast, records in the SEER database show that about 6.2% of young patients die of the disease, noting that these children—especially those who haven’t reached their first birthday—often don’t live long enough to receive treatment or enroll in a clinical trial.
  • In a multicenter, randomized phase III trial, patients with high-risk chronic lymphocytic leukemia were significantly more likely to respond to the combination of ublituximab (TG-1101) plus ibrutinib (Imbruvica; Pharmacyclics, Janssen) than ibrutinib alone, TG Therapeutics announced. (“High risk” was defined as having a 17p deletion, an 11q deletion and/or a p53 mutation.) Among 117 treated patients, 80% of those who received the combination responded, compared with 47% of those receiving ibrutinib alone. Ublituximab is a glycoengineered anti-CD20 monoclonal antibody.

March 3, 2017

  • Kite Pharma announced positive data from a pivotal phase II trial evaluating its lead chimeric antigen receptor T-cell therapy, axicabtagene ciloleucel, in non–Hodgkin lymphoma (NHL). Among 101 patients with multiple types of aggressive NHL, each given a single infusion of the CD19-targeting cells, the objective response rate was 82%, of which 54% were complete responses; the median overall survival (OS) has not been reached. Based on these results, the Santa Monica, CA–based company intends to seek FDA approval of axicabtagene ciloleucel later this year.
  • The NCI launched its largest study, to date, of African American cancer survivors. Through interviews, information from medical records, and biospecimen collection, the Detroit Research on Cancer Survivors study—funded by a 5-year, $9 million grant—will acquire comprehensive data on 5,560 cancer survivors in three counties surrounding Detroit, MI.
  • San Diego, CA–based OncoSec Medical Inc. received the FDA’s Fast Track designation for developing ImmunoPulse IL-12 to treat patients with metastatic melanoma who have progressed on pembrolizumab (Keytruda; Merck) or nivolumab (Opdivo; Bristol-Myers Squibb). ImmunoPulse is designed to stimulate expression of IL12 in the tumor microenvironment, thereby potentially converting tumors from being immunologically “cold” to “hot.”
  • The FDA granted Priority Review to two investigational agents, enasidenib (Celgene) and avelumab (Pfizer, EMD Serono), for relapsed/refractory acute myeloid leukemia (AML) and locally advanced or metastatic urothelial carcinoma, respectively. Enasidenib is a first-in-class targeted inhibitor of mutant IDH2, which occurs in 8% to 19% of AML cases; avelumab targets the PD-L1 ligand in the PD-1 immune checkpoint pathway. The agency’s approval decisions are expected by the end of August.
  • According to a Journal of the National Cancer Institute study, rates of colon and rectal cancers are rising among young adults in the United States. “Compared with adults born circa 1950, those born circa 1990 have double the risk of colon cancer and quadruple the risk of rectal cancer,” the researchers report. Obesity, sedentary behavior, and diet are among the probable culprits; the findings suggest that some screening tests may need to begin before the age of 50, which is the age currently recommended for the average individual.
  • Amgen reported positive results from a phase III trial comparing its proteasome inhibitor carfilzomib (Kyprolis) with Takeda’s bortezomib (Velcade) in relapsed/refractory multiple myeloma. Among patients randomly assigned to receive either drug plus dexamethasone, the median OS was 47.6 months in the carfilzomib arm, versus 40 months for those given bortezomib. Bortezomib has long been the standard of care, but these results support carfilzomib as the treatment of choice in this patient population, the company says.

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February 2017

February 11–24, 2017

  • The U.S. Senate confirmed Georgia Representative Tom Price, MD, as secretary of the Department of Health and Human Services (HHS). The NIH and the FDA fall under the aegis of the HHS.
  • Genome-editing tools, such as CRISPR/Cas9, have created new research opportunities and potential treatments for both heritable and nonheritable health conditions, but many people consider the genome “ethically inviolable.” However, according to a report released by the National Academy of Sciences and the National Academy of Medicine, heritable germline editing clinical trials could one day be permitted if certain stringent criteria are met, such as the absence of reasonable alternatives, the development of comprehensive plans for long-term multigenerational follow-up, and credible data on the risks and potential health benefits.
  • The government of Botswana announced a $100 million initiative to create a pediatric hematology/oncology treatment network and build clinical infrastructure in southern and east Africa, thanks to $50 million from the Bristol-Myers Squibb Foundation and an additional $50 million to be raised by Houston’s Baylor College of Medicine International Pediatric AIDS Initiative at Texas Children’s Hospital. In the United States, about 80% of children with cancer survive, but in sub-Saharan Africa, the survival rate is estimated to be about 10% due to inadequate health care infrastructure and a lack of physicians and health care providers. The initiative, dubbed Global HOPE, aims to address these challenges and improve the ability to diagnose pediatric blood disorders and cancer in Botswana, as well as in Malawi and Uganda.
  • The Cleveland Clinic announced that the new Taussig Cancer Center will open on March 6. Costing an estimated $276 million, the seven-story, 377,000-square-foot facility will house all outpatient cancer treatment services, organized by cancer type. For example, all clinical and treatment areas for breast cancer will be located on the same floor to offer greater convenience for patients.
  • The FDA approved lenalidomide (Revlimid; Celgene) as maintenance therapy for patients with multiple myeloma following autologous stem cell transplant. The decision was based upon two randomized, controlled trials that included more than 1,000 patients in total and that demonstrated a 15-month and 18-month progression-free survival advantage for those who received lenalidomide compared with those who received a placebo.
  • Novartis announced that the FDA granted Priority Review for the expanded use of ceritinib (Zykadia) as a first-line treatment for patients with metastatic non–small cell lung cancer whose tumors are ALK-positive as detected by an FDA-approved test.
  • Cell Medica’s investigational immunotherapeutic CMD-003 received Fast Track designation from the FDA for the treatment of Epstein-Barr Virus (EBV)–associated malignancies. EBV is tied to a variety of cancers, including about 15% to 20% of lymphomas. The therapy consists of autologous EBV-specific T cells that target four EBV-associated antigens; the cells are activated, expanded, and infused back into the patient.

February 10, 2017

  • The NCI launched the NCTN/NCORP Data Archive, a centralized repository of deidentified patient-level data from phase III studies carried out by trial groups affiliated with the National Clinical Trials Network (NCTN) and NCI’s Community Oncology Research Program (NCORP). “Greater sharing of research data underlies much of the thinking behind the Beau Biden Cancer Moonshot and the initiatives emerging from it,” the agency noted, and the new database “is building on and contributing to this momentum.”
  • The FDA gave French biopharmaceutical company Cellectis the go-ahead to begin phase I evaluations of its universal CAR T-cell therapy, UCART123, in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm. These CD123-targeting cells have been gene-edited, using TALEN, so they can be given to multiple patients without the need for donor–recipient matching. This is the first “off-the-shelf” CAR T-cell product to gain FDA approval for clinical trials, which will be led by Weill Cornell Medicine in New York, NY, and The University of Texas MD Anderson Cancer Center in Houston.
  • The nonprofit Chan Zuckerberg (CZ) Biohub selected its first cohort of 47 investigators from three institutions: University of California, Berkeley; University of California, San Francisco; and Stanford University in Palo Alto. Each will receive a 5-year appointment and up to $1.5 million for life science research in their areas of expertise, ranging from biology to physics. These funds will be unrestricted, to spur the pursuit of creative, high-risk projects aimed at helping the CZ Biohub achieve its founders’ goal of “curing, preventing, or managing every disease in our children’s lifetime.”
  • A phase II trial of the investigational immune checkpoint inhibitor lirilumab (Innate Pharma) failed to demonstrate clinical efficacy in AML. Among 150 elderly patients randomly assigned to receive lirilumab or placebo, leukemia-free survival and other efficacy endpoints were not statistically different between the study arms. France-based Innate is partnering with Bristol-Myers Squibb (BMS) to develop lirilumab, which targets the KIR2DL receptor family; the drug will continue to be evaluated in combination with BMS’s PD-1 inhibitor nivolumab (Opdivo) against several other cancers.
  • GW Pharmaceuticals reported positive results from its phase II trial evaluating two cannabis-derived drugs against recurrent glioblastoma multiforme. The London, UK-based company assessed the combination of tetrahydrocannabinol and cannabidiol (THC:CBD) in 21 patients being treated with temozolomide for their disease, who were randomly assigned to receive THC:CBD or placebo as add-on therapy. The 1-year survival rate was 83% among those given the cannabinoid combination, versus 53% in the control arm.
  • According to Fierce Biotech, Eli Lilly plans to cut 200 research and development jobs globally. This move has been framed as a “voluntary reallocation program” in which the company will seek out staffers willing to leave of their own volition. A spokesperson stated that Eli Lilly also plans to increase hiring in strategic areas, including immunology, across its United States research sites; Fierce Biotech noted that given the current political climate, “there is value in being seen to be on board with the ‘America First’ approach, particularly as it relates to jobs.”

February 3, 2017

  • Six scientific and medical organizations, including the American Association for Cancer Research, called on the Trump administration to consider the negative impact of denying people from seven countries entry to the United States. “We remain deeply concerned that restricting travel will prohibit participation in scientific meetings, where cutting-edge science and treatment methods are often first introduced,” they said. The order will also limit international collaborations, they noted, and “any loss of researchers and physicians will render the U.S. less competitive over time.”
  • President Trump’s proposal to slash regulations around drug development had a mixed reception. According to FierceBiotech, one lawmaker expressed concern that Trump “would consider putting the profits of pharmaceutical corporations ahead of safety.” PhRMA and BIO, however, appeared more upbeat about removing “outdated regulations that drive up costs and slow innovation,” estimating that limiting regulation could create 350,000 jobs over the next decade.
  • The House Committee on Energy and Commerce asked President Trump to clarify the potential impact of a federal hiring freeze on the FDA. Noting that the Cures Act, signed into law last December, includes provisions to assist the FDA in recruiting and retaining new scientific and technical staff, the committee queried whether this hiring authority would now be frozen, and, if yes, why. “Hamstringing the ability of our most critical public health agency to hire the personnel needed will hamper the FDA’s ability to fulfill its mission,” they concluded.
  • Guardant Health announced a multiyear partnership with The University of Texas MD Anderson Cancer Center in Houston, aimed at making comprehensive liquid biopsy a standard part of cancer care. The company will help MD Anderson build multiple liquid biopsy centers to speed the development of novel noninvasive assays using Guardant’s Digital Sequencing technology.
  • Roughly 9.9% of patients with colorectal cancer harbor germline cancer susceptibility gene mutations, researchers reported in the Journal of Clinical Oncology. This prevalence is higher than expected: Until now, physicians have focused on patients with hereditary Lynch syndrome (LS), about 3% in all, who are known to be at high risk for colorectal cancer. Germline mutations were found in non-LS genes, notably BRCA1/2; the researchers concluded that genetic factors underlying this disease “extend beyond well-recognized familial syndromes and are markedly more common than previously appreciated.”
  • According to a Nature Medicine study, although Ewing sarcomas share a DNA methylation signature, they also exhibit considerable epigenetic heterogeneity. Examining 140 samples of this bone cancer, the researchers observed consistent hypomethylation around EWS–FLI1, the fusion oncogene that marks this disease. Additionally, they reported epigenetic variation among tumors that fell along a continuum between mesenchymal and stem cell signatures; this heterogeneity was particularly pronounced in patients with metastatic disease.

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January 2017

January 27, 2017

  • British researchers generated universal chimeric antigen receptor (CAR) T cells to treat two infants with refractory relapsed CD19+ B-cell acute lymphoblastic leukemia, inducing remission prior to allogenic stem cell transplantation. Both responded within 28 days, according a report published in Science Translational Medicine. Currently, CAR T-cell therapies are developed one at time and personalized for each patient, but this new research suggests that an off-the-shelf, ready-made product could be a viable alternative approach.
  • A new analysis reveals that the risk of dying from cervical cancer is higher than previously thought—and that significant racial differences in death rates exist. Unlike prior estimates that included women who had had a hysterectomy and were therefore no longer at risk, this analysis, published in Cancer, included only women with a cervix. The researchers found that black women are dying from cervical cancer at a rate 77% higher than previously thought, whereas white women are dying at a rate 47% higher.
  • The FDA greenlighted Culver City, CA–based NantKwest’s application to launch a first-in-human clinical trial evaluating genetically modified natural killer (NK) cells against cancer. Researchers at NantKwest have engineered this immune cell type to express a variant of the CD16 receptor, as well as IL2; preclinically, adding these high-affinity (haNK) cells to therapeutic antibodies boosted antitumor immune responses. In the planned phase I trial, the safety of haNK cells will be evaluated in up to 16 patients with metastatic solid tumors.
  • According to a study published in Pediatrics, electronic cigarettes, also called e-cigarettes, are attracting adolescents who might not otherwise have started using tobacco products. Some experts have thought that e-cigarettes might be responsible for a decline in cigarette smoking among youth, but this first national analysis found that combined e-cigarette and cigarette use among middle and high school students in 2014 was higher than total cigarette use in 2009.
  • An international, 22-member panel of experts—European LeukemiaNet—released updated recommendations for the diagnosis and treatment of acute myeloid leukemia in adults. The new guidelines, published in Blood, were prompted by recent insights into the molecular and genomic causes of the disease, new genetic tests and tests for detecting minimal residual disease, and the development novel therapies.
  • Bristol-Myers Squibb reported positive data on its PD-1 inhibitor nivolumab (Opdivo) in two gastrointestinal cancers. In a phase I/II study of 214 patients with advanced hepatocellular carcinoma, the overall response rate (ORR) to nivolumab was 20%, with a 9-month overall survival (OS) of 74%. In a phase III trial of 493 patients with advanced gastric or gastroesophageal junction cancer, the ORR was 11.2% among those who received nivolumab, versus 0% in those given placebo; nivolumab also more than doubled the 12-month OS, from 10.9% to 26.6%.
  • The FDA granted Orphan Drug designation to two investigational agents from New York, NY–based TG Therapeutics for treating diffuse large B-cell lymphoma. The combination of TG-1101, a monoclonal antibody targeting CD20, and TGR-1202, a PI3Kδ inhibitor, is being evaluated in a phase II trial for patients with this disease; orphan status qualifies TG Therapeutics for various development incentives, including tax credits toward the costs of clinical trials.

January 20, 2017

  • An NIH spokesman announced that Francis Collins, MD, will continue to serve as the director of the NIH—at least temporarily. It remains unclear whether President Donald Trump will formally reappoint Collins or eventually name a successor.
  • The U.S. Department of Health and Human Services’ Office of Human Research Protections published a revised, final version of the so-called Common Rule, originally promulgated in 1991, to modernize and strengthen regulations that protect people who take part in scientific studies. Among other changes, the final rule establishes new requirements regarding information that must be given to prospective participants as part of the informed consent process, and it allows for the use of “broad consent,” meaning that subjects agree to the use of their information in unspecified future research. The rule goes into effect on January 19, 2018.
  • The U.S. Supreme Court announced that it will wade into a dispute between Sandoz and Amgen regarding when approved biosimilar versions of drugs can be brought to market. A lower court made Sandoz wait 180 days to sell Zarxio (filgrastim-sndz), a biosimilar version of Amgen’s Neupogen (filgrastim), after earning approval from the FDA; Sandoz has argued that giving Amgen 180 days’ notice prior to marketing the drug was sufficient and that the company shouldn’t have to wait another 6 months following FDA approval to sell its product.
  • The FDA issued a guidance document for industry on the nonproprietary naming of biologic products. The nonbinding recommendations call for the use of a “distinguishing suffix” composed of four lowercase letters “devoid of meaning” at the end of names for “originator biological products, related biological products, and biosimilar products containing related drug substances when other means to track a specific dispensed product are not readily accessible or available.” The distinguishing suffixes should, according to the agency, “help minimize inadvertent substitution of any such products that have not been determined to be interchangeable.”
  • The FDA also published drafts of several other guidance documents for industry. These include documents titled “Multiple Endpoints in Clinical Trials,” “Considerations in Demonstrating Interchangeability with a Reference Product,” and “Medical Product Communications That Are Consistent with the FDA-Required Labeling—Questions and Answers.” Comments on the draft documents can be submitted electronically at www.regulations.gov.
  • Following the presidential election in November, the FDA announced that it would not release a final guidance document on the regulation of laboratory-developed tests (LDT). This week, however, the agency issued a “discussion paper” on LDTs to synthesize comments it received following the publication of a draft document in October 2014 that was designed to balance concerns about overlapping and burdensome regulations with fears that a lack of oversight could compromise patient safety. According to the FDA, the document is meant to promote public discussion about LDT management; it contains no enforceable provisions.
  • Reuters reported that 22 of the world’s largest drug companies will contribute seed funding of $50 million over the next 3 years to a World Bank project to fight cancer and other noncommunicable diseases in poor countries. Dubbed Access Accelerated, the initiative was announced in Davos, Switzerland, during the World Economic Forum.
  • The Princess Margaret Cancer Foundation and the Princess Margaret Cancer Centre at University Health Network in Toronto, Canada, successfully concluded their “Billion Dollar Challenge” ahead of schedule. In less than 5 years, the organizations secured $532 million in philanthropic support and $520 million in research grants. The money will support personalized cancer medicine, including the expansion of research in epigenetics, bioinformatics, and immunotherapy.

January 13, 2017

  • Takeda Oncology acquired ARIAD Pharmaceuticals for $5.2 billion. The Japanese company has been looking to replenish its oncology portfolio, with its patent on bortezomib (Velcade), a blockbuster drug for multiple myeloma, expiring this year. This deal nets Takeda two key drugs: ponatinib (Iclusig), approved for chronic myeloid leukemia and Philadelphia chromosome–positive acute lymphoblastic leukemia; and brigatinib, an investigational ALK inhibitor under FDA review for non–small cell lung cancer (NSCLC).
  • The NCI Formulary, a public–private partnership between the agency and the pharmaceutical industry, launched with 15 targeted drugs from six companies. The formulary will enable researchers at NCI-designated cancer centers to more quickly access approved and investigational agents for preclinical studies and clinical trials—an otherwise lengthy negotiation process that can take up to 18 months. The NCI expects to double the number of participating companies and available drugs by the end of 2017.
  • Smoking costs the global economy more than $1 trillion annually in healthcare expenditures and lost productivity, according to a report from the World Health Organization and the NCI. By 2030, the number of tobacco-related deaths is projected to increase by a third—from 6 million to 8 million annually—with more than 80% of these occurring in low- and middle-income countries.
  • The FDA granted Priority Review to Merck’s PD-1 inhibitor pembrolizumab (Keytruda) combined with pemetrexed plus carboplatin as first-line treatment for advanced or metastatic NSCLC, with a decision expected by May 10. If approved, this could be the first regimen combining chemotherapy with an immunotherapeutic agent for lung cancer, the company said.
  • The FDA also granted Priority Review to Roche’s atezolizumab (Tecentriq) as first-line treatment for patients with advanced or metastatic urothelial carcinoma who are ineligible for cisplatin chemotherapy. The PD-L1 inhibitor is currently approved for treating patients with bladder cancer or NSCLC whose disease has progressed during or following platinum-based chemotherapy. A decision is expected by April 30.
  • According to a study in Cancer, mammography rates increased among women 70 years of age or older under the Affordable Care Act (ACA). The ACA eliminated out-of-pocket expenses for this preventive service, resulting in more women across all income and education levels opting to be tested, the researchers reported. However, there were no appreciable changes in the rates of colonoscopy, also free under the ACA; the researchers noted that “other procedural factors may remain as deterrents”—for instance, the test’s invasiveness.

January 6, 2017

  • The American Association for Cancer Research’s (AACR) Project Genomics Evidence Neoplasia Information Exchange (GENIE) made public nearly 19,000 deidentified genomic records collected from patients with cancer who were treated at one of eight renowned medical centers in the United States, Europe, or Canada. The data cover 59 types of cancer, including genomic information on almost 3,000 patients with lung cancer, more than 2,000 patients with breast cancer, and more than 2,000 with colorectal cancer. Data from Project GENIE, which was launched in November 2015, can be accessed through the AACR at www.aacr.org or downloaded from Sage Bionetworks.
  • In an interview with the Washington Post, Vice President Joe Biden said he will create a nonprofit organization so he can continue to focus on cancer issues after leaving office. He indicated his desire to “get Congress and advocacy groups to make sure [cancer] treatments are accessible for everyone … and that we have a more rational way of paying for them while promoting innovation.” He added that the new nonprofit, tentatively called the Biden Cancer Initiative, will be based in Wilmington, DE, or Washington, DC.
  • The cost of cancer drugs is the top challenge to cancer care, according to 83% of respondents to the Association of Community Centers’ seventh annual Trends in Cancer Programs survey; last year, 45% of respondents cited drug costs as the most significant challenge. A lack of reimbursement for supportive care services, such as survivorship care and financial counseling; the need for greater understanding among patients of what commercial insurance policies will cover; the burden of paperwork; and the need for greater, sustained funding for cancer research rounded out the top five issues.
  • According to a study published in JAMA Dermatology, the national incidence of melanoma in the United States continues to increase, but states in the Northeast are bucking that trend. Between 2003 and 2013, melanoma incidence dropped in five of the region’s nine states—Vermont, Rhode Island, New Hampshire, Connecticut, and Massachusetts. The researchers attributed the decline to strong prevention efforts by groups such as the Melanoma Foundation of New England.
  • Halozyme Therapeutics in San Diego, CA, reported positive data from a phase II study of its investigational drug PEGPH20 in advanced pancreatic cancer. PEGPH20, a PEGylated form of recombinant human hyaluronidase, is designed to temporarily degrade hyaluronan—a dense component of the tumor microenvironment that constricts blood vessels around cancer cells and impedes drug delivery. In the study, PEGPH20 plus nab-paclitaxel (Abraxane; Celgene) and gemcitabine nearly doubled median progression-free survival—from 4.5 months to 8.6 months—compared with nab-paclitaxel and gemcitabine alone.
  • The UK’s drug-cost watchdog, the National Institute for Health and Care Excellence (NICE), OK’d the use of pertuzumab (Perjeta; Roche), in combination with trastuzumab and chemotherapy, for the treatment of HER2-positive, locally advanced breast cancer—as long as Roche provides the drug at a discount, the amount of which was not disclosed. Several months ago, NICE rejected coverage of pertuzumab, saying that the cost outweighed the drug’s long-term benefits. NICE said that Roche agreed to a smaller price discount on trastuzumab emtansine (Kadcyla), but the drug was still not deemed cost effective.
 
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