Abstract
The FDA has awarded 15 grants totaling $22 million over 4 years through its Orphan Products Clinical Trials Grant Program. One third of the awards will support ongoing clinical trials of therapies for rare cancers for which there are few approved or effective treatment options.
The FDA has awarded 15 grants totaling $22 million to support research programs aimed at finding new therapies for patients with rare diseases. About a third of the money, to be dispersed over 4 years, will fund clinical trials investigating experimental treatments for rare cancers, including glioblastoma and neuroblastoma.
The Orphan Products Clinical Trials Grant Program was created in recognition of the challenges investigators face in securing funding for and recruiting patients into clinical trials focused on rare diseases, says program director Katherine Needleman, PhD, in the FDA's Office of Orphan Products Development. Since its inception in 1983, the program has funded over 600 studies, leading to the approval of more than 55 products.
“These grants are helping to support development of products for conditions where no current therapy exists or where the proposed product will be superior to the existing therapy,” says Needleman. A full list of awardees is available through the FDA's website.
Five of the grants will fund clinical trials related to rare cancers. For example, Steven DuBois, MD, a pediatric oncologist and director of experimental therapeutics at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center in Boston, MA, is leading a phase I study of SF1126 (SignalRX), a novel PI3K and mTOR inhibitor, in patients with high-risk neuroblastoma that has progressed despite treatment with chemotherapy and stem cell transplant.
SF1126 targets MYCN, which is highly amplified in about a third of children with this advanced form of the disease, says DuBois. Dual inhibition of PI3K/mTOR has been shown to degrade MYCN protein. An award of $750,000 will enable his group to complete their study of the drug, which was launched with private funding.
“It can be a challenge to convince funding agencies to direct significant funding toward research into a rare disease that affects so few patients,” says DuBois, who works with the New Approaches to Neuroblastoma Therapy consortium, a network of pediatric cancer centers that recruits patients with high-risk neuroblastoma for clinical trials. “Moreover, when it's time to conduct a trial, we need to open the trial at enough hospitals to be able to complete the study in a timely manner.”
Another award provides $2 million over 4 years for a phase II trial evaluating a combination of the tyrosine kinase inhibitor imatinib and the MEK inhibitor binimetinib (MEK162; Array BioPharma) in patients with newly diagnosed gastrointestinal stromal tumors. There are about 5,000 new cases of gastrointestinal stromal cancers each year in the United States, and almost all patients with metastatic or inoperable tumors eventually develop resistance to imatinib, the only approved first-line therapy, says the trial's principal investigator Ping Chi, MD, PhD, a medical oncologist at Memorial Sloan Kettering Cancer Center in New York, NY.
The goal is to develop a more effective first-line therapy to prevent or delay imatinib resistance, she says. The FDA award will cover a significant portion of the cost of the trial, which also received support from industry and private funders.
The potential benefits of funding research on rare diseases extend beyond treating patients enrolled in the current trials, notes DuBois.
“Even though neuroblastoma is rare, MYCN and MYC proteins play an important role in a wide range of human cancers,” he says. “If we can demonstrate proof of concept in neuroblastoma, that may open the door to new research and treatments in more common cancers.” –Janet Colwell