Abstract
The NIH has launched a new collaboration with 11 biopharmaceutical companies and four academic cancer centers to identify the most effective biomarkers for immunotherapy. The public–private partnership will analyze patient samples from clinical trials and aims to develop a standard panel of biomarkers to determine which patients are likely to benefit from immunotherapy and to track their responses.
The NIH has announced a 5-year public–private partnership to increase the usefulness of cancer immunotherapy. The $215 million project's first goal is to pinpoint new biomarkers and refine existing ones so that doctors can identify patients who might benefit from immunotherapy and track their responses to treatment.
The FDA has approved two chimeric antigen receptor T-cell therapies as well as several checkpoint inhibitors for more than a dozen indications. Immunotherapy's track record is inconsistent, however, and it doesn't help many patients. Lack of standardized biomarkers remains one of the main obstacles to increasing the treatments' effectiveness.
“We need to know more about which patients will respond and how to improve the diagnostic testing that will allow us to predict that,” says James Doroshow, MD, director of the Division of Cancer Treatment and Diagnostics at the NCI. A uniform panel of biomarkers and standardized assays for measuring them would make it easier to compare results across trials, allow study replication, promote discovery of additional biomarkers, and provide other benefits.
Announced last month, the Partnership for Accelerating Cancer Therapies (PACT) involves 11 biopharmaceutical companies: AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Genentech, Gilead Sciences, GlaxoSmithKline, Janssen Pharmaceuticals, Novartis, and Pfizer. They will provide up to $55 million, and the NIH will supply the remaining $160 million of the project's budget.
The initiative also involves four academic cancer centers: Dana-Farber Cancer Institute in Boston, MA; The University of Texas MD Anderson Cancer Center in Houston; Stanford Cancer Institute in California; and Precision Immunology Institute and the Tisch Cancer Institute at Icahn School of Medicine at Mount Sinai in New York, NY. To discover potential biomarkers and evaluate existing ones, researchers at these centers will perform molecular and immune profiling on patient samples from industry-sponsored and publicly sponsored clinical trials.
Doroshow estimates that project scientists will analyze data from about 1,000 patients a year, using techniques such as whole-genome sequencing and immunohistochemistry. With this data, researchers may be able to discover which biomarkers are most informative—and determine the best ways to measure them.
“Success means initiating the development of standardized reagents and assays that are published and become standard operating procedure across the spectrum of clinical trials” for immunotherapies, says Doroshow. “That's achievable in a few years.”
Whether PACT reaches its goals depends on how much data it can gather, says Fred Ramsdell, PhD, of the Parker Institute for Cancer Immunotherapy in San Francisco, CA, who has no role in the initiative. “If we can get comparable data from different groups, I do think this is going to be useful,” he says.
Lisa Butterfield, PhD, of the University of Pittsburgh in Pennsylvania, who also isn't connected to PACT, gives it credit for addressing an important problem—the lack of uniformity among labs. “Coordination, standardization, and continuity in biomarker research are a big plus,” she says. If the project works out as planned, it “will move us closer to the goal of personalized, precision medicine.” –Mitch Leslie