Unleashing the Potential of the Immune System
On September 4, 2014, the Food and Drug Administration approved a new type of immunotherapy drug that will provide a much-needed option for patients with advanced or inoperable melanoma who no longer respond to other drugs – including the well-established immunotherapy agent ipilimumab (Yervoy).
The newly approved drug, pembrolizumab, will be marketed under the name Keytruda. Based on impressive results in clinical trials at Dana-Farber Cancer Institute and elsewhere, the FDA had designated pembrolizumab a “breakthrough therapy” and placed it on a fast track for approval.
“This drug is exciting because of its really striking response rate and good indications that these responses are durable” for a least a year, says Patrick Ott, MD, PhD, clinical director of the Center for Immuno-Oncology at Dana-Farber. “And it is very well tolerated, with very manageable side effects,” he adds.
Both Keytruda and Yervoy unleash the search-and-destroy power of the body’s immune system against cancer cells. These drugs, as well as others in clinical trials, are arriving on the crest of a new wave of immunotherapy research in which Dana-Farber scientists have played a prominent role.
The approval of Keytruda marks a first in the United States for a new class of immunotherapy drugs that block the PD-1 protein, which is commandeered by melanoma and other cancers to avoid attack by the body’s immune system.
PD-1 works with a pair of partner molecules, PD-L1 and PD-L2, to protect normal cells from being mistakenly harmed by immune soldier cells. Many cancers exploit this PD-1 pathway to hide from the immune system, but the PD-1/PD-L1 relationship was unknown until it was discovered at Dana-Farber Cancer Institute in 2004 by Gordon Freeman, PhD, (above) and colleagues engaged in basic research.
This discovery launched an entire field of immunotherapy research, which is now poised to open up a whole new era of targeted treatments with fewer side effects. PD-1 inhibitor drugs are in the pipeline for a wide range of cancers including lung, kidney, neurologic, sarcoma, head and neck, lymphoma, multiple myeloma, and assorted blood cancers, all of which are in clinical or pre-clinical trials at Dana-Farber.
Basic research, which leads to this kind of game-changing discovery in cancer science and medicine, is among the institutional priorities made possible at Dana-Farber Cancer Institute through the unrestricted gifts of the Monell Foundation.