The science of using immunotherapy to treat cancer is advancing rapidly, marked by the National Cancer Institute's recent disclosure that a metastatic breast-cancer patient is now cancer-free, regulators' expected approval of a major lymphoma treatment this fall, and the unveiling Thursday of a partnership between government researchers and drug makers.
Immunotherapy, or immune cell therapy, describes a range of treatments that harness a patient's own immune system to target cancer. The approach doesn't work in all patients, but its success against some hard-to-treat cancers makes it the most closely watched area in cancer pharmaceuticals.
Underscoring the rapid advances, the National Institutes of Health and the NCI Thursday announced a $215 million medical collaboration with 11 medical companies, including AbbVie, Novartis AG and Johnson & Johnson. The NIH will contribute $160 million over five years to the research, and the companies will contribute $55 million.
Meanwhile a lymphoma drug from Kite Pharma Inc., expected to be approved soon, would be the second immunotherapy drug of its type to get a green light from the Food and Drug Administration and has promise for thousands of patients with a type of non-Hodgkin lymphoma that resisted other treatments. Kite agreed in August to be acquired by drug giant Gilead Sciences Inc. for about $11 billion, based on the hopes for the therapy.
Called axi-cel, the Kite medicine stems from a years long scientific collaboration with the NCI, underscoring the government agency's central role in developing immunotherapies.
NCI was the first to develop an experimental immunotherapy called CAR T, for "chimeric antigen receptor," a kind of genetically engineered immune cell. The NCI, a division of the National Institutes of Health, transferred the technology to develop the drug to Kite, and the company has paid up to $3 million a year to support the research.
The FDA recently approved another gene-based immunotherapy, Novartis's Kymriah, for a form of leukemia.
In another significant development, the cancer institute's prominent cancer researcher and chief of surgery, Steven A. Rosenberg, detailed for the first time an immunotherapy success against metastatic breast cancer, in a talk earlier this month.
In the lecture at a Boston meeting of the American Association of Cancer Research, Dr. Rosenberg reported on the first patient with metastatic breast cancer who is disease free nearly two years after her first immunotherapy treatment.
In the therapy, a person's own cells are multiplied billions of times and reinfused into the patient. Dr. Rosenberg's lab has already reported successes in treatment of melanoma, lymphoma, colorectal cancer and bile-duct cancer.
The patient is Judy Perkins, a 51-year-old structural engineer from Port St. Lucie, Fla. She was diagnosed with metastatic cancer—cancer that spread beyond the original location— in 2013. Then she underwent multiple regimens of chemotherapy and other standard treatment, to little avail.
But she learned of the NCI research, and in August 2015, doctors in Bethesda, Md., harvested her immune cells. In December 2015, she got an infusion of her own, intensified immune cells. Driving home, she said she already could feel a tumor that had shrunk. "I thought this thing could be working," she said.
By May 2016, her scans at the NCI came back clean—no detectable cancer. They have stayed clean, including during a visit to the NCI in Bethesda just last week.
Ms. Perkins is only one case. But the fact that she had metastatic breast cancer that is no longer detectable makes it very consequential. It follows reports from the Rosenberg lab about other internal-organ cancers, specifically colorectal and bile duct.
"We now see this treatment as a blueprint. We've taken the first steps in treatment of these common solid-tumor cancers that don't respond to anything," Dr. Rosenberg said. But he cautioned, "Each patient is a puzzle."
Dr. Rosenberg's interest in immunotherapy was piqued three decades ago, when he was struck by a chance encounter with a stomach-cancer patient who improbably recovered despite no treatment. It became a lifelong quest to discover how that patient had, in effect, cured himself. Scores of recoveries at the cancer institute of melanoma and lymphoma patients followed after immunotherapy treatment from his lab.
Now, his lab is exploring the promise of treating and accomplishing tumor regressions in far more common solid-tumor cancers of internal organs, including the breast, colon and bile-duct.
BY THOMASM. BURTON