Doctors at Seattle Cancer Care Alliance gained significant ground last week after the U.S. Food and Drug Administration granted approval of a drug used in a clinical trial to treat patients with a rare form of skin cancer, prompting one SCCA cancer expert to describe results as “unheard of.”
“While skin cancer is one of the most common cancers, patients with a rare form called Merkel cell carcinoma have not had an approved treatment option until now,” said Dr. Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence, in a statement.
“The scientific community continues to make advances targeting the body’s immune system mechanisms for the treatment of various types of cancer. These advancements are leading to new therapies—even in rare forms of cancer where treatment options are limited or non-existent.”
This is the first FDA-approved treatment for metastatic Merkel cell carcinoma, commonly referred to as MCC.
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The Phase II clinical trial of avelumab (pronounced a-VELL-oo-mab), called JAVELIN Merkel 200, focused on a drug therapy used to treat 88 patients who had metastatic MCC that had come back despite at least one round of chemotherapy or other treatments used off-label. Twenty-eight of those patients’ tumors shrank or disappeared in response to avelumab, and 23 of those 28 have not seen their cancers grow again in the average of 10 months since they started treatment, according to the study’s most recently published data. In contrast, the typical patient with metastatic MCC has only a 5 percent chance of surviving without disease progression one year after starting chemotherapy.
“That’s an unheard-of number,” said MCC expert Dr. Paul Nghiem of Seattle Cancer Care Alliance, University of Washington and Fred Hutchinson Cancer Research Center, who was the senior investigator on the avelumab trial. These patients “had no hope at all of surviving, because they had failed chemotherapy already, and that really is a grim situation: immune-suppressed, and the cancer is angry and becomes resistant to additional chemotherapy.”
Metastasis is common in MCC ― the cancer spreads in about a third of patients. The FDA has granted avelumab a broad approval based on the results of this clinical trial, for use in any adult or adolescent patient with metastatic MCC.
The approval as a first- and second-line therapy “is a really big deal,” said Dr. Nghiem. Nghiem’s team conducted foundational work on the role of immune cells in MCC that paved the way for immunotherapy trials in the disease, including a trial he leads of another immunotherapy drug as a first-line therapy in advanced MCC.
Researchers are now extending the avelumab trial to patients who have never received chemotherapy.
Dr. Nghiem noted that the avelumab trial tested the drug in “a worst-case scenario” ― in patients whose immune systems were suppressed as a result of chemotherapy ― and the FDA’s broad approval means that real-world data will likely look better as patients are treated on the drug before receiving chemotherapy. But there’s still room for improvement, he said.
“Just use the best drug first, that’s clear now,” Dr. Nghiem said. “But even when you do that, only a little more than half of patients will have a good response that lasts. We need to make that better.”
The avelumab trial is led by principal investigator Dr. Howard Kaufman of the Rutgers Cancer Institute of New Jersey and funded by industry. One of its 35 sites worldwide is the Fred Hutch/University of Washington Comprehensive Cancer Consortium. Kaufman presented updated data from the trial on April 3 at the annual meeting of the American Association of Cancer Research.
Only 2,000 Americans a year are diagnosed with MCC. In contrast, according to the American Cancer Society, more than 250,000 are diagnosed with breast cancer; 135,000 are diagnosed with colorectal cancer; and 87,000 are diagnosed with melanoma, which is often called the most deadly form of skin cancer. But MCC is three times more likely to be lethal than melanoma.
– SCCA and Fred Hutch staff