Benefit seen in nearly one–third of patients ‘with no hope at all of surviving’ their treatment–resistant Merkel cell carcinoma.
Almost two years ago, Judd received his first of many infusions of a new immunotherapy drug called avelumab on a clinical trial, and today over 90 percent of his tumor mass has disappeared. The devastating lack of options patients like him face at diagnosis has lessened, too: Because of the data from this clinical trial, avelumab (brand name Bavencio) has become the first systemic therapy approved by the U.S. Food and Drug Administration for Merkel cell carcinoma, and the first treatment of any kind approved for metastatic MCC. This is also the first approval the immunotherapy drug has notched from the federal agency — for any disease.

The Phase 2 trial included 88 patients like Judd who had metastatic MCC that had come back despite at least one round of chemotherapy or other treatments used off–label. Including Judd, 28 of those patients’ tumors shrank or disappeared in response to avelumab, and 23 of those 28 people have not seen their cancers grow again in the average of 10 months since they started treatment, according to the team’s most recent published data. In contrast, the typical patient with metastatic MCC has only a five percent chance of surviving without disease progression one year after starting chemo.

The approval as a first– and second–line therapy “is a really big deal,” said MCC expert Dr. Paul Nghiem of the University of Washington and Fred Hutchinson Cancer Research Center, who was a senior investigator on the avelumab trial. 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.

The avelumab trial, called JAVELIN Merkel 200, 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 will present updated data from the trial on April 3 at the annual meeting of the American Association of Cancer Research.

In comparison to the near–zero survival rates otherwise expected in patients like those on the trial, the team’s most recent calculations, presented by Kaufman at a scientific meeting in February, project that about a third of patients on the trial are on track to survive more than a year and a half ? and counting ? without their disease progressing.

“That’s an unheard–of number,” Nghiem said. 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.”

The researchers are now extending the avelumab trial to patients who have never received chemotherapy. Nghiem and colleagues at Fred Hutch are also studying avelumab in combination with a T–cell therapy for MCC, and they plan to present their first results from that trial later this year.