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Immunotherapy trial in rare skin cancer named one of 2016’s major advances in clinical cancer research and care

Study found long-lasting responses to immunotherapy drug in patients with Merkel cell carcinoma

The American Society of Clinical Oncology has recognized an immunotherapy study led by scientists at Fred Hutch and the University of Washington as one of the top advances of 2016 in clinical cancer research and care.

Paul Nghiem gives a presentation
Dr. Paul Nghiem speaks at an immunotherapy symposium at Fred Hutch last December. Photo by Robert Hood / Fred Hutch News Service

The study, published last April, found that patients with the rare skin cancer Merkel cell carcinoma, or MCC, who received the immunotherapy drug pembrolizumab had much longer-lasting responses to therapy than what is typically seen with chemotherapy. The study is included in ASCO’s “Clinical Cancer Advances 2017: Annual Report on Progress Against Cancer,” an independent annual review of the biggest breakthroughs and latest trends in clinical research and care for cancer.

“It is very exciting for patients with Merkel cell carcinoma, that this rare, ‘orphan’ disease is getting attention at the national level and that new, promising therapies are increasingly available,” said study leader Dr. Paul Nghiem of UW, who is a Fred Hutch affiliate clinical researcher.

When the first results from the study were released, it became easier for patients to access the drug off-label, Nghiem said. It also generated increased interest from pharmaceutical companies in studying this type of drug in MCC, which is particularly important for a rare and aggressive cancer with no U.S. Food and Drug Administration-approved therapies.

The study was conducted at eight sites nationwide through the Fred Hutch-based Cancer Immunotherapy Trials Network, which is led by the Hutch’s Dr. Martin “Mac” Cheever.

The study enrolled 26 participants who had not yet received a systemic therapy, like chemotherapy, for their disease. Around half of the participants’ cancers responded to the pembrolizumab ― similar to the response rate for chemo in MCC. However, of the 14 patients in the trial who had confirmed responses to pembrolizumab, 12 still had tumor regressions between two to 10 months later, when the scientists analyzed their data for publication.

Dr. Paul Nghiem explains how his team uses immunotherapy to combat a rare, often misdiagnosed form of skin cancer. Nghiem is affiliate investigator in Fred Hutch’s Clinical Research Division and head of dermatology at the University of Washington. Video by Robert Hood / Fred Hutch News Service

The researchers calculated that more than two-thirds of patients on the trial would have no evidence of disease progression for at least six months after treatment. In contrast, previous research has shown that chemotherapy only keeps this cancer at bay for an average of three months; by 10 months after starting chemotherapy, more than 90 percent of patients will have had their disease return, typically more aggressive than before.

Furthermore, the therapy triggered anti-cancer immune responses in participants with two very different types of MCC — one type caused by the Merkel cell polyomavirus, the other caused by extensive DNA damage from ultraviolet light exposure.

Also notable was that most patients experienced only relatively minor side effects, another contrast to chemotherapy.

Several immunotherapy advances were recognized in the ASCO annual report, which named immunotherapy as its “advance of the year.”

Read more about Fred Hutch achievements and accolades.

Susan Keown, a staff writer at Fred Hutchinson Cancer Research Center, has written about health and research topics for a variety of research institutions, including the National Institutes of Health and the Centers for Disease Control and Prevention. Reach her at skeown@fredhutch.org or on Twitter @sejkeown.

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Last Modified, February 13, 2020