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Promise and Progress - New Immune Therapy Shows Promise Against Melanoma

Reprogramming Cancer Cells - The Story of Epigenetics
Issue No. 1

New Immune Therapy Shows Promise Against Melanoma

Date: July 16, 2014


Suzanne Topalian
Suzanne Topalian, M.D.
Keith Weller

An experimental drug that targets immune cells and augments the immune system’s ability to specifically recognize and attack cancer cells shrank tumors or halted progression in a significant portion of patients with advanced melanoma.  The patients were part of a multi-institutional trial, testing a PD-1 inhibitor.  PD-1 is part of a suppressive pathway that prevents immune cells, which are poised to eliminate cancer cells, from delivering the fatal blow. In 2012, Kimmel Cancer Center investigators, led by Suzanne Topalian, M.D., Julie Brahmer, M.D., Drew Pardoll, M.D., Ph.D., and team, reported in the New England Journal of Medicine that the

PD-1 blockade appears to unleash a therapeutic immune reaction to several kinds of tumors in certain patients.   As a result of these Kimmel Cancer Center findings, a number of new clinical trials have begun. 

The current report on 107 patients with advanced melanomas that had not responded to two or more prior cancer treatments was published by the Kimmel Cancer Center and 11 collaborating institutions.  Thirty-three patients (31 percent) experienced significant reduction in the size of their tumors and extended survival.  The research team is working in the laboratory to better understand the mechanisms of PD-1 inhibitors to identify cancer biomarkers that will help pinpoint those patients most likely to respond to the treatment and further define the optimal dosage and duration of treatment.  Clinical trials in melanoma comparing anti-PD-1 therapy to standard therapy are ongoing.

Promising research and clinical outcomes involving anti-PD-1 therapies, were among the discoveries that led Science magazine to call cancer immunotherapy the scientific “Breakthrough of the Year” in 2013.

Funding for the trial was provided by Bristol-Myers Squibb and Ono Pharmaceuticals Co. Ltd. Bristol-Myers Squibb also provided the anti-PD-1 drug studied in the trial.

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