ASH 2017 | Rapid responses with targeted agent in Phase I trial for systemic mastocytosis

Daniel DeAngelo

In a Phase I trial, patients with an advanced or aggressive form of systemic mastocytosis (AdvSM), a rare blood disorder, had rapid and durable responses with few adverse effects following treatment with an investigational drug that targets the genetic mutation found in more than 90 percent of cases.

“We are seeing a high rate of rapid and durable responses, with a very low rate of adverse side effects, in patients with an advanced or aggressive form of the disease,” said lead study author Daniel J. DeAngelo, MD, PhD, an associate professor of medicine at Harvard Medical School and a member of the adult leukemia program at the Dana-Farber Cancer Institute in Boston. “The rapidity of the improvement is extremely dramatic.”

The primary objective of the Phase I study was to identify the “maximum tolerated dose” of BLU-285 — that is, the highest dose that could be given without unacceptable levels of adverse effects. Secondary objectives were to assess the drug’s activity in the body, including anti-cancer activity.

This session was recorded at the American Society of Hematology 2017 Annual Meeting, held in Atlanta, GA.

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