New Drug Shows Promise for Rare Blood Cancers

New Drug Shows Promise for Rare Blood Cancers

Organ damage improved in 60 percent of patients with advanced systemic mastocytosis

WebMD News from HealthDay

By Amy Norton

HealthDay Reporter

WEDNESDAY, June 29, 2016 (HealthDay News) -- An experimental drug called midostaurin may reverse organ damage in people with certain rare, fatal blood cancers, an early clinical trial finds.

Researchers found that the drug at least partly reversed organ damage in 60 percent of patients with advanced systemic mastocytosis -- an umbrella term for several rare cancers that affect blood cells called mast cells.

Normally, mast cells release histamines and other chemicals during inflammatory and allergic reactions. In aggressive cases of systemic mastocytosis, abnormal mast cells build up in the bone marrow, skin, gut, spleen, liver and lymph nodes.

As a result, people with the cancers constantly feel sick, said Dr. Robert Hromas, a blood cancer specialist at the University of Florida.

"They're debilitating," he said. "You feel miserable all the time."

No good treatment options exist for people with advanced mastocytosis. So the new findings are "a real advance," said Hromas, who wasn't involved in the study.

"This is a drug that works," he said. "And until now, we've really had nothing."

The study does not prove that midostaurin extends people's lives, said Hromas, who is also a spokesman for the American Society of Hematology.

But patients who responded to the drug did see most of their symptoms improve, and they reported a higher quality of life -- which is important, Hromas pointed out.

The findings, published in the June 30 issue of the New England Journal of Medicine, add to evidence of midostaurin's benefits.

The drug, a twice-daily pill, has already been shown to lengthen the lives of patients with a subtype of acute myeloid leukemia.

In February, the U.S. Food and Drug Administration granted midostaurin "breakthrough therapy" status, which will speed the agency's review of the drug.

As it stands, the only drug approved to treat advanced systemic mastocytosis is Gleevec (imatinib). But it doesn't work for most patients, said study leader Dr. Jason Gotlib, an associate professor of medicine-hematology at Stanford University School of Medicine, in Stanford, Calif.

That's because about 90 percent of patients have tumors with a mutated gene that makes them resistant to Gleevec, Gotlib explained.

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