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IMAGE: This is Courtney DiNardo, M.D. view more 

Credit: MD Anderson Cancer Center

HOUSTON ? Ivosidenib, an experimental drug that inhibits a protein often mutated in several cancers has been shown to be safe, resulting in durable remissions, in a study of acute myeloid leukemia (AML) with relapsed or refractory disease.

The multi-center Phase I trial, led by researchers at The University of Texas MD Anderson Cancer Center, was designed to determine ivosidenib’s safety and efficacy in treatment of patients with a form of AML in which the enzyme isocitrate dehydrogenase 1 (IDH1) is mutated. IDH1 mutations occur in 6 to 10 percent of AML patients. Findings are published today in the June 2 online issue of the New England Journal of Medicine and presented at the American Society of Clinical Oncology Annual Meeting in Chicago.

This first study of ivosidenib in humans, which enrolled patients between March 2014 and May 2017, administered a daily dose of the targeted IDH1 inhibitor to 258 patients.

“Ivosidenib, when administered orally as a single agent, was associated with acceptable side effects and induced durable and deep remissions,” said Courtney DiNardo, M.D., assistant professor of Leukemia at MD Anderson. “In the trial’s primary

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