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IMAGE: At ASCO 2018, Daniel A. Pollyea, MD, MS presents positive results for phase 1 trial of ivosidenib against AML. view more 

Credit: University of Colorado Cancer Center

Early phase clinical trial results presented today at the American Society of Clinical Oncology (ASCO) Annual Meeting 2018 of 258 patients with IDH1+ acute myeloid leukemia (AML) treated with the IDH1 inhibitor ivosidenib show an overall response rate of 41.9 percent, with median progression free survival of 8.2 months. Twenty-four percent of patients achieved a complete response.

The gene IDH1 is cousin to another AML driver, IDH2, which is targeted by the drug enasidenib, which earned FDA approval in 2017. Though the function of both IDH genes is complex, previous work has shown that mutations in either IDH1 or IDH2 can activate other oncogenes while muting the action of tumor-suppressor genes. Both enasidenib and now ivosidenib seek to disrupt the action of these mutated IDH genes.

The current study takes place in the context of increased understanding of the underlying genetics that drive AML.

“Ten years ago, the first cancer genome was sequenced, and it was from an AML patient,” says Daniel A. Pollyea, MD, MS, investigator at the University of

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