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New Target Therapy for AML

Published: 2017-08-01 |

Source: Leukemia & Lymphoma Society

Today marks another significant advance in the offensive against acute myeloid leukemia (AML), one of the most deadly blood cancers that takes more than 10,000 lives in the U.S. each year. The Leukemia & Lymphoma Society (LLS) applauds the U.S. Food & Drug Administration's approval today of a new targeted therapy, enasidenib (IDHIFA), for patients with AML, who have a specific genetic mutation called IDH2.

The approval is for AML patients who have relapsed or do not respond to standard chemotherapy, and have the IDH2 mutation; this genetic marker represents approximately 12 percent of the 21,000 people in the United States diagnosed with AML each year.

Despite progress in treating other blood cancers, there have been few new therapies approved for AML in the past years, with the majority of patients treated with the same two chemotherapeutic agents, which are especially toxic in older adults. The prognosis for AML patients is very poor, especially for patients older than 60, with less than 20 percent of those patients surviving five years after diagnosis.

Recent advances in genomic technology that enable doctors to identify the underlying genetic drivers of an individual patient's disease have opened the door to a more personalized approach to treatment.

"AML patients desperately need new and better options for treatment. Adding enasidenib and midostaurin to the treatment armamentarium is a very encouraging step for AML patients, and is further proof that we are headed in the right direction with an exciting precision medicine approach to conquering this difficult cancer," said Louis J. DeGennaro, LLS President and CEO. "For too long, we've treated AML as a one-size-fits-all disease and we are changing that."

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