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DFDA Approval of New Targeted Therapy for Acute Myeloid Leukemia is a Significant Advance for Deadly Blood Cancer

Last week marked 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 yesterday of a new targeted therapy for patients with relapsed AML, who have a specific genetic mutation.

There have been few new therapies approved for AML in the past 40 years. The prognosis for AML patients is very poor, especially for patients older than 60, with less than 20 percent of these patients surviving five years after diagnosis. But after four decades of little progress in AML, the FDA has approved two new therapies over the past three months, and more are under review.

“AML patients desperately need new and better options for treatment.” said Louis J. DeGennaro, LLS President and CEO. “These new approvals are further proof that we are headed in the right direction by taking a more personalized approach to treatment.”

Last year, LLS launched its own groundbreaking precision medicine study, the Beat AML Master Trial, a significant collaboration in which multiple targeted treatments for newly diagnosed AML patients are being tested simultaneously at multiple cancer centers around the country. The drug approved yesterday is among the investigational drugs being tested in the Beat AML trial, as a first-line therapy for newly diagnosed AML patients with the mutation.

“The Beat AML Master Trial is designed to deliver the right drug to the right patient at the right time,” said DeGennaro.

“LLS invests in research areas in which we know we can make a significant impact,” explains DeGennaro. “We recognized early on the urgency in finding new and better treatments for AML. After four decades and millions of dollars invested, we are finally seeing results for AML patients that give us real reason for optimism.”

Louis J. DeGennaro, Ph.D.
President and CEO