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FDA Approves New Targeted Therapy for AML

FDA Approves New Targeted Therapy for AML

By Edward A. Faber, DO, MS, OHC; Real World Health Care, December 13, 2017, Blogs, 0 comments
August 9, 2017

Edward A. Faber, DO, MS

Edward A. Faber, D.O., MS
Blood & Marrow Transplant Specialist with OHC

Last week, news broke that the FDA had approved a new treatment for patients with acute myeloid leukemia (AML). I want to share the news from the Leukemia & Lymphoma Society (LLS) because it’s an exciting breakthrough for these patients. Despite progress in treating other blood cancers, there have been few new therapies approved for AML in the past 40 years.

People with acute leukemia need to be treated right away, and many can be cured. The goal is to make symptoms go away, which is called a remission. After going into remission, more therapy may be given to prevent a relapse. If you have chronic leukemia without symptoms, you may not need treatment right away. When treatment for chronic leukemia is needed, it can often control the disease and its symptoms, but it is seldom cured. People may receive maintenance therapy to help keep the cancer in remission. Stem cell transplants offer some people with chronic leukemia the chance for cure.

In addition to these and other treatments, new treatments are being tested in clinical trials that OHC often participates in. Check with your OHC doctor for more information about participating in a clinical trial or visit our Clinical Trials page on this web site a list of available trials.

Article from LLS

Rye Brook, N.Y. (August 1, 2017) – 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. 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.

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.

After four decades of little progress, today’s approval of enasidenib, developed through a partnership between Celgene and Agios, marks the second approval of a targeted therapy for AML patients in just the past three months and the first for patients with the IDH2 mutation. On April 28, the FDA approved Novartis’s midostaurin (Rydapt®), in combination with chemotherapy, for a subset of patients with a mutation called FLT3, which constitutes approximately 30 percent of the AML population.

“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.”

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. Enasidenib is among the investigational drugs being tested in the Beat AML trial, as a first-line therapy for newly diagnosed AML patients with the IDH2 mutation. In addition to its inclusion in the Beat AML Master Trial, enasidenib continues to be tested in other trials, both as a monotherapy and in combination with standard chemotherapy.

“The Beat AML Master Trial is designed to deliver the right drug to the right patient at the right time,” said DeGennaro. “As a patients-first organization, LLS is committed to leading and innovating new approaches to the way cancer research is conducted.”

To learn more about enasidenib (IDHIFA), visit https://www.idhifa.com/.



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