According to early clinical trial results published in the journal Blood, some patients with relapsed or treatment-resistant acute myeloid leukemia may achieve remission with an experimental targeted therapy.
AML is the most lethal of the blood cancers, which together are the third leading cause of cancer deaths in the U.S.; AML is responsible for more than 10,000 deaths each year. Despite advances in treating other blood cancers, the standard of treatment for AML – a combination of toxic chemotherapies – has remained the same for more than 40 years. Overall prognosis remains poor, with a five-year survival rate below 20 percent for patients over age 60.
Up to 15 percent of people with AML have a mutation in the IDH2 gene, which prevents their white blood cells from maturing into neutrophils, an integral part of the body’s immune system. Instead, these white blood cells become leukemia cells.
Unlike standard AML therapies, which aggressively target all white blood cells, the experimental therapy enasidenib inhibits the mutated IDH2 gene, allowing immature white blood cells to naturally mature into neutrophils.
Of the 239 IDH2 mutation positive AML patients included in a clinical trial evaluating enasidenib, 74 percent had treatment-resistant disease with few or no remaining therapy options available. Of those 176 patients, 71 (40.3%) responded to treatment, with 34 (19.3%) achieving a complete remission.
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Overall survival for these patients nearly tripled compared to previous studies evaluating other treatments in this patient population, up from 3.3 months to 9.3 months.
Further trials to assess enasidenib as a frontline therapy, in combination with standard AML treatments, and in treating other mutant IDH2 disorders like myelodysplastic syndrome (MDS) are planned or are ongoing.
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