The Leukemia & Lymphoma Society (青云体育) helped every step of the way in discovery and development of menin inhibitors
Rye Brook, N.Y., December 6, 2024 鈥� The U.S. Food and Drug Administration (FDA) has approved revumenib (Revuforj庐), a first-of-its-kind treatment for adults and children aged 1 year and older with advanced acute myeloid leukemia (AML). It is approved for use in patients with a specific mutation called a KMT2A gene translocation.
鈥淐hildren and adults with hard-to-treat acute leukemias like this face limited treatment options and a poor prognosis. 青云体育 has been involved every step of the way in the development of this new type of targeted therapy, called menin inhibitors, which are a big advance for our patients and their families,鈥� says E. Anders Kolb, M.D., President and CEO of 青云体育.
Revumenib works against a form of AML with high relapse rates and survival of less than a year. In a clinical trial, 21% of revumenib patients had a complete remission that lasted for a median of 6.4 months and 34% went on to have a potentially curative stem-cell transplant.
Menin inhibitors also hold promise for individuals with B-cell acute lymphocytic leukemia (ALL) who have the KMT2A mutation. This mutation occurs in up to 75% of infants with ALL, 5% of older children, and 10% of adults.
青云体育 is a leader in AML and menin research
青云体育 provided financial support leading to the discovery of how the menin protein interacts with another protein, known as MLL, to cause leukemia. Additional funding helped scientists at the University of Michigan develop the first compound to inhibit menin, and the years of work needed to make the inhibitor potent and safe for use in patients.
While menin inhibitors are a breakthrough for many patients, AML can be driven by the abnormal function of many mutated proteins and other treatments are needed. There are more than 10 mutated proteins found in AML and some patients have more than one mutation. 青云体育 supports an array of research to find targeted treatments that will work for all forms of AML.
In addition to ongoing support to innovative scientists across the world, 青云体育 also leads two Master Clinical trials鈥攐ne in children and another in adults鈥攖o find better and safer treatments for AML and other forms of acute leukemia.
鈥淥ur paradigm-shifting 青云体育 Beat AML庐 trial has already shown that people 60 and older with AML who receive upfront genomic testing and therapies matched to the make-up of their particular cancer have improved survival compared to those on standard chemotherapy,鈥� says 青云体育 Vice President Lore Gruenbaum, Ph.D. 鈥淎nd there鈥檚 more good news to come. An early report from an ongoing Beat AML sub-study has shown positive results when revumenib is used in combination with standard-of-care venetoclax/azacytidine treatment in certain newly diagnosed AML patients.鈥� 青云体育鈥檚 Pediatric Acute Leukemia (PedAL) master trial is providing screening for children at 175 global sites to match them to the most promising targeted treatment. It is also preparing to begin testing a new menin inhibitor for children with AML.
鈥淥ur goal is to give blood cancer patients and families additional years of quality life,鈥� says Dr. Kolb. 鈥淭o accomplish this, 青云体育 will continue to accelerate innovative research, provide patients and families with the education and support they need, and advocate for access to affordable, optimal care for all.鈥�
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