The Leukemia & Lymphoma Society (LLS) helped every step of the way in discovery and development of menin inhibitors
Rye Brook, N.Y., December 6, 2024 – The U.S. Food and Drug Administration (FDA) has approved revumenib (Revuforj®), 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.
“Children and adults with hard-to-treat acute leukemias like this face limited treatment options and a poor prognosis. LLS 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,” says E. Anders Kolb, M.D., President and CEO of LLS.
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.
LLS is a leader in AML and menin research
LLS 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. LLS 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, LLS also leads two Master Clinical trials—one in children and another in adults—to find better and safer treatments for AML and other forms of acute leukemia.
“Our paradigm-shifting LLS Beat AML® 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,” says LLS Vice President Lore Gruenbaum, Ph.D. “And there’s 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.” LLS’s 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.
“Our goal is to give blood cancer patients and families additional years of quality life,” says Dr. Kolb. “To accomplish this, LLS 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.”
If you or a loved one need personalized disease, treatment or support information, you can contact one of our Information Specialists: https://www.lls.org/support-resources/information-specialists.