The Leukemia & Lymphoma Society (LLS) announced the first pediatric patient has received treatment in a new subtrial of its Pediatric Acute Leukemia (PedAL) Master Clinical Trial (NCT04726241). PedAL is a first-of-its-kind integrated worldwide master trial that tests multiple targeted drugs simultaneously to accelerate treatments for leukemia, the most common childhood cancer.

Infants and children with specific types of relapsed/refractory leukemia (KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant acute leukemia) are now eligible to enroll in the subtrial, which is investigating a combination of chemotherapy and a menin inhibitor.

This subtrial involves pediatric patients with some of the most common and hardest-to-treat leukemias. In fact, 80% of infants with acute lymphoblastic leukemia have a genetic rearrangement of the KMT2A gene. About 40% of infants with acute myeloid leukemia have one of the three specific types of leukemia that the trial's treatment targets.

Menin inhibitors are a new and promising class of drugs that block the interaction between other proteins and the menin protein. Research has shown that the menin protein is responsible for driving progression of certain types of leukemia.

The new subtrial is open in North America at Children's Hospital of Philadelphia, Cincinnati Children's Hospital, St. Jude Children's Research Hospital, Memorial Sloan Kettering Cancer Center, Lurie Children's Hospital of Chicago and SickKids - The Hospital for Sick Children in Toronto. Global trial sites are open in Spain, the Netherlands, France and Austria.

"It is rare for a trial to be so ambitious and forward thinking about the needs of children while studies in adults are still ongoing. The new subtrial is evaluating the safety, efficacy and dosing for the treatment of certain acute leukemias in pediatric patients," says E. Anders Kolb, M.D. President and Chief Executive Officer of The Leukemia & Lymphoma Society. "Historically, cancer treatments have been evaluated in adults first and if successful, then children. This has resulted in a nearly decade-long delay between when therapies are first available for adults compared to children."