Research by one of Versiti’s top scientists has contributed to development of a new drug that promises to increase survival time and dramatically improve the quality of life for people with advanced systemic mastocytosis (ASM), a debilitating and often fatal blood disorder.
Dr. Michael Deininger, Versiti’s chief scientific officer and director of the Versiti Blood Research Institute, is a co-senior author of a paper published Dec. 6 in the journal Nature Medicine detailing the safety and efficacy of avapritinib, a drug approved June of 2021 by the U.S. Food and Drug Administration for use against ASM.
Deininger participated in the research while serving at the University of Utah/Huntsman Cancer Institute (HCI) as Professor and Chief of Hematology and Hematologic Malignancies as well as Senior Director of Transdisciplinary Research and Director of the Huntsman Center of Excellence in Hematologic Malignancies and Hematology. He joined Versiti on Sept. 1.
Systemic mastocytosis is a rare disorder of malignant mast cells. It occurs most commonly in older adults but is not associated with any racial or geographic groups. Mast cells are found in multiple tissues and play a role in the body’s immune system and reaction to allergens. When mast cells accumulate and become unstable, they often release substances that can trigger severe allergic reactions, flushing, bone pain and diarrhea.
In ASM, the aggressive form of systemic mastocytosis, the malignant cells destroy normal tissues and compromise their function, leading to weight loss, fractures and bone marrow failure, with anemia and excessive bleeding due to low platelet counts.
Deininger estimates that 4,000 to 5,000 people in the U.S. may have the disorder. He is also a Professor of Medicine in the Division of Hematology/Oncology at the Medical College of Wisconsin (MCW) and a member of the MCW Cancer Center. He will soon start to see patients at MCW.
Avapritinib was developed to target a mutant receptor kinase on the surface of mast cells that triggers the growth of mast cells.
Deininger describes the drug as “undoubtedly a game-changer” for its effect on ASM’s symptoms and patients’ survival prospects. With previously available therapies, the survival for ASM patients is 28 months. With avapritinib, more than three quarters of patients were alive after two years.
Versiti is a provider of blood products and a national leader in blood health innovation. The Blood Research Institute provides innovations in the fields of transfusion medicine, transplantation and blood-related diseases. The development of avapritinib, Deininger said, “really highlights the power of translational research. If you know what’s wrong, you can fix it for the benefit of patients. This is what we are pushing at the Versiti Blood Research Institute”.