Dana-Farber Cancer Institute researchers are breaking new ground in the fight against systemic mastocytosis, a rare and heterogeneous condition that is often challenging for clinicians to accurately diagnose and treat.
Dana-Farber Cancer InstituteDec 9 2024 Dana-Farber Cancer Institute researchers are breaking new ground in the fight against systemic mastocytosis, a rare and heterogeneous condition that is often challenging for clinicians to accurately diagnose and treat.
A randomized phase 2, open-label, multicenter trial known as Apex, is evaluating the safety and early efficacy of bezuclastinib, a new inhibitor of KIT D816V, in patients with advanced SM. This oral drug is a potent and highly selective inhibitor that targets KIT D816V yet spares other closely related kinases. Dana-Farber researchers are reporting results from part one of the Apex trial focused on dose optimization that identified not only the optimal dose but also remarkable response rates.
The part 2 portion of the trial is now underway, involving 55 patients with advanced SM who will undergo treatment with bezuclastinib at the optimized dose.
Mastocytosis Blood Cancer Cell Children Clinical Trial Gene Hematology Hospital Kinase Laboratory Leukemia Mutation Receptor Research Tyrosine
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