A new chapter in CML treatment: the promise of asciminib

Abstract

Asciminib, a groundbreaking treatment for chronic myeloid leukemia (CML), has gained FDA approval as the first STAMP inhibitor. Targeting the myristoyl pocket of the BCR-ABL1 protein, this drug offers a unique mechanism to overcome resistance to traditional tyrosine kinase inhibitors (TKIs). Clinical trials, such as the ASC4FIRST study, demonstrate asciminib's superior efficacy compared to existing TKIs, achieving higher molecular response rates and fewer severe side effects. Its favorable safety profile, characterized by reduced vascular and pulmonary risks, supports improved patient adherence and long-term outcomes. While asciminib shows promise as a second-line or even frontline therapy, long-term data on its durability, adverse effects, and potential drug interactions are still evolving. Current research highlights its potential use in pediatric cases and in combination therapies for other leukemia types. Asciminib represents a significant advancement in CML treatment, paving the way for more targeted and tolerable cancer therapies.

Keywords: asciminib; chronic myeloid leukemia; tyrosine kinase inhibitors.