A first-in-class therapy to target MYC, one of the most sought-after and difficult targets in cancer biology, showed promise in hard-to-treat blood cancers. Researchers led by Michael Andreeff, M.D., Ph.D., professor, and Yuki Nishida, M.D., Ph.D., assistant professor, both of Leukemia, found that experimental drug GT19630 interrupts a newly discovered cycle between MYC and GSPT1, resulting in strong anti-cancer activity in preclinical models of leukemia, lymphoma and multiple myeloma, including treatment-resistant and TP53-mutated disease.
“For decades, scientists have struggled to develop therapies that successfully block MYC, leading many in the field to describe it as undruggable,” Andreeff said. “By identifying a vulnerability in the relationship between MYC and GSPT1, we found a way to eliminate both proteins and disable a pathway many cancers depend on for survival.”