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, according to a new preclinical study from The University of Texas MD Anderson Cancer Center published in Blood.

Researchers led by Michael Andreeff, M.D., Ph.D., professor, and Yuki Nishida, M.D., Ph.D., assistant professor, both of Leukemia, found that the experimental drug GT19630 interrupts a newly discovered cycle between MYC and GSPT1, resulting in strong anticancer 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.”