MD Anderson researchers develop therapy targeting undruggable MYC protein

Researchers at The University of Texas MD Anderson Cancer Center have developed a first-in-class therapy that targets the MYC protein, a driver in approximately 70% of all human cancers. Long considered ‘undruggable,’ MYC has been difficult for scientists to block effectively.

A preclinical study published in the journal Blood reveals that the experimental drug GT19630 disrupts a reinforcing cycle between the MYC protein and the GSPT1 protein. This ‘feedforward loop’ allows cancer cells to produce MYC, which in turn activates GSPT1 to sustain the cycle. By binding to both proteins, GT19630 marks MYC for disposal through the cell’s natural recycling system and simultaneously degrades GSPT1.

The therapy demonstrated strong anti-cancer activity in preclinical models of leukemia, lymphoma, and multiple myeloma. Notably, the treatment showed promise in addressing treatment-resistant and TP53-mutated diseases.