A study published in Science Translational Medicine, titled “Restoration of PP2A AαB56αC trimer formation by RPT04402 molecular glue in high-grade uterine cancer,” identifies a potential therapeutic strategy for aggressive uterine cancers driven by PPP2R1A mutations.
Case CCC members associated with the study include co-author Derek Taylor, PhD, Associate Director for Shared Resources and member of the Developmental Therapeutics Program, and Wei Huang, PhD, a member of the Immune Oncology Program.
Researchers investigated RPT04402, a small-molecule “molecular glue” that restores assembly of the tumor-suppressive PP2A protein complex disrupted by these mutations. In preclinical models, including patient-derived tumors, treatment induced tumor regression and extended survival.
The findings suggest a potential precision oncology strategy that restores defective protein-complex assembly, although clinical efficacy remains to be established.