A new class of experimental therapy is showing early promise against hard-to-treat blood cancers by targeting a protein long considered 'undruggable,' according to research presented at a major medical conference.
The therapy, known as a molecular glue degrader, works by hijacking the cell's protein disposal system to eliminate a specific protein that drives cancer growth. This protein, called GSPT1, has been difficult to target with traditional drugs because it lacks a clear binding pocket.
In a Phase 1 clinical trial, the drug, designated as CC-90009, demonstrated clinical activity in patients with relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). The study, led by researchers from the University of Pennsylvania, reported an overall response rate of 20% in the AML cohort, with some patients achieving complete remission.
'This is a proof-of-concept that we can target proteins that were previously considered undruggable,' said Dr. Selina Luger, the study's lead author and a professor of medicine at Penn. 'While the responses were modest, they are meaningful in a patient population with very limited options.'
The most common side effects included gastrointestinal issues and low blood counts, which were manageable. The findings were published in the journal Blood and presented at the 2026 American Society of Hematology annual meeting.
