New, Tolerable γ-Secretase Inhibitor Takes Desmoid Down a Notch.

Summary

A phase I trial of PF-03084014, an oral reversible γ-secretase inhibitor (GSI), in solid tumor malignancies shows drug tolerability in patients. Evidence of Notch pathway inhibition was demonstrated in peripheral blood. A surprisingly high rate of response was seen in desmoid tumors, a rare but often locally aggressive sarcoma.