New, tolerable γ-secretase inhibitor takes desmoid down a notch.

A phase I trial of PF-03084014, an oral reversible gamma-secretase inhibitor, 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 sometimes locally aggressive sarcoma. (C) 2014 AACR.