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

Hughes, Dennis P M; Kummar, Shivaani; Lazar, Alexander J · Clin Cancer Res · 2015

case_series · Level IV

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Abstract

A phase I trial of PF-03084014, an oral reversible γ-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.

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