The Desmoid Tumor Research Foundation

October 2014

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

Author Information

Alexander J Lazar,Dennis P M Hughes,Shivaani Kummar

Department of Cancer Biology, The University of Texas MD Anderson Cancer Centeralazar@mdanderson.org.,Medical Oncology, National Cancer Institute.,Pediatrics, The University of Texas M.D. Anderson Cancer Center.

Publication

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.

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