Deforolimus is a novel small-molecule inhibitor of the protein mTOR, a “master switch” in cancer cells. Blocking mTOR creates a starvation-like effect in cancer cells by interfering with cell growth, division, metabolism, and angiogenesis. Deforolimus has been designated both as a fast-track and orphan drug product by the U.S. Food and Drug Administration (FDA) and as an orphan drug by the European Medicines Agency (EMEA) for the treatment of soft-tissue and bone sarcomas. ARIAD is pursuing this indication as the initial registration path for deforolimus.
Preliminary results suggest that it may be an effective agent for sarcoma. AP23573 exhibits single-agent activity in pts with advanced sarcomas in a phase II study. A clinical study with AP23573-07-302 (Ariad) is ongoing.
JS. P. Chawla, A. W. Tolcher, A. P. Staddon, S. Schuetze, G. Z. D’Amato, J. Y. Blay, J. Loewy, R. Kan, G. D. Demetri Survival results with AP23573, a novel mTOR inhibitor, in patients (pts) with advanced soft tissue or bone sarcomas: Update of phase II trial.
Journal of Clinical Oncology, 2007 ASCO Annual Meeting Proceedings Part I. Vol 25, No. 18S (June 20 Supplement), 2007: 10076
Rizzieri, David A., Feldman, Eric, DiPersio, John F., Gabrail, Nashat, Stock, Wendy, Strair, Roger, Rivera, Victor M., Albitar, Maher, Bedrosian, Camille L., Giles, Francis J.
A Phase 2 Clinical Trial of Deforolimus (AP23573, MK-8669), a Novel Mammalian Target of Rapamycin Inhibitor, in Patients with Relapsed or Refractory Hematologic Malignancies
Clin Cancer Res 2008 14: 2756-2762