On May 2, 2017 ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP), a biopharmaceutical company focused on new immunotherapies, reported that an investigator-initiated Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1 trial infusing the Company’s CD33-specific CAR+ T therapy for relapsed or refractory acute myeloid leukemia (AML) is now active, with the first patient to be enrolled in the study expected to begin treatment in the third quarter of 2017 (Press release, Ziopharm, MAY 2, 2017, View Source [SID1234518785]). The CD33-specific CAR+ T cells incorporate a kill switch designed to eliminate the modified T cells under potential adverse safety conditions.
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"Relapsed AML is an aggressive disease with very poor outcomes," said William G. Wierda, M.D., Ph.D., Professor and Center Medical Director, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and principal investigator for the CD33 study. "In vivo preclinical animal studies have demonstrated that these CAR-T cells targeting CD33 exhibit specific killing of AML cells, eliminating disease burden, and significantly enhancing survival compared to controls, and I look forward to evaluating the safety and effectiveness of this gene therapy for patients with AML."
Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM added, "CAR-T cells expressing CD33 have shown promise in preclinical studies, but to-date, there has been limited experience in humans, representing a significant white space for us in treating AML. We look forward to seeing the positive preclinical results with our CD33-specific CAR-T cells translate to the clinic for relapsed/refractory AML patients who have far too few treatment options. In parallel with this Phase 1 study, we have also begun preclinical studies to evaluate rapid non-viral manufacturing of CAR+ T CD33-specific therapy for treatment of AML under point-of-care."
AML is a rapidly progressing cancer of the blood and bone marrow characterized by uncontrolled proliferation of immature blast cells with multiple associated gene mutations. The American Cancer Society estimates that there were approximately 20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. A majority of AML patients relapse or present with refractory disease and have overall poor prognosis.
This will be the second CAR target for genetically modified T cells to be studied at The University of Texas MD Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD Anderson.