Ziopharm preclinical data suggests ZIO-201 may enhance blood cell, bone marrow transplants

By Elaine Rigoli

Tampa, Fla., Feb. 17 - Ziopharm Oncology, Inc. presented preclinical data Friday demonstrating that ZIO-201, the company's proprietary alkylating agent, may have a role in conditioning for blood cell and bone marrow transplants.

Lee Roy Morgan, MD, PhD, Robert Peter Gale, MD, PhD and colleagues presented the data at the 2006 Bone Marrow Transplant Meetings in Honolulu.

Alkylators like cyclophosphamide and ifosfamide are widely used to prepare patients for blood cell and bone marrow transplants.

However, use of these drugs in high doses is complicated by substantial kidney, bladder and central nervous system toxicities because of the presence of certain metabolites, according to a company news release.

ZIO-201 is the active metabolite of ifosfamide and therefore is not associated with the adverse effects from other metabolites generated from the metabolism of ifosfamide. Further protective measures, like mesna and intensive intravenous hydration, are not required, the release stated.

ZIO-201 was active against a range of animal and human cancers in model systems. ZIO-201 was active against cancers resistant to cyclophosphamide and/or ifosfamide. These data suggest a possible role for ZIO-201 in conditioning regimens for blood cell and bone marrow transplants, the company stated.

ZIO-201 is currently in a phase 1 study in humans; one subject has stable disease for more than one year. The maximum tolerated dose of ZIO-201 has not been reached and the study is ongoing.

A phase 1/2 study in advanced sarcoma is expected to begin patient enrollment in the near future, the company announced.

Ziopharm is a biopharmaceutical company that develops and markets a portfolio of in-licensed cancer drugs to address unmet medical needs. The company's executive headquarters is located in New York City, with its business development operations located in Charlestown, Mass.

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