Pharmaceutical Business review

Ziopharm drug shows anti-leukemia activity

Preliminary results from the phase I hematology study are published in the 2006 American Society of Clinical Oncology annual meeting proceedings.

An anti-leukemia effect was seen in two of eight patients who had received extensive prior therapies. Blood leukemia cells disappeared in two patients and were substantially decreased in a third. The fourth patient stopped requiring frequent red blood cell (RBC) transfusions and had a marked improvement in functional ability. The company said that the trial is ongoing and dose escalation continues.

“We are encouraged by these results as they suggest ZIO-101 may have a role in treating patients with leukemia,” said Dr Kornblau. “Arsenic trioxide (Trisenox) is a very effective drug in acute progranulocytic leukemia, a rare form of AML, but ineffective in the far more common types of AML. ZIO-101, which can be given at more than 50-fold higher doses than arsenic trioxide because of a greater safety margin, may have broader applicability in leukemia and other blood and bone marrow cancers.”

ZIO-101 is a small molecule licensed from The University of Texas MD Anderson Cancer Center and Texas A&M University.