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New Drug Effective Against AML in Lab Research from Wake Forest Baptist

WINSTON-SALEM, N.C. – April 30, 2012 – A new drug based on an older type of chemotherapy has proven less toxic and very effective for treating Acute Myeloid Leukemia (AML).

Scientists from Wake Forest Baptist Medical Center found that fluoropyrimidine, FdUMP [10], showed efficacy in AML cell lines from humans when tested initially in the laboratory.

“We wanted to know whether or not FdUMP [10] had any affect against AML,” said Timothy S. Pardee, M.D., Ph.D., an assistant professor of hematology and oncology at Wake Forest Baptist. “We looked at AML cell lines from humans, and we saw profound efficacy so it works really well. For almost every cell line, it was by far the most potent drug – even when compared to standard chemotherapy treatments.

AML is an aggressive malignancy of the bone marrow where the white blood cells that usually protect people from infections become cancerous, leading to bone marrow failure and death. This cancer is characterized by a high relapse rate and resistance to chemotherapy.

Pardee’s findings are published in this month’s issue of the journal Blood.

FdUMP [10] was first developed 10 years ago by Pardee’s collaborator and co-author, Wake Forest Baptist biochemist William H. Gmeiner, Ph.D., who currently is testing the drug on other types of cancer.

Pardee said the drug works by incorporating itself into the cell’s DNA, making it impossible for the cell to complete DNA replication. “That ends with the cell committing suicide and that’s what you want,” he said.

To better compare the toxicities of the FdUMP [10] to standard chemotherapies, Pardee and colleagues exposed normal mice to the various treatments to determine the effects on their organs. A veterinary pathologist reviewed all the slides with no knowledge of which mouse was treated with what drug. “He couldn’t tell the difference between the untreated mouse and the FdUMP [10]-treated mouse when it came to the bone marrow because the treated mouse had normal looking marrow. He could not see a difference,” Pardee said.

Pardee then did a bone marrow transplant experiment to see if the bone marrow stem cells were injured during any of the treatments. “We found that FdUMP [10] is very good at killing leukemia cells without harming normal ones,” Pardee said. “We’re really excited about these outcomes.”

Next steps include preclinical toxicology studies to meet FDA requirements to proceed to Phase 1clinical trials. "We know it's an uphill battle to get this drug into clinical trials, but we’re committed to seeing it through.”

FdUMP [10] is manufactured by Salzburg Therapeutics, located in Yadkinville, N.C. Gmeiner is founder/president and Pardee is Chief Medical Officer.

Funding support for this research was provided by the Doug Coley Foundation for Leukemia Research, the Frances P. Tutwiler Fund and the Translational Science Institute of Wake Forest Baptist. Additional support was provided by grant funding from the National Cancer Institute and National Institute of Health.

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