City of Hope Awarded NCI Research Grant for Potential Acute Myeloid Leukemia Drug

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At City of Hope, we push the boundaries of science to develop breakthrough therapies for AML and other aggressive cancers.

—Steven T. Rosen, MD

A City of Hope research team led by Steven T. Rosen, MD, City of Hope’s Provost and Chief Scientific Officer, has been awarded a $2.3 million Research Project Grant (R01) from the National Cancer Institute (NCI) to fund studies associated with a phase I/II clinical trial in relapsed/refractory adult acute myeloid ­leukemia (AML).

Steven T. Rosen, MD

Steven T. Rosen, MD

The drug—known as 8-chloro-adenosine—is a therapeutic small molecule that has shown potent anticancer activity against patient-derived leukemia cells, including leukemia stem cells. It has been well tolerated in animal studies. The Rising Tide Foundation is funding the clinical trial that tests the drug’s safety and efficacy, and the NCI grant will fund correlative studies in genomic profiling and the drug’s mechanism of action in patients.

Need for More Effective, Less Toxic Therapies 

“At City of Hope, we push the boundaries of science to develop breakthrough therapies for AML and other aggressive cancers,” Dr. Rosen said. “AML patients need targeted therapies that are more effective and less toxic than current chemo­therapies.”

The research project will evaluate the safety of 8-chloro-adenosine in AML patients. A phase II clinical trial will test the drug’s efficacy in AML patients whose disease has failed to respond to initial chemotherapy. The research will also detail the drug’s mechanism of action and further characterize the cytotoxic effect of the drug on leukemia stem cells. In addition, researchers will conduct genomic profiling of AML cells to generate gene-expression signatures that may help identify patients who may particularly benefit from 8-chloro-adenosine treatment. 

“We’re hopeful that successful completion of the study will not only extend or save the lives of those enrolled in the initial trial but also set the stage for continued clinical trials,” Dr. Rosen added. ■




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