Moffitt Cancer Center’s Phase 3 Study May Be Game-Changer for Acute Myeloid Leukemia

April 23, 2014

TAMPA, Fla. – Moffitt Cancer Center researchers say clinical trials for a new experimental drug to treat acute myeloid leukemia (AML) are very promising. Patients treated with CPX-351, a combination of the chemotherapeutic drugs cytarabine and daunorubicin, are showing better responses than patients treated with the standard drug formulation.

“Acute myeloid leukemia is an aggressive blood cancer with very low rates of treatment success, especially in older patients,” explained Jeffrey Lancet, M.D., senior member of the Department of Malignant Hematology and chief of the Leukemia Section at Moffitt.

AML is diagnosed in approximately 19,000 people in the United States each year and results in over 10,000 deaths annually. Many patients with AML are older and have additional medical conditions, which makes treatment difficult. Only 50 percent of patients respond to existing therapies and the average survival time is less than 1 year. Therefore, researchers are trying to find new treatment options to increase survival.

The phase 2 study included 126 newly diagnosed AML patients from 18 cancer centers across the United States and Canada. The patients received the standard chemotherapeutic agents, cytarabine and daunorubicin, or the novel drug combination CPX-351.

“Patients with AML who received CPX-351 had a higher likelihood of remission than patients who received standard chemotherapy,” said Lancet, who is the principal investigator and lead author of this study. “In addition, CPX-351 led to longer survival in the large subset of patients whose AML arose out of a previously diagnosed hematologic disorder, such as myelodysplastic syndrome.”

Scientists know that cytarabine and daunorubicin work best in a particular concentration ratio. Unfortunately, it is difficult to maintain that ratio in the body when the drugs are administered; therefore, they don’t work as effectively as they could. The drug CPX-351 is composed of substances called lipids. Lipids make up the outer membrane of cells and can form hollow spheres or vesicles that can be filled with drugs. Cytarabine and daunorubicin are present inside the CPX-351 lipid vesicle in a fixed ratio that is known to work most effectively.

“The results from this study show a high degree of activity in AML, leading to the opening of the new, larger randomized phase 3 study, which will definitively determine whether CPX-351 is superior to standard chemotherapy,” said Lancet.

The phase 3 CPX-351 clinical trial is currently open and recruiting patients. Those interested in learning more about study should contact Clinical Research Coordinator Nancy Hillgruber at Nancy.Hillgruber@Moffitt.org.

The results from the phase 2 study appeared in the journal Blood. The study was funded by Celator Pharmaceuticals.

About Moffitt Cancer Center
Located in Tampa, Moffitt is one of only 41 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitt’s excellence in research, its contributions to clinical trials, prevention and cancer control. Moffitt is the No. 1 cancer hospital in Florida and has been listed in U.S. News & World Report as one of “America’s Best Hospitals” for cancer since 1999. With more than 4,200 employees, Moffitt has an economic impact on the state of nearly $2 billion. For more information, visit MOFFITT.org, and follow the Moffitt momentum on Facebook, Twitter and YouTube.

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