Acute Myelogenous Leukemia (AML) Patients May Benefit from New Class of Drug
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Source: H. Lee Moffitt Cancer Center & Research Institute (4/24/2007)


Moffitt doctor looks at the benefits of new drug in AML patients



Tampa, FL (April 24, 2007) – Because of its harmful risk and side effects, chemotherapy may not be an option for older adults suffering from acute myelogenous leukemia (AML). Doctors at H. Lee Moffitt Cancer Center & Research Institute are testing a new class of drugs that they hope will block the signals certain leukemia cells need to survive.

Dr. Jeffrey Lancet, a member of Moffitt’s Malignant Hematology Program, conducted a National Cancer Institute-sponsored Phase II study in which more than 150 previously untreated, poor-risk AML patients over the age of 65 were given an oral drug called tipifarnib. Fourteen percent of patients had complete remission and an additional 10 percent had partial remission or hematologic improvement. The drug was well tolerated by most patients, and was able to be administered on an outpatient basis.

The findings are published in a recent issue of the journal Blood.

“Tipifarnib is a member of a novel class of anti-cancer compounds that probably works very differently than traditional chemotherapy in the way that it kills leukemia cells,” said Lancet, principal investigator (PI) on the study. “Our study showed that this drug could benefit some patients without some of the attendant risks and side effects that you see from intensive intravenous chemotherapy in older patients. There is still a lot that needs to be done to optimize this drug’s benefit and to predict which patients are the most likely to benefit, but the first step was to prove that it has activity and is well tolerated.”

Several other projects are underway involving the drug tipifarnib. One is a recently completed Southwest Oncology Group Phase II trial in which different dosing levels and frequency were being tested. Moffitt was one of the leading institutions for accrual to this important trial. The second is a Phase I trial at Moffitt combining tipifarnib and bortezomib, a combination that appears to have better effect than either drug alone in preclinical studies. Lancet is the PI on that study. A third project looks at the combination of tipifarnib and chemotherapy in younger patients with AML. The project is being conducted at various institutions within the United States and Canada. Finally, there is an ongoing trial sponsored by Johnson & Johnson (the maker of tipifarnib) comparing tipifarnib to supportive care only in elderly patients with AML, a study that is designed to assess the benefit of tipifarnib in elderly patients who are not eligible to receive standard chemotherapy.

About H. Lee Moffitt Cancer Center & Research Institute

Located in Tampa, Florida on the University of South Florida campus, H. Lee Moffitt Cancer Center & Research Institute (www.moffitt.org) is the only Florida-based cancer center with the NCI designation as a Comprehensive Cancer Center for its excellence in research and contributions to clinical trials, prevention and cancer control. Moffitt currently has 15 affiliates in Florida, one in Georgia and two in Puerto Rico. Additionally, Moffitt is a member of the National Comprehensive Cancer Network, a prestigious alliance of the country’s leading cancer centers, and is listed in U.S. News & World Report as one of America’s Best Hospitals for cancer. Moffitt’s sole mission is to contribute to the prevention and cure of cancer.


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