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Leukemia drug breakthrough study in New England Journal of Medicine


Alan List, M.D., leader of the Hematologic Malignancies Program at the H. Lee Moffitt Cancer Center & Research Institute, recently conducted a phase I/II trial of the experimental drug Revlimid showing promise as an innovative way to treat patients with myelodysplastic syndrome (MDS), a form of pre-leukemia. Given in pill form, Revlimid simultaneously blocks the growth of new blood vessels that nourish tumors (anti-angiogenesis) and stimulates the immune system to fight cancer cells. The study is reported in the Feb.10 issue of the New England Journal of Medicine.

Nearly 90 percent of MDS patients are anemic and require regular transfusions of red cells. In this study, 91 percent of the MDS patients with a chromosome abnormality named 5q minus syndrome became transfusion independent. The defective 5q chromosome abnormality may be linked to other serious cancers, including leukemias and small cell lung cancer.

In another finding of the same study, all the patients with the 5q deletion who became transfusion independent also went into cytogenetic remission, meaning that the chromosome abnormality disappeared.

List, a professor of interdisciplinary oncology at the University of South Florida, initially developed the phase I clinical trial of Revlimid for treatment of MDS following his laboratory observations that the agent improved the growth of red blood cell precursors from MDS bone marrow. Celgene will submit the seminal findings of List’s phase I trial - along with data from two recent confirmatory phase II clinical trials performed nationwide involving more than 350 patients with red blood cell transfusion-dependent MDS - to the U.S. Food and Drug Administration in a new drug application (NDA) for Revlimid as an innovative approach to treat the anemia of MDS patients with the 5q minus deletion.

In 2001, the National Cancer Institute awarded Moffitt the status of a Comprehensive Cancer Center in recognition of its excellence in research and contributions to clinical trials, prevention and cancer control. 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 the U.S. News & World Report as one of the top cancer hospitals in America. Moffitt’s sole mission is to contribute to the prevention and cure of cancer.

Andrea Brunais | EurekAlert!
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