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MDS Drug Creates Great Interest at Major Oncology Meeting

Tuesday, May 24, 2005

The FDA is reviewing a new drug derived from thalidomide, once used for morning sickness, to treat a malignant blood disorder know as MDS, following a series of clinical trials involving the University of Rochester Medical Center.

In one study, about 75 percent of the 148 voluntary participants in the United States who took Revlimid, the experimental medication, either went into complete remission or stabilized enough they no longer required monthly blood transfusions to stay alive. The results excited oncologists who gathered in Orlando last week for the annual meeting of the American Society of Clinical Oncology. An MDS research group was invited to present its findings to the ASCO general assembly.

“I think we can say the majority of these patients will enjoy a year or more of remission, without being dependent on blood transfusions,” said John Bennett, M.D., clinical director of the hematology-oncology service at the James P. Wilmot Cancer Center at the URMC, and a co-author for the MDS research group. Bennett also founded the national Myelodysplastic Syndromes (MDS) Foundation and serves as chair.

The Food and Drug Administration has agreed to quickly review Revlimid, which is being tested as well in patients with multiple myeloma, brain and kidney cancer. New Jersey-based Celgene Corp., the drug manufacturer, is funding the research.

MDS is caused by an abnormal stem cell that changes the way blood cells are produced. Sometimes referred to as “pre-leukemia,” MDS is a serious a form of anemia that can evolve into acute myeloid leukemia. It afflicts more than 30,000 elderly Americans at any given time. Median survival is about five years.

 Among MDS patients are subgroups with various levels of disease stability. In this Phase II trial, the drug was tested in a subgroup of patients with a form of MDS that results from a chromosomal abnormality. Within that group, 75 percent had some positive response to the experimental therapy. In another trial involving MDS patients without the missing chromosome, the response rate was about 40 percent, Bennett said.

Celgene developed Revlimid by modifying thalidomide, which was restricted by the FDA after it was determined to cause birth defects. Revlimid was designed to be more potent against cancer but less toxic to the body than thalidomide. It works by interfering with the growth and pathways of malignant blood vessels, said Bennett, an emeritus professor of Medicine.

The clinical trial results presented at ASCO also showed that Revlimid works rather quickly (within about four weeks), and produces some side effects such as stomach upset and low blood counts. About 10 percent of the enrolled volunteers left the trial due to serious side effects. Two deaths occurred during the trial – less than 1 percent of patients enrolled -- that might have been related to side effects from Revlimid, the investigators reported.

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