Drug could cut anemia blood tranfusions
Source: (cancerfact.com)
Friday, May 16, 2003

PARIS -- May 16, 2003 -– Patients with a form of anemia called myelodysplastic syndrome (MDS) may no longer face risky blood transfusions if early results of a new drug trial prove lasting, according to research presented this week at the Seventh International Symposium on Myelodysplastic Syndromes in Paris.

The drug, called CC-5013 PubMed Entry, is one of a new class of drugs to treat myelodysplastic syndromes, or MDS, a disorder that interferes with normal blood cell production, that can eventually lead to acute leukemia. Unlike aplastic anemia, in which the marrow is lacking all blood-forming cells, MDS marrow produces a mixture of normal and abnormal cells. The cause of the disease is unknown and so far, there is no cure.

The drug, named Revimidâ„¢PubMed by its maker Celgene, has fast-track designation from the Food and Drug Administration, which will speed up the regulatory process as there are no approved treatments for MDS on the market. At the moment patients simply use blood transfusions to manage the disease.

The study, funded by Celgene, showed that after an average of 36 weeks, 16 of 25 patients with MDS responded to the drug. Of those, 11 no longer needed blood transfusions, while four needed half the number of transfusions they had before taking the drug.

Revimid reduced or eliminated altogether the need for red blood cell transfusions in most of the severely anemic patients tested. Instead, patients began to produce their own red blood cells.

Celgene said the drug worked particularly well on patients with a form of MDS, called 5q syndrome, in which there is damage to one specific chromosome. None of the 5q patients needed blood transfusions after taking Revimid.

Revimid belongs to a new class of drugs called IMiDs, or Immunomodulatory Drugs, which are oral drugs chemically similar to thalidomide. Like thalidomide, IMiDs can modify or regulate the functioning of the immune system. IMiDs appear to have multiple actions, including the ability to inhibit marrow cell growth, as well as the growth of new blood vessels (angiogenesis). However, the precise mechanism of action is unknown and under investigation.

MDS patients can become anemic as a result of the failure to produce new red blood cells. One suspected cause is an excessive production of molecules known to cause inflammation, a process typically initiated to help the body heal. What follows is a cycle of marrow cell death and, in some cases, a rapid takeover by pre-cancerous cells.

Around 14,000 new cases of MDS are diagnosed each year in the U.S., and after the onset of the disorder patients survive between six months and five years.

The company said the results could be useful for understanding other types of blood disorders.

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Mr. Jan B. Wade
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Enhance Outcomes - Control Cost
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