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New Moffitt study reveals drug for MDS and anemia

A new study conducted by the H. Lee Moffitt Cancer Center and Research Institute shows promising results for an experimental drug that could help people suffering from myelodysplastic syndrome and anemia.

The new drug, Revlimid, has shown success in increasing the production of red blood cells as well as helping to give rise to cytogenetic remission, which occurs when the symptoms of a disease subside. Because of the rapid success of the drug, Alan List, the lead investigator of the study and the new program leader for hematological malignancies at Moffitt, said he hopes to get it approved by the FDA sometime next year.

“This summer, the company is putting the data together and if it looks anywhere near as good as it did from our Arizona trial, then we are going to take it to the FDA later this year,” List said.

In the two clinical trials to study the drug, it was shown that the drug successfully treats myelodysplastic syndromes and multiple myeloma, a bone marrow cancer with more than 14,000 new cases each year. MDS is a group of malignant disorders of blood cell production that produces deficits in blood production and, in some cases, eventually leads to acute leukemia. Specifically, MDS is a malignancy of the stem cells in bone marrow, which are the cells that make all blood cells — red, white and platelets.

“Some are lower grade that you can live for (5 to 10) years with and deal with just deficits in blood production. There really is a problem with ineffective production of the cells. They tend to die prematurely in the bone marrow so you’re left with a low white count, platelet count or hemoglobin count,” List said.

Of the three types of blood cell deficits caused by MDS, the most common deficit is of the red blood cells. Because of a low red blood count, most people who have MDS develop anemia and require blood transfusions an average of two to four times a week said List.

“Ninety percent of the people who have myelodysplastic syndrome develop anemia. It’s a precursor,” List said.

Revlimid, if approved by the FDA, will replace its parent drug thalidomide, which caused neurological toxicity and inheritable birth defects in some patients. Originally, thalidomide was used for anemia and MDS related disorders because of the drug’s ability to inhibit angiogenesis, or the production of blood vessel formations in a tumor.