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Sickle Cell Anemia Studies

Researchers have found that Phenylacetate, a naturally occurring chemical in the body that is known to be non-toxic at high doses, sharply increases production of the fetal hemoglobin. The finding is so promising that doctors at Johns Hopkins quickly received approval from the Food and Drug Administration to conduct an initial human trial with anemia patients of Phenylbutyrate, a drug that produces Phenylacetate in the body.

While investigating the use of Phenylacetate as a potential anti-cancer drug, Dr. Samid and her colleagues found that the compound increased fetal hemoglobin production in human blood cells in the laboratory. In a letter published in the New England Journal of Medicine, the researchers wrote that increased levels of fetal hemoglobin were found in the blood of 15 children who had been treated for five to 65 months for a rare genetic disorder unrelated to sickle cell. The letter said that Phenylbutyrate and related compounds should be considered potential sickle cell treatments, either alone or in combination with other agents that enhance fetal hemoglobin production. Cells containing fetal hemoglobin increased four-fold in treated patients, from an average of 3 percent of red cells to 12 percent."

Copies of clinical trials are available upon request for doctors and medical researchers.

 

 

 

 

 

 

DISCLAIMER:The information presented is intended for educational purposes for health professionals and practitioners, and is obtained from published research. It is not intended to be prescriptive nor to replace the care of a licensed health professional in the diagnosis and treatment of illness.
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