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.