On August 20, 1992, Warren E. Leary of the New York Times reported in the Atlanta Journal/The Atlanta Constitution "Researchers have discovered that a drug being examined as a cancer therapy may also turn out to be a non-toxic treatment for sickle cell anemia and related blood disorders.

Scientists at Johns Hopkins School of Medicine and the National Cancer Institute said that the drug, which is commonly used for rare metabolic disorders in children, has been found to increase production of a fetal type of hemoglobin that is beneficial to sickle cell patients.

In the United States, sickle cell anemia affects tens of thousands of blacks, and variations of it are seen in whites of Mediterranean, Middle-Eastern and East Indian descent.

Hemoglobin is the oxygen-carrying protein of red blood cells. In sickle cell anemia, abnormal blood cells turn rigid, assume a sickle shape and block vessels. The painful episodes associated with the disease are fewer among patients who have a naturally higher level of fetal hemoglobin, which normally diminishes as people age.

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."

Since then clinical studies have been conducted on the effect of Sodium Phenylbutyrate combined with other drugs on Thalassemia and Cooley's Anemia. Some studies are in Phase II at this time.

On August 20, 1992, Warren E. Leary of the New York Times reported in the Atlanta Journal/The Atlanta Constitution "Researchers have discovered that a drug being examined as a cancer therapy may also turn out to be a non-toxic treatment for sickle cell anemia and related blood disorders.

Scientists at Johns Hopkins School of Medicine and the National Cancer Institute said that the drug, which is commonly used for rare metabolic disorders in children, has been found to increase production of a fetal type of hemoglobin that is beneficial to sickle cell patients.

In the United Sates, sickle cell anemia affects tens of thousands of blacks, and variations of it are seen in whites of Mediterranean, Middle-Eastern and East Indian descent.

Hemoglobin is the oxygen-carrying protein of red blood cells. In sickle cell anemia, abnormal blood cells turn rigid, assume a sickle shape and block vessels. The painful episodes associated with the disease are fewer among patients who have a naturally higher level of fetal hemoglobin, which normally diminishes as people age.

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."

Since then clinical studies have been conducted on the effect of Sodium Phenylbutyrate combined with other drugs on Thalassemia and Cooley's Anemia. Some studies are in Phase II at this time.


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