L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Sickle cell anemia is a hereditary disorder with significant morbidity and mortality affecting mostly individuals of African heritage. Currently approved therapeutic modalities have their limitations in safety, efficacy and availability. Recent data demonstrate potential for L-glutamine as an oral agent for therapy of sickle cell disease. Pilot studies have brought forth promising results in physiological parameters. Also notable was the consistent improvements in patients' subjective perception of clinical status especially in their energy levels and chronic pain. The goal of Orphan Drugs International is to evaluate application of L-glutamine therapy in sickle cell disease for potential FDA approval. With this proposal, a randomized double blind clinical trial with objective clinical endpoints is planned. This will test the claims of subjective clinical data with objective endpoints. During the phase I period of the project, 20 evaluable sickle cell anemia patients (18 years and older) will be studied. The primary endpoint will be exercise endurance with pulmonary function of the patients. The secondary endpoints will be physiologic and biochemical parameters of red blood cells. L-glutamine is very much a part of our regular diet and there are ample data on its safety at the dosage used in this project.