L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者的研究机构。 镰状细胞性贫血是一种遗传性疾病，发病率和死亡率很高，主要影响非洲血统的个体。目前批准的治疗方式在安全性、有效性和可用性方面有其局限性。最近的数据表明，L-谷氨酰胺作为一种口服药物治疗镰状细胞病的潜力。 初步研究在生理参数方面取得了可喜的成果。同样值得注意的是，患者对临床状态的主观感知持续改善，特别是在他们的能量水平和慢性疼痛方面。孤儿药国际的目标是评估L-谷氨酰胺治疗镰状细胞病的应用，以获得FDA的批准。根据该提议，计划进行一项具有客观临床终点的随机双盲临床试验。这将检验主观临床数据与客观终点的声明。在项目的第一阶段，将对20名可评价的镰状细胞贫血患者（18岁及以上）进行研究。主要终点将是患者的运动耐力和肺功能。次要终点将是红细胞的生理和生化参数。L-谷氨酰胺是我们日常饮食的一部分，并且有足够的数据证明其在本项目中使用的剂量的安全性。

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.