L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。镰状细胞性贫血是一种遗传性疾病，发病率和死亡率高，主要影响非洲血统的个体。目前批准的治疗方式在安全性、有效性和可获得性方面存在局限性。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结果。值得注意的是，患者对临床状态的主观感知，特别是他们的能量水平和慢性疼痛的持续改善。孤儿药物国际的目标是评估l -谷氨酰胺治疗镰状细胞病的应用，以获得FDA的批准。根据这一建议，计划进行一项具有客观临床终点的随机双盲临床试验。这将用客观的终点来检验主观临床数据的主张。在项目的第一阶段，将研究20名可评估的镰状细胞性贫血患者（18岁及以上）。主要终点是患者的运动耐力和肺功能。次要终点将是红细胞的生理生化参数。l -谷氨酰胺是我们日常饮食的重要组成部分，在这个项目中使用的剂量下，有充足的数据表明它的安全性。

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.