生物谷报道：美国弗吉尼亚联邦大学研究人员7日宣布，他们开发出一种新型化合物，可以有效缓解红细胞的镰状程度。研究人员认为，这种物质可望用于治疗镰状细胞贫血病。
镰刀型细胞贫血症是一种较常见的遗传性血液疾病，患者的红细胞中含有异常血红蛋白，细胞由正常情况下的圆形变为镰刀形，而且变得僵硬，导致红细胞阻塞小血管。
弗吉尼亚联邦大学研究人员开发出的这种名为5－ＨＭＦ的化合物，与红细胞中的正常血红蛋白和异常血红蛋白均能结合，能增强血红蛋白对氧气的吸附能力。这时，患者体内镰状红细胞的移动能力大大增强，镰状程度有效缓解，可以在血管内顺利前行，避免了血管阻塞。
研究负责人唐纳德·亚伯拉罕说，研究小组多年来针对镰状细胞贫血病开发出多种化合物。试验证明，5－ＨＭＦ是最为有效的一种。
弗尼吉亚联邦大学发表的新闻公报说，美国专利和商标局已于近日完成了5－ＨＭＦ相关专利的审查工作，该校享有利用5－ＨＭＦ开发相关药物的专利权。
英文原文：
Researchers Develop Novel Method for Treatment of Sickle Cell Disease
Virginia Commonwealth University researchers have developed a unique anti-sickling agent that may one day be effective in treating sickle cell disease, a painful and debilitating genetic blood disorder that affects approximately 80,000 Americans.
The research team led by Donald Abraham, Ph.D., the Alfred and Frances Burger Professor of Biological and Medicinal Chemistry, in the Department of Medicinal Chemistry in VCU's School of Pharmacy, has shown that 5-HMF, a pure compound developed by the team, has a high affinity for sickle cell hemoglobin and holds promise for the treatment of sickle cell disease.
"Our findings suggest that this anti-sickling agent may lead to new drug treatments and may one day help those suffering with sickle cell disease. This molecule, 5-HMF, is the most promising molecule to treat sickle cell anemia to come from our research group in more than 30 years," said Abraham, who is also the director of the Institute of Structural Biology and Drug Discovery.
The United States Patent and Trademark Office recently issued VCU a Notice of Allowance for a patent relating to a method of treating sickle cell disease with 5-HMF compound. A Notice of Allowance is a written notification that a patent application has cleared an internal review and it has been approved for issuance.
Sickle cell disease is caused by an abnormality in the hemoglobin molecule. Normal red blood cells carrying hemoglobin are smooth, round and flexible and can travel easily throughout blood vessels. However, sickle cells are stiff, abnormally shaped, red blood cells that do not flow freely through blood vessels. The sickle cells also may clot together causing a blockage to form which results in pain and potentially dangerous complications that can compromise a patient's organs.
According to Abraham, the 5-membered, heterocyclic, anti-sickling agent binds to hemoglobin to increase the oxygen affinity of both normal and sickle hemoglobin. In a patient with sickle cell disease, the binding action of 5-HMF would allow sickle cells to move more smoothly throughout the blood vessels of the body and prevent blockages from forming.
Abraham is internationally known for his groundbreaking work discovering and developing drugs that interact with hemoglobin. His research focus is to develop targeted therapeutics in sickle cell anemia, cardiovascular disease, stroke, cancer, Alzheimer's disease and radiation oncology.
This research was supported in part by a grant from the National Institutes of Health.
Xechem International, Inc., a biopharmaceutical company headquartered in New Brunswick, N.J., has entered into a licensing agreement with VCU Technology Transfer and has the exclusive worldwide rights for the production, sales and marketing of 5-HMF for use to fight sickle cell disease.
A recent grant from the National Heart, Lung and Blood Institute, part of the National Institutes of Health, awarded to Xechem International Inc., will allow researchers to carry out toxicity studies on 5-HMF. The research team will include researchers from VCU and Children's Hospital of Philadelphia, University of Philadelphia.
Working with Abraham to develop the anti-sickling agent were: Martin K. Safo, Ph.D., Richmond Danso-Danquah, Ph.D., and Gajanan S. Joshi, Ph.D., all researchers in the VCU Department of Medicinal Chemistry.
