生物谷報(bào)道：美國弗吉尼亞聯(lián)邦大學(xué)研究人員7日宣布,，他們開發(fā)出一種新型化合物,，可以有效緩解紅細(xì)胞的鐮狀程度,。研究人員認(rèn)為,，這種物質(zhì)可望用于治療鐮狀細(xì)胞貧血病,。
鐮刀型細(xì)胞貧血癥是一種較常見的遺傳性血液疾病,，患者的紅細(xì)胞中含有異常血紅蛋白，細(xì)胞由正常情況下的圓形變?yōu)殓牭缎?，而且變得僵硬,，?dǎo)致紅細(xì)胞阻塞小血管。
弗吉尼亞聯(lián)邦大學(xué)研究人員開發(fā)出的這種名為5－ＨＭＦ的化合物,，與紅細(xì)胞中的正常血紅蛋白和異常血紅蛋白均能結(jié)合,，能增強(qiáng)血紅蛋白對(duì)氧氣的吸附能力。這時(shí),，患者體內(nèi)鐮狀紅細(xì)胞的移動(dòng)能力大大增強(qiáng),，鐮狀程度有效緩解，可以在血管內(nèi)順利前行,，避免了血管阻塞,。
研究負(fù)責(zé)人唐納德·亞伯拉罕說,，研究小組多年來針對(duì)鐮狀細(xì)胞貧血病開發(fā)出多種化合物。試驗(yàn)證明,，5－ＨＭＦ是最為有效的一種,。
弗尼吉亞聯(lián)邦大學(xué)發(fā)表的新聞公報(bào)說，美國專利和商標(biāo)局已于近日完成了5－ＨＭＦ相關(guān)專利的審查工作,，該校享有利用5－ＨＭＦ開發(fā)相關(guān)藥物的專利權(quán),。
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.
