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【medical-news】常规普药也能帮助治疗肾病

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这个帖子发布于14年零212天前，其中的信息可能已发生改变或有所发展。

Scientists discover that widely available drug also helps fight kidney disease

(Santa Barbara, Calif.) –– A widely available drug may be effective in treating kidney disease, report scientists at the University of California, Santa Barbara.

The drug is rapamycin, also called sirolimus, and is currently used as an immunosuppressant, to help prevent rejection of a new, transplanted kidney.

Over 600,000 people in the U.S., and 12 million worldwide, are affected by the inherited kidney disease known as ADPKD, short for autosomal-dominant polycystic kidney disease. In the U.S., the number of individuals affected by ADPKD is greater than the number affected by cystic fibrosis, muscular dystrophy, hemophilia, Down's syndrome, and sickle cell anemia combined. The disease is characterized by the proliferation of cysts that eventually debilitate the kidney, causing kidney failure in half of all patients by the time they reach age 50.

Currently no treatment exists to prevent or slow cyst formation, and most ADPKD patients require kidney transplants or life-long dialysis for survival, explained Thomas Weimbs, director of the laboratory where the discovery was made. Weimbs is assistant professor in the Department of Molecular, Cellular and Developmental Biology at UCSB.

The scientists studied the effects of rapamycin on mice. "When we administered rapamycin to mice with PKD and looked at their kidneys afterwards, we were absolutely amazed," said Weimbs. "The kidneys were smaller, had smaller cysts and had retained their function."

"We have known the genetic mutations that cause PKD for over a decade," explained Weimbs. "The genetic mutations are located in the gene for the polycystin-1 protein. Unfortunately, the function of polycystin-1 has remained poorly understood, which has made it difficult to devise a treatment strategy for this disease."

Weimbs and his research team found that polycystin-1 controls an important regulatory protein called mTOR. A defect in polycystin-1 leads to over-activation of mTOR. This, in turn, causes excess growth and proliferation of kidney cells, which results in the formation of thousands of cysts that eventually destroy the kidney.

Fortunately, a highly effective inhibitor of mTOR is well known. This drug, rapamycin, was originally discovered in the 1970s in soil from Easter Island. It is used for immunosuppression in kidney transplant patients to prevent rejection of the new kidney.

Weimbs and his colleagues wondered about treating kidney patients with rapamycin. Most kidney transplant patients keep their diseased kidneys in place and the transplanted kidney is an additional, third kidney. So his colleagues, David A. Goldfarb and Andrew Novick, at the Cleveland Clinic in Ohio, suggested studying transplant patients who had received rapamycin to help their bodies accept the new kidney.

The research team identified a group of four rapamycin-treated patients and found that their polycystic kidneys shrank in size by 25% over two years. The polycystic kidneys in a control group showed no change.

"Even though we only had a very small number of patients, this result is highly encouraging because it points in the right direction," said Weimbs.

It shows, for the first time, a connection between polycystin-1 and mTOR, and strongly suggests that rapamycin may be a promising drug for treating PKD, explained Weimbs. "The fact that rapamycin is already clinically approved for other uses will facilitate future clinical trials of the drug."

Co-authors on the paper with Weimbs are: Jonathan M. Shillingford and Seng Hui Low in the Department of Molecular and Cellular Biology at UCSB; Claire H. Larson at the Department of Cell Biology, Lerner Research Institute, Cleveland Clinic, Cleveland Ohio; Ryan Hedgepeth, Andrew C. Novick, and David A. Goldfarb at the Glickman Urological Institute, Cleveland Clinic, Cleveland, Ohio; Noel S. Murcia and Nicole Brown at the Department of Pediatrics, Case Western Reserve University, Cleveland, Ohio; Chris A. Flask at the Department of Radiology and Biomedical Engineering, Case Western Reserve University, Cleveland, Ohio; Albrecht Kramer-Zucker and Gerd Walz at the Department of Medicine, Nephrology, University Hospital Freiburg, Freiburg, Germany; Klaus B. Piontek and Gregory G. Germino at the Department of Medicine, The Johns Hopkins University School of Medicine, Baltimore, Md.

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2006-09-13 23:09 浏览 : 704 回复 : 2

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灵枢

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我认领此篇。

2006-09-14 06:24

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灵枢

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试着翻译如下，请各位战友多多指教。

科学家发现常规普药也能治疗肾脏病。
加利福尼亚大学科学家Santa Barbara报道：一种广泛被应用的药物可以有效地治疗肾脏疾病。
这种药物是瑞帕霉素，又被称为西罗莫司，目前被用于作为一种免疫抑制剂，有助于防止新移植肾脏发生排异。
在美国有六十万、全世界一千二百万人罹患一种遗传性肾脏病，常染色体隐性多囊肾病，简称为ADPKD。美国患ADPKD的人数超过囊性纤维化、肌营养不良、血友病、唐氏综合症、镰刀细胞性贫血数种疾病加起来的患者。这种疾病的特点是囊肿增殖，最终使肾脏衰竭，在一半的患者中，到达50岁时，发生肾衰。

托马斯.威母波斯，是这一发现所在实验室的系长，他解释到：目前还没有治疗能预防或延缓囊肿形成的方法，绝大部分ADPKD患者需要肾移植或终身透析来维持生命。威母波斯是UCSB分子，细胞发育生物系的副教授。

科学家们研究瑞帕霉素对老鼠的作用：“当我们将瑞帕霉素用于PKD老鼠，随后观察它们的肾脏，我们绝对很吃惊。”威母波斯说道：“肾脏变小了，囊肿也小了，而且保存了他们的功能。”

威母波斯解释道：“我们已经知道导致PKD的基因突变有二十年了。”“基因突变定位在多囊蛋白蛋白-1基因，不幸的是有关多囊蛋白-1的功能还知道得很少，这导致设计一个治疗方案治疗此病变得很难。

威母波斯和他的研究小组发现多囊蛋白-1控制一重要调节蛋白称为mTOR。多囊蛋白-1的缺陷使得mTOR过度激活。这反过来就导致了肾脏细胞的过度增殖，形成数以千计的囊肿，最终破坏肾脏。

幸运地是，一个mTOR高度有效抑制子已经被知道。这一药物，瑞帕霉素，最初是在1970年复活节岛土壤中被发现的。它被作为免疫抑制剂用于肾移植患者中，来防止新肾脏的排斥。

威母波斯和他同事想知道瑞帕霉素是否能治疗肾脏病人。大多数肾脏移植患者他们生病的肾脏保留在原位，而移植肾脏作为额外的、第三个肾脏。因此，他在俄亥俄州Cleveland诊所的同事大卫A.Goldfarb和安德鲁洛威克提示：研究移植患者接受瑞帕霉素有助于他们身体接受新肾脏。

研究小组观察了四位瑞帕霉素治疗的患者，发现他们多囊肾脏大小两年内减小了25％，而对照组的多囊肾没有改变。

尽管我们只有很少的病人，结果仍然是非常鼓舞人心的，因为它指明了正确方向。”威母波斯说道。

威母波斯解释，它首次证明了多囊蛋白-1与mTOR间的联系，强烈地提示瑞帕霉素可能是治疗PKD地一个有希望地药物。“实际上，瑞帕霉素已被批准临床用于其他用途，这将有助与将来该药地临床验证。”

本文与威母波斯合作者有：UCSB分子细胞生物系的Jonathan M. Shillingford and Seng Hui Low；俄亥俄州Cleveland诊所，Lerner研究院、细胞生物系Claire H. Larson；俄亥俄州Cleveland诊所Glickman 泌尿系的Ryan Hedgepeth, Andrew C. Novick, and David A. Goldfarb；俄亥俄州Cleveland， Case Western Reserve 大学儿科系Noel S. Murcia and Nicole Brown；俄亥俄州Cleveland， Case Western Reserve 大学生物工程、放射科Chris A. Flask；德国Freiburg,大学医院肾脏医学部Albrecht Kramer-Zucker and Gerd Walz；巴尔迪摩港市约翰霍奇金大学医学院医学部Klaus B. Piontek and Gregory G. Germino。

2006-09-14 17:49

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