iPS药物发现：阿尔茨海默氏病全球首个临床试验：京都大学/三重大学的7家医院
使用iPS进行药物发现：
对患者使用“在使用iPS细胞的实验中已被证实具有减少阿尔茨海默氏病/病原体作用的药物”。
京都大学及其他研究小组于4日宣布：“我们将使用这种药物进行阿尔茨海默氏病的临床试验。”
该临床试验将于5日开始，预计该药物将于本月晚些时候开始。
这是使用iPS细胞进行药物发现的全球首例阿尔茨海默氏病临床试验。
阿尔茨海默氏病：
淀粉样β蛋白在脑中过度积聚会引起认知障碍等。
没有治愈方法，到2050年，全球将有超过1亿患者。
试验中的患者：
在包括京都大学和三重大学附属医院在内的7家机构进行了临床试验。
受试者是患有轻度至中度认知障碍的10名遗传性阿尔茨海默氏病患者。
不会进行公开发售。
采取的补救措施：
帕金森病药物的使用
每天三次“溴西汀”
管理超过36周，
确认安全性和有效性。
旨在通过抑制进展来改善症状。
iPS细胞的药物发现：开发过程
通过从患者的皮肤制造iPS细胞并将其转化为脑神经元来重现疾病状态。
通过施用约1200种现有药物，研究了抑制淀粉样蛋白β产生的作用。
结果，“发现溴隐亭减少了一半的产量”，从而进行了这项临床试验。
产经新闻
https://www.sankei.com/smp/life/news/200604/lif2006040023-s1.html
Investigator-initiated clinical trial for familial Alzheimer’s disease
- Haruhisa Inoue, Professor, Center for CiRA, Kyoto University,
- Hidekazu Tomimoto, Mie University Graduate School of Medicine,
- Haruhiko Banno, Associate Professor, Kyoto University Graduate School of Medicine,
is leading a new investigator-initiated clinical trial to study safety and efficacy of TW-012R (bromocriptine, Towa) for Alzheimer’s disease patients with mutations in presenilin-1 gene1).
This study consists of an initial double-blind trial period2) and the latter open-label trial period2).
A new clinical trial notification
was approved by the Pharmaceuticals and Medical Devices Agency (PMDA), and the clinical trial starts at the participating institutes.
CiRA Junior Associate Professor Takayuki Kondo Prof. Inoue
have prepared iPS cells derived from Alzheimer’s patient and differentiated them into cortical neurons3) to screen efficacy of medicinal compounds.
Many of these agents have been marketed as approved medicine for treating other diseases.
One of the main causes of Alzheimer’s disease is an accumulation of amyloid beta protein in the brain.
Thus, agents that abbreviate its accumulation will be effective for Alzheimer disease.
Our screening4) results identified bromocriptine with the most potent effect among the screening compounds.
Bromocriptine is an approved medicine with indications Parkinson’s syndrome and pituitary disorders such as hyperprolactinaemia in Japan.
CiRA | Center for iPS Cell Research and Application, Kyoto University
https://www.cira.kyoto-u.ac.jp/e/pressrelease/news/200609-150000.html