阿斯利康联姻哈佛干细胞向糖尿病发起挑战，背后有着非常感人的故事

英国2大制药巨头葛兰素史克（GSK）和阿斯利康（AZN）最近好忙！前面葛兰素史克刚牵手冷泉港向肥胖和二型糖尿病发起冲锋，这边阿斯利康（AZN）也不甘示弱联姻哈佛干细胞研究所（HSCI）向糖尿病发起挑战。该公司近日与HSCI签署了一项为期5年的研究合作，将一种干细胞制造人类胰岛β细胞的突破性技术，用于其化合物文库的筛选，寻找有望恢复胰岛β细胞活力的潜在新药。该项合作的另一初衷，是为更好地了解糖尿病患者体内胰岛β细胞功能的衰退机制，相关研究成果将通过同行评议的期刊向科学界公布。

在1-型糖尿病患者体内，胰岛β细胞被自体免疫反应所摧毁，患者必须注射胰岛素来维持正常的血糖水平。而在2型糖尿病患者体内，胰岛β细胞要么不正常工作要么数量减少。但人类β细胞在数量和可用性方面的局限，限制了该方面的研究。若能通过干细胞产生无限的胰岛β细胞，就能满足众多患者的需要，同时将成为干细胞生物学应用到临床治疗工作的重大尝试。

哈佛干细胞研究所（HSCI）联合主席兼霍华德休斯医学研究所研究员Douglas A. Melton教授领导的研究团队开发出了一种突破性技术，可利用成人细胞直接生成的人类诱导多能干细胞（iPSC）制备大量的胰岛β细胞，这些细胞能够产生胰岛素，而且在所有重要指标方面均与正常人体中的胰岛β细胞相似。

根据双方达成的协议，阿斯利康将为HSCI的一支研究队伍提供资金支持，同时将在瑞典建立一支公司的内部队伍。双方的科学家将协力探索糖尿病患者体内胰岛β细胞功能和质量衰退背后的生物学机制，同时将采用制备的胰岛β细胞来筛查化合物文库，寻找有望恢复胰岛β细胞活力的潜在新药。该项合作的财务条款尚未披露。

背后的感人故事

特别值得一提的是，这一合作的关键人物Douglas A. Melton教授背后有着非常感人的满满正能量的故事，他的儿子和女儿（现年24和28岁）均在幼年时期确诊1型糖尿病，而他本人最初的研究方向也并非糖尿病。为了救孩子，他大胆将实验室重组转向糖尿病研究，在2001年干细胞研究没有联邦资金支持的情况下，自己拉私募资金潜心研究多年于2008年取得重大技术突破，成为该领域的泰山北斗。就在最近，他创立的干细胞公司Semma Therapeutics成功融资4400万美元，并且获得了诺华的非公开协议。

英文原文：AstraZeneca to collaborate with the Harvard Stem Cell Institute in diabetes

Wednesday, 25 March 2015

AstraZeneca today announced that it has entered into a five-year research collaboration with the Harvard Stem Cell Institute (HSCI) to adapt a technique that creates human beta cells from stem cells for use in screens of AstraZeneca’s compound library in the search for new treatments for diabetes. The collaboration also aims to better understand how the function of beta cells declines in diabetes and research findings will be made available to the broader scientific community through peer-reviewed publications.

In people with Type 1 diabetes, beta cells are destroyed by an autoimmune response and patients must inject insulin to maintain normal blood glucose levels. In Type 2 diabetes, the beta cells either fail to function properly or their numbers decrease. Human beta cells for research are extremely limited in number and availability. However, a team led by HSCI co-chairman and Howard Hughes Medical Institute Investigator, Professor Doug Melton, has developed a technique which allows limitless quantities of beta cells to be produced from human induced pluripotent stem cells generated directly from adult cells, similar in all important respects to those found in healthy individuals1.

AstraZeneca will provide funding for a team of investigators at HSCI lead by Professor Melton as well as establishing an in-house team in Mölndal, Sweden, dedicated to the collaboration. Scientists from each organisation will work together to understand the biology behind the loss of human beta cell function and mass in diabetes, and to screen compounds against the cells produced to search for potential new medicines that could restore beta cell activity in diabetic patients.

Marcus Schindler, Head of Cardiovascular and Metabolic Diseases, Innovative Medicines and Early Development, AstraZeneca, said: “We are excited about the potential of this latest collaboration with Harvard University. Professor Melton’s group has made an extraordinary breakthrough in the differentiation of human stem cells into human beta cells and our scientists are extremely excited to be working alongside his team. Harnessing this new technology has the potential to transform the research and development of new treatments for patients with diabetes.”

Isaac T. Kohlberg, Head of the Office of Technology Development at Harvard University, said: “AstraZeneca’s commitment to establish and fund this collaboration will help advance the development of new medicines that may ameliorate the need for diabetics to inject insulin, and prevent the numerous, potentially fatal complications of diabetes. This collaboration is an ideal example of how academia and industry should work together to serve the public interest and make a difference in the lives of patients.”

The collaboration is aligned with AstraZeneca’s strategic research approach in diabetes which is aimed at restoring the function of the pancreatic beta cells as well as insulin sensitivity, irrespective of therapeutic modality.

NOTES TO EDITORS

About the Department of Stem Cell and Regenerative Biology and Harvard Stem Cell Institute (HSCI)

The Department of Stem Cell and Regenerative Biology at Harvard University and HSCI advance the understanding of human development and disease, support the discovery of stem cell-based therapies and cures for diseases, create collaborations across traditional institutional and disciplinary boundaries, and teach and train the next generation of leading stem cell scientists.

About Harvard University’s Office of Technology Development

The Harvard Office of Technology Development (OTD) is responsible for all activities pertaining to the evaluation, patenting and licensing of new inventions and discoveries made at Harvard University and Harvard Medical School. OTD also serves to further the development of Harvard technologies through the establishment of sponsored research collaborations with industry. OTD's mission is to promote the public good by fostering innovation and translating new inventions made at Harvard into useful products available and beneficial to society.