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量产干细胞,糖尿病将迎来个体化疗法


2018-05-15 22:46:26      来源:药明康德

近期,美国波士顿地区的几家医院联手哈佛大学干细胞研究所、生物技术公司Semma Therapeutics,正筹划组织基于个体化细胞疗法的新型糖尿病临床试验。此次强强合作的研究计划名为“波士顿自体胰岛更换计划”(Boston Autologous Islet Replacement Program),是美国国内几个旨在进行大规模干细胞疗法的倡议性项目之一,致力于逐步实现这类治疗性细胞的规模化和工业化。


量产干细胞,糖尿病将迎来个体化疗法


在1型糖尿病患者的体内,免疫系统会攻击并破坏产生胰岛素的胰岛β细胞。多年来,医生们通过将已去世捐赠者的正常β细胞移植到病人体内,以恢复患者体内的胰岛素水平。然而,目前的这一方案能够提供的帮助很有限,无法满足全球数以千万计的1型糖尿病患者。不仅如此,患者的免疫系统有时还会排斥这些外来的胰岛β细胞。因此,我们需要开发出新的手段,以满足这一巨大的治疗需求。

根据“波士顿自体胰岛更换计划”,研究人员将采集患者的血液细胞,然后把它们诱导成为多潜能干细胞(iPSC),再使其再分化成为健康的胰岛β细胞,并将其批量扩增。最后,患者将接受这些胰岛β细胞的移植手术,并在术后接受效果评估和后续观察。由于这些胰岛β细胞来源于患者自身的干细胞,出现免疫排斥的风险就被大大降低了。

iPS细胞能分化成多种不同的体细胞,包括了胰岛β细胞(图片来源:Sigmaaldrich)

该项创新疗法来源于哈佛大学干细胞研究所共同负责人Douglas Melton博士几十年来在干细胞领域的学术研究,他领导的研究团队率先提出并实践了把干细胞分化成β细胞的想法,为这项创新疗法奠定了基础。

这其中的第一项临床试验将在三年内启动。研究人员首先选择了那些因胰腺炎或其他疾病摘除了胰腺的患者作为移植对象。如果该疗法被证明是安全有效的话,研究人员将继续把该疗法拓展到糖尿病患者群体中去。

该研究项目的领导者之一、来自哈佛大学的Richard Lee教授表示:“我们正处于一个技术转化的历史转折点,我们可以实实在在地开始考虑,如何应用干细胞移植技术来治疗糖尿病患者了。"



Stem cell factory opens door for trials of personalized diabetes treatment
     In a step that could lead to a new diabetes treatment, several Boston-area hospitals have teamed up with the Harvard Stem Cell Institute and biotech Semma Therapeutics to make personalized cell-based therapies and organize clinical trials.It is one of several initiatives around the country aimed at manufacturing cell-derived treatments, as the hope for such therapies creates a demand for the production of the cells.In patients with type 1 diabetes, the immune system attacks the pancreatic cells that produce insulin, called beta cells. Doctors have for years transplanted beta cells from dead donors into patients, but the supply is insufficient for the millions of people with type 1 diabetes, and patients’ bodies sometimes reject the donor cells.

The new program, officially called the Boston Autologous Islet Replacement Program, could provide enough high-quality beta cells needed to treat patients in upcoming clinical trials. And the cells that will be transplanted into these volunteers will be derived from their own blood cells, cutting the risk of rejection. For the first trial, which won’t start for at least three years, researchers intend to transplant the cells into people who have had their pancreas removed because of pancreatitis or another condition. If the treatment proves effective and safe, doctors could conduct another study with diabetic patients.

As part of the collaboration, clinicians at Brigham and Women’s Hospital and the Joslin Diabetes Center will recruit patients. Experts at the Dana-Farber Cancer Institute will derive the stem cells from the patients’ own blood cells — creating what are called induced pluripotent stem cells — and coax them into becoming beta cells. Doctors at the Brigham will then perform the transplant, with experts there and at Joslin monitoring the patients and their progress.

The program relies on research conducted by Douglas Melton, codirector of the Harvard Stem Cell Institute, who pioneered the idea of converting stem cells into beta cells. Melton is also the scientific founder of Semma.5h“We’ve reached a point with the technology where we can legitimately start thinking about cell replacement,” said Dr. Richard Lee, a Brigham cardiologist and researcher at the stem cell institute who helped wrangle the different institutions to form the program.

' L The Harvard collaboration represents just one cell production effort as researchers and patients eagerly try to move treatments derived from induced pluripotent stem cells, or iPS cells, into clinical trials — and to get them to patients. In addition to offering a patient-specific treatment, iPS cells avoid the ethical issues that come with conducting research or developing therapies with embryonic stem cells.

The National Cell Manufacturing Consortium, for example, on Monday announced its own plan for manufacturing a variety of cells for treatments, including iPS cells. The consortium is an initiative led by the Georgia Research Alliance and Georgia Institute of Technology and includes academic institutions, companies, and government agencies.)

Organizers of the Boston program still need to secure more funding if it is going to be successful. The stem cell institute has committed to funding the program for the first year, but Lee and other Harvard officials would not say how much the institute is contributing, or how much they think the program will cost to sustain.5z “There’s a need, but there’s a big risk when you set up one of these,” said Dr. Mahendra Rao, the vice president for regenerative medicine at the New York Stem Cell Foundation, who is not involved with the Harvard project.

Maintaining a cell production facility that meets manufacturing standards can alone be very expensive, Rao said. What helps in the Harvard case is that Dana-Farber has a history of producing cell transplant therapies to treat certain cancers, so it meets what are known as “current good manufacturing practices” for these types of therapies.

But developing the treatments for the patients in the trials and the trials themselves will take more funding than the program has so far identified. “It’s going to be very expensive,” Lee said. “The big challenge is, can the strategy of using an individual’s stem cells come down to a price that’s really feasible?”

The effort to bring iPS cell treatments to patients has been moving forward since the cells were discovered a decade ago, but it hasn’t always been a smooth road.
"  In 2014, a patient in Japan with macular degeneration became the first person treated with a treatment derived from iPS cells. That study was later put on hold because of problems with the cells meant for a second patient, although Japanese media reported recently that researchers are ready to restart the work.Also in 2014, Rao resigned as the director of the National Institutes of Health’s Center for Regenerative M
转自原文:https://www.statnews.com/2016/06/16/stem-cell-diabetes-treatment/


利用iPSC替代传统上的胚胎多能干细胞,是人类科学史上的一项伟大进步。它不会产生伦理方面的约束,因此创造出多样性的个体化治疗方案和途径。我们衷心期望“波士顿自体胰岛更换计划”顺利进行,为全世界范围内的糖尿病患者早日开辟切实可行的干细胞移植疗法。

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