布莱根总医院启动自体干细胞移植治疗帕金森病一期临床试验

布莱根总医院（massachusetts General Brigham）最近启动了一项一期临床试验，旨在检验一种治疗帕金森病的突破性方法的安全性和可行性。该方法通过对患者的干细胞进行重新编程，以取代被帕金森病损伤的大脑中的多巴胺细胞。

A recently launched Phase 1 clinical trial at Mass General Brigham is examining the safety and feasibility of a groundbreaking treatment approach for Parkinson's disease in which a patient's stem cells are reprogrammed to re-place dopamine cells in the brain damaged by the disease.

基于麦克林医院神经再生研究所（NRI）发明并经临床前验证的研究和技术，首例自体干细胞移植试验已在布里格姆妇女医院招募并治疗了三名患者。

The first-of-its-kind trial of an au-tologous stem cell transplant, based on re-search and technologies invented and vali-dated preclinically at McLean Hospital's Neuroregeneration Research Institute (NRI), has enrolled and treated three patients at Brigham and Women's Hospital.

总共有六名参与者将参与一期试验，该试验将对患者进行为期12个月或更长时间的跟踪，以确定该手术的安全性，并监测帕金森病的任何改善。

A total of six participants will be included in the Phase 1 trial that will track the patients for 12 months and beyond to determine the safety of the procedure and monitor for any improvements in Parkinson's disease.

这种治疗帕金森病的新方法包括使用从患者自身血液中的细胞，转化为诱导多能干细胞（iPSCs）。

This novel therapeutic approach for treating Parkinson's disease incorporates the use of stem cells derived from a patient's own blood that had been converted into induced pluripotent stem cells (iPSCs).

然后，这些细胞被重新编程，变成特定的中脑多巴胺能神经元，用于移植。

These cells are then repro-grammed to turn into specific midbrain dopaminergic neu-rons ready for transplantation.

使用自身细胞的自体移植方法避开了免疫抑制治疗的要求，当使用其他供者的细胞时，免疫抑制治疗是必要的。

The autologous transplan-tation approach of using a person's own cells circumvents the requirement for immunosuppressive treatments, which are necessary when cells from other donors are used.

NRI的创始主任Ole Isacson，医学博士，同时也是哈佛医学院和麻省总医院Brigham的神经学教授，在过去的30年里开创了帕金森病细胞疗法的工作，并为这项临床试验奠定了基础。

The NRI's founding director, Ole Isacson, Dr Med Sci, who is also a professor of neurology (neuro-science) at Harvard Medical School and Mass General Brigham, has pioneered work in cell therapy for Parkinson's disease over the past 30 years and laid the foundation for this clinical trial.

2015年，由Isacson和Penny Hallett博士（麦克莱恩研究所NRI联合主任、哈佛医学院精神病学副教授）领导的NRI团队，在高度相关的帕金森病非人类灵长类动物模型中，首次提供了自体干细胞治疗长期安全性和益处的证据。

In 2015, the NRI team, led by Isacson and Penny Hallett, PhD, co-director of the NRI at McLean and associate professor of psychiatry at Harvard Medical School, provided the first evidence of long-term safety and benefits of autologous stem cell ther-apy in a highly relevant Parkinson's disease non-human primate animal model.

NRI于2023年8月23日获得了美国食品和药物管理局（FDA）的批准，进行1期临床试验申请。

The NRI received official authorization from the U.S. Food and Drug Administration (FDA) on August 23, 2023, ap-proving its Investigational New Drug (IND) application for a phase 1 clinical trial to test this unique, autologous dopamine neuron cell therapy.