脐带血治疗遗传性运动失调症
中国广东医学大学附设医院、中国顶尖干细胞生技公司-Bieke与美国圣地牙哥生技公司-Medistem日前发表合作成果，首次发现利用脐带血干细胞与物理治疗的合并疗法，可显著改善「遗传性运动失调症」(Hereditary Ataxia)病患。这项研究已刊登在「转录医学」期刊中。
「遗传性运动失调症」是一种先天性的神经退化症，包括小脑、脑干、脊髓的神经组织出现萎缩，导致运动神经受损，说话、行走、吞咽等运动功能有障碍，影响生活机能与品质，目前没有有效的治疗方式。
此项临床试验募集30位病患，经由静脉注射或脑部穿刺，接受4~6次脐带血干细胞移植，每2天再接受30分钟物理治疗，整个治疗过程中，明显看见病患的运动功能获得改善。
接下来的试验，将会近一步评估移植后的有效性、安全性、和持久性，但此初步结果，已带给病患一大希望。
New Study Finds Cord Blood-derived Stem Cell Treatments Improve Ataxia Patients' Functionality and Quality of Life
The Journal of Translational Medicine reports on a new study in which 30 patients with Hereditary Ataxia showed statistically significant improvements in functionality and quality of life after being treated with a combination of cord blood-derived stem cell treatments and physical therapy.
The study was conducted at the Nanshan Affiliated Hospital of Guangdong Medical College in China, and included the participation of Beike Biotechnology Company, China's leading stem cell research and regenerative medicine company, and the San Diego biotech company Medistem, Inc.The peer-reviewed article is available at: http://www.translational-medicine.com/content/9/1/65 .
Dr. Shengqin Ye, CEO of Beike Biotechnology commented, "This is the first published study of the effects of adult stem cell treatments on patients with Hereditary Ataxia. The results should be enormously encouraging for those who suffer from this difficult condition. With the combination of stem cells and physical therapy, study participants showed significant and sustained improvement. We are cautiously optimistic that further study will validate the efficacy and safety conclusions we have drawn from the study data."
Hereditary Ataxia is a neurodegenerative disorder characterized by atrophy of the cerebellum, brain stem, or spinal cord. Manifestations of the condition include a variety of functional impairments relating to speech, walking, swallowing, and other physical activities. Currently there is no cure for this condition.
Over the course of the study, participants received 4-6 stem cell treatments via intravenous injection or by cervical or lumbar puncture, and underwent twice-daily 30-minute physical therapy sessions. The study concluded, "In addition to the early effect seen in some subjects, the measured symptomatic improvements persisted throughout the period of the study, as noted with the follow-up data from a subset of subjects." Study authors recommend further double blind placebo controlled studies to validate the efficacy, safety and long-term effects of the treatments.