臍帶血治療遺傳性運動失調症
中國廣東醫學大學附設醫院、中國頂尖幹細胞生技公司-Bieke與美國聖地牙哥生技公司-Medistem日前發表合作成果，首次發現利用臍帶血幹細胞與物理治療的合併療法，可顯著改善「遺傳性運動失調症」(Hereditary Ataxia)病患。這項研究已刊登在「轉錄醫學」期刊中。
「遺傳性運動失調症」是一種先天性的神經退化症，包括小腦、腦幹、脊髓的神經組織出現萎縮，導致運動神經受損，說話、行走、吞嚥等運動功能有障礙，影響生活機能與品質，目前沒有有效的治療方式。
此項臨床試驗募集30位病患，經由靜脈注射或腦部穿刺，接受4~6次臍帶血幹細胞移植，每2天再接受30分鐘物理治療，整個治療過程中，明顯看見病患的運動功能獲得改善。
接下來的試驗，將會近一步評估移植後的有效性、安全性、和持久性，但此初步結果，已帶給病患一大希望。
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.