神經(jīng)干細(xì)胞移植在極罕見腦病中顯現(xiàn)治療潛力
StemCells 公司今天公布的數(shù)據(jù)顯示，移植了該公司專有的HuCNS-SC（純化的人類神經(jīng)干細(xì)胞）后的佩 - 梅病（Pelizaeus- Merzbacher disease，PMD）患者， 經(jīng)磁共振成像（MRI）表明，移植2年后，髓鞘的形成比移植1年后更加明顯，同時，患者移植1年后所獲得的神經(jīng)功能臨床收 益，在2年后仍能繼續(xù)維持，并且未出現(xiàn)安全性問題。
佩-梅病（Pelizaeus-Merzbacherdisease，PMD）是一種罕見的彌漫性腦白質(zhì)髓鞘形成障礙的X連鎖隱性遺傳疾病，由于基因缺陷，導(dǎo)致大腦中髓磷脂不足，最終導(dǎo)致神經(jīng)功能的逐漸喪失和死亡。
StemCells公司從事基于干細(xì)胞療法和工具的研究、 開發(fā)和商品化，用于基于干細(xì)胞的研究和藥物發(fā)現(xiàn)。該公司目前開發(fā)的先導(dǎo)治療候選產(chǎn)品 HuCNS-SC（ 純化的人類神經(jīng)干細(xì)胞 ），有望用于廣泛的中樞神經(jīng)系統(tǒng)紊亂疾病的治療。
英文原文：Stem cell transplants show potential in patients with extremely rare brain disease
NEWARK, Calif., Aug. 2, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM) today presented data which show that two years after transplantation of the Company's proprietary HuCNS-SC? cells (purified human neural stem cells) into patients with Pelizaeus-Merzbacher disease (PMD), the evidence of myelination, by magnetic resonance imaging (MRI), is more pronounced compared to one year post-transplantation, the gains in neurological function reported after one year were maintained, and there were no safety concerns. Patients with PMD have a defective gene which leads to insufficient myelin in the brain, resulting in progressive loss of neurological function and death. The neurological and MRI changes suggest a departure from the natural history of the disease and may represent signals of a clinical effect. The data was presented today by Stephen Huhn, MD, FACS, FAAP, Vice President, CNS Clinical Research at StemCells, Inc., at the 2013 Pelizaeus-Merzbacher Disease Symposium and Health Fair being held at Nemours/Alfred I. duPont Children's Hospital in Wilmington, Del.
"We are encouraged that the MRI data continue to indicate new and durable myelination related to the transplanted cells and that the data is even stronger after two years compared to one year," said Dr. Huhn. "Even in the context of a small open-label study, these MRI results, measured at time points long after transplantation, make an even more convincing case that the HuCNS-SC cells are biologically active and that their effect is measureable, sustainable and progressive. Our challenge now is to reach agreement with the FDA on how best to correlate changes in MRI with meaningful clinical benefit, as this will be a critical step in determining a viable registration pathway for PMD."
In the Company's Phase I trial, which was conducted at the University of California, San Francisco, four patients with connatal PMD, the most severe form of the disease, were enrolled and transplanted with HuCNS-SC cells. The patients were followed for twelve months after transplantation, during which time they underwent intensive neurological assessments and magnetic resonance imaging at regular intervals. The Phase I trial results indicate a favorable safety profile for the HuCNS-SC cells and the transplantation procedure. Analysis of the MRI data showed changes consistent with increased myelination in the region of the transplantation, which progressed over time and persisted after the withdrawal of immunosuppression at nine months. The results support the conclusion of durable cell engraftment and donor cell-derived myelin in the transplanted patients' brains. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. The Phase I trial results were published in October 2012 in Science Translational Medicine, the peer review journal of the American Association for the Advancement of Science. Upon completion of the Phase I trial, all four patients were enrolled into a long-term follow up study, which is designed to follow the patients for four more years.
The Pelizaeus-Merzbacher Disease Symposium and Health Fair is intended to help educate families and healthcare professionals about PMD and related disorders. Sponsors include The PMD Foundation, a non-profit organization formed to raise awareness of the disease, create support for people with PMD, reduce misdiagnosis of PMD, and fund scientific research, and Nemours Biomedical Research, a branch of the Nemours Foundation, which is dedicated to improving the health and health care of all children.
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