Yazarlar : Glass JD, Boulis NM, Johe K,
Yayın : Stem Cells
Yayın Yılı : 2012
Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/22415942
Konu : Rejeneratif Tıp
Literatür İçeriği :
Abstract
Advances in stem cell biology have generated intense interest in the prospect of transplanting stem cells into the nervous system for the treatment of neurodegenerative diseases. Here we report the results of an ongoing Phase I trial of intraspinal injections of fetal-derived neural stems cells in patients with Amyotrophic Lateral Sclerosis (ALS). This is a first-in-human clinical trial with the goal of assessing the safety and tolerability of the surgical procedure, the introduction of stem cells into the spinal cord, and the use of immunosuppressant drugs in this patient population. Twelve patients received either 5 unilateral or 5 bilateral (10 total) injections into the lumbar spinal cord at a dose of100,000 cells/injection. All patients tolerated the treatment without any long-term complications related to either the surgical procedure or the implantation of stem cells. Clinical assessments ranging from 6 to18 months after transplantation demonstrated no evidence of acceleration of disease progression due to the intervention. One patient has shown improvement in his clinical status, though these data must be interpreted with caution since this trial was neither designed nor powered to measure treatment efficacy. These results allow us to report success in achieving the Phase I goal of demonstrating safety of this therapeutic approach. Based on these positive results we can now advance this trial by testing intraspinal injections into the cervical spinal cord, with the goal of protecting motor neuron pools affecting respiratory function, which may prolong life for patients with ALS.
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