An interim report of data from an ongoing open-label trial supports the safety of intraspinal cord injection of stem cells but suggests that dose-limiting toxicity may have been reached.
Intra–spinal cord injection of neural stem cells derived from human spinal cord is one of many stem cell–based approaches being investigated to treat amyotrophic lateral sclerosis (ALS). Researchers now report 9-month safety data from an ongoing, partially manufacturer-funded (Neuralstem, Inc.) phase II study (n=15), along with data from a previously reported phase I study (n=9). Participants received one of three escalating doses (cells/injection) and numbers of injections in the cervical and lumbar spinal cord.
Most participants had uneventful surgical and postoperative courses. Two of the six patients in the highest dosing group (20 injections of 800,000 cells/injection, spread across both sides of the lumbar and cervical cord) had severe…
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DisclosuresGrant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense
Editorial boardsCochrane Collaboration
Leadership positions in professional societiesMuscle Study Group Executive Committee
DisclosuresGrant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense
Editorial boardsCochrane Collaboration
Leadership positions in professional societiesMuscle Study Group Executive Committee