Researchers completed the first American clinical trial involving stem cell-based treatment of Amyotrophic Lateral Sclerosis (ALS). They were encouraged by the trial study's results, noting that this delivery approach could be a helpful therapeutic approach for other traumatic spine-related problems.
The group focused on the safety of a direct microinjection-based technique and neural stem cell transplantation to the cervical and thoracolumbar spinal cord. Eighteen microinjection procedures delivered NSI-566RSC, a human neural stem cell, to a total of 15 patients in five cohorts. Each of the injection procedures consisted of five injections of 10Âµl at 4mm intervals. Group A (n=6) was non-ambulatory and received unilateral (n=3) or bilateral (n=3) thoracolumbar microinjection. Groups B through E were ambulatory and received unilateral (group B, n=3) or bilateral (group C,n=3) bilateral thoracolumbar microinjection. Groups D and E received unilateral cervical (group D,n=3) or cervical plus bilateral thoracolumbar microinjection (group E,n=3). Detailed pre- and post-operative neurological outcomes were recorded, such as post-operative pain, as well as urologic, sensory and motor functions. The results of this study, Intraspinal Stem Cell Transplantation in ALS, A Phase I Trial: Cervical Microinjection Safety Outcomes, will be presented by Jonathan Patrick Riley, MD, from 10:24-10:35 a.m. on Monday, April 29. Co-authors are Jonathan Glass, MD, PhD; Karl Johe, PhD; Meraida Polak, RN; Thais Federici, PhD; Eva Feldman, MD, PhD; and Nicholas Boulis, MD, FAANS.
"We are excited that the safety results of this trial have borne out what has been shown by our preclinical studies - that both the cervical and thoracolumbar spinal cord are able to safely tolerate multiple targeted injections of a cellular graft," said Jonathan Patrick Riley, MD. "These results support the exploration of the cellular graft "dose range" that may be delivered and safely tolerated. Identification of a safe dose range that the spinal cord tolerates is an important first step prior to completion of Phase II efficacy studies."