Neuralstem has announced that Jonathan D Glass, site investigator at Emory University, presented long-term follow up data on the phase I trial testing NSI-566 human neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS). The presentation, which occurred at the Annual Symposium on ALS of the Foundation Andre-Delambre, in Montreal, Canada, and was not open to the public, covered data up to approximately 1200 days post the stem cell treatment.
Glass reported that patients in the last safety cohort (n=3), who received treatments in both the lumbar and the cervical region with the highest number of cells per injection, all showed significant slowing of the progression of the disease. One patient showed functional improvement from pre-treatment baseline, which is maintained to present day. The other two patients are maintaining the same level of functionality as they had at the baseline for over three years since the stem cell treatment.
“The long-term follow up data is very encouraging,” says Karl Johe, Neuralstem’s chairman and chief scientific officer. “In phase I, patients 10, 11, and 12 each received 10 lumbar and five cervical injections, of 100,000 cells each, which was far below the safe maximal dose. Even so, the data shows a significant slowing of the disease progression for over three years. If replicated on a larger scale, this could represent meaningful improvement in quality of life, and lifespan, compared to untreated patients. In our phase II dose escalation trial, we successfully reached the maximal dose planned, which consisted of 20 lumbar and 20 cervical injections of 400,000 cells each, more than ten times the number of stem cells delivered in the highest dose cohort of the phase I trial.”
“The progress in this trial is truly groundbreaking,” says Glass, who is director of the Emory ALS Center at Emory University, the first site in the trial. “It has provided data on the safety of multiple injections and multiple transplantation surgeries in ALS patients, as well as the long- term survival of the transplanted cells in the human spinal cord. This provides a strong foundation for moving ahead with more definitive trials focused on the potential therapeutic efficacy of spinal cord transplantation of neural stem cells for ALS.”