Neuralstem, Inc. announced that the final patient was treated in its Phase II trial using NSI-566 spinal cord-derived neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
The multicenter Phase II trial treated 15 ambulatory patients in five different dosing cohorts. The first 12 patients received injections in the cervical region of the spinal cord only, where the stem cells could help preserve breathing function, in escalating doses ranging from five injections of 200,000 cells per injection, to 20 injections of 400,000 cells each. The final three patients in the trial received both cervical and lumbar injections, for a total of 40 injections of 400,000 cells each, or a total of 16 million cells transplanted. In contrast, the final three patients in the Phase I trial received the maximum 15 injections of 100,000 cells each, for a total of 1.5 million cells. The trial will continue until six months past the final surgery, at which point the data will be evaluated.
"We are all extremely pleased to have completed the transplantations in this historic Phase II trial," said principal investigator, Dr. Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "By early next year, we will have six-month follow up data on the last patients who received what we believe will be the maximum safe tolerated-dose for this therapy. We look forward to seeing what the data tell us about safety and efficacy of this approach. It is also worth noting that we will have completed this Phase II trial within a year, roughly. I would like to thank Dr. Parag Patil, and my collaborators at Emory, Drs. John Glass and Nick Boulis, and at Mass General, Drs. Merit Cudkowicz and Larry Borges, for helping us reach this goal." Dr. Feldman is an unpaid consultant to Neuralstem.
"The completion of Phase II of this important clinical research program is a major milestone, demonstrating that patients can tolerate the transplantation of high doses of cells and multiple spinal cord injections," said site principal investigator, Jonathan D. Glass, MD, Director of the Emory ALS Center. "From both a clinical and scientific perspective, I think we are now ready to move forward toward a true therapeutic trial to test the efficacy of this surgical approach for slowing the course of ALS."
"We would like to express our thanks to all of the doctors and medical staff who made this possible, as well as the patients and their families. Without their bravery, none of this would have happened," said Karl Johe, PhD, Neuralstem's Chairman of the Board and Chief Scientific Officer. "With this landmark trial, the first to transplant stem cells in this volume and through so many injections along the length of the human spinal cord, we hope to establish the dose that is both safe and which may be optimal for treatment. We are excited about the collection and analysis of the final data and look forward to advancing to our next trial."