Neuralstem, a company that specializes in producing commercial quantities of neural stem cells of the brain and spinal cord, publicly presented the findings of their Phase 1 clinical trial involving amyotrophic lateral sclerosis (ALS) at the Keystone Symposia “Engineering Cell Fate and Function,” occurring April 6-11 in Olympic Valley, California. Results were published in Annals of Neurology in mid-March, but principal investigator Eva Feldman, PhD, MD, discussed the results of Neuralstem’s NSI-566 stem cell trial in ALS during a workshop on “Clinical Progress for Stem Cell Therapies” at the symposia.
The Phase 1 trial is the first of its kind to use intraspinal stem cell transplantation. Beginning in January 2009, patients were assessed for the safety of human spinal cord-derived neural stem cell transplantation, as well as for improved muscle function and reduced pain. “Although this was a Phase I trial, and functional outcome data were collected for the purpose of assessing safety, we performed secondary analyses of these data as a means to gain insight into how cellular transplantation affected disease progression rates and to inform outcome assessment approaches in future trial phases,” said Dr. Feldman. These secondary analyses revealed potential therapeutic windows that suggest benefits of more and multiple injections during treatment, which is consistent with the pre-conceived hypothesis for a neuroprotective mechanism-of-action for the therapy.
In addition to the results of the Phase 1 trial, Dr. Feldman also updated the session attendees on the progress of Neuralstem’s Phase 2 clinical trial to assess the safety of transplanting a maximum tolerated dose of human spinal cord-derived stem cells into the spinal cords of patients with ALS. So far, since the May 2013 study start date, 11 patients have been enrolled in the trial. There are five sequential cohorts, with each cohort following a dose escalation plan for spinal cord injections of human-derived spinal cord stem cells. Patients will be assessed for motor function, respiratory capacity, and graft stability. The anticipated study complete date in this month.
Neuralstem is also investigating the use of its NSI-566 spinal cord-derived stem cell therapy in spinal cord injury and ischemic stroke.