The Burrill Report

The U.S. Food and Drug Administration has given Neuralstem approval to begin an early-stage human clinical trial of its lead cell therapy candidate in chronic spinal cord injury patients. The trial is believed to be the first stem cell trial to treat this group of patients with such a therapy. This open-label, multi-site study will enroll up to eight patients with thoracic spinal cord injuries that are completely paralyzed with no motor or sensory function at and below their injury after two years. The study will seek to determine the safety and toxicity of transplantation of NSI-566, the company’s human spinal stem cells. The secondary objectives of the study are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression. The study will look at exploratory objectives to evaluate the ability of human spinal cord stem cell transplantation to positively affect motor and sensory ability, bowel and bladder function, pain, and other measures. Patients in the study will receive six injections in, or around, the injury site. The first four patients will receive 100,000 cells per injection, the second four patients will receive 200,000 cells per injection. All patients will also receive physical therapy post surgery, as well as immunosuppressive therapy for three months as tolerated. The trial study period will end six months post-surgery for each patient. “This is another critical milestone for the company," says Richard Garr, Neuralstem president and CEO. “We believe the FDA approval of a trial for a second indication for our NSI-566 cells demonstrates an increasing level of comfort at the FDA with our technology and acknowledges the positive strides being made in the ALS trial.” Neuralstem says it has conducted 18 successful surgeries using the same cells and surgical device in an early-stage trial for patients with the neurodegenerative disease amyotrophic lateral sclerosis, or ALS. That trial, the company says, demonstrated that the surgical route of administration and the cells are safe and well tolerated and that the cells survive long-term in the patients. The company hopes to address multiple indications with NSI-566. It plans to begin dosing patients with NSI-566 to treat paralysis from stroke in the next month in China, and it is preparing for a trial of NSI-566 to treat acute spinal cord injury patients in Korea this summer. It is also in discussions with the FDA over the design and scope of a mid-stage clinical trial for ALS with NSI-566.

January 18, 2013
http://www.burrillreport.com/article-fda_green_lights_neuralstem_trial.html