Success of ALS clinical trial builds promise for neural stem cell therapies

Ann Arbor, Mich. — Patients with amyotrophic lateral sclerosis (ALS) who received cervical intraspinal injections of neural stem cells tolerated the procedure well and showed no signs of accelerated disease progression, a finding that lays critical groundwork for the use of neural stem cells in treating degenerative neurological disorders like ALS, or Lou Gehrig’s disease, and other neurodegenerative disorders.

Results of the first-of-its kind clinical trial led by researchers at the University of Michigan, “Intraspinal neural stem cell transplantation in amyotrophic lateral sclerosis: Phase 1 trial outcomes,” will be published in the March 2014 edition of Annals of Neurology.

The research team led by Eva Feldman, M.D., Ph.D., used a novel spinal-mounted stabilization and injection device to deliver up to 1.5 million neural stem cells to the cervical, or upper, spinal cord of six ALS patients in the study’s second cohort. The first cohort included 12 patients who received stem-cell injections in the lumbar, or lower, spinal cord. Three patients were in both cohorts and received both treatments.

The Phase 1 trial is the first ever to use human subjects to test the safety of intraspinal stem cell injections. A Phase 2 trial is under way at the University of Michigan, Emory University and Massachusetts General Hospital to determine how patients do with larger doses. In Phase 2, subjects will receive as many as 16 million stem cells in the 9-hour procedure.

“We are extremely excited by the findings of this clinical trial,” said Dr. Feldman, Director of the A. Alfred Taubman Medical Research Institute, Director of Research for the ALS Clinic at the University of Michigan Health System, principle investigator for the trial and lead author. “If we can show that injecting these stem cells can slow or even halt the progress of this devastating disease, we can begin to pursue further therapies and trials in the promising area of regenerative medicine.”

Researchers are hopeful that the lessons learned from the ALS stem-cell trial will help inform efforts to treat other serious neurological disorders, including Alzheimer’s disease and multiple sclerosis.

That concept is echoed in a commentary piece also to be published in the March 2014 edition of the Annals of Neurology titled “Cell therapy in amyotrophic lateral sclerosis: A significant step forward,” by Tamir Ben-Hur, M.D., Ph.D., of Hadassah University Hospital in Israel. In the article, Dr. Ben-Hur noted that the ALS study not only established a technical benchmark for stem-cell injections, but also breaks a critical psychological barrier to stem-cell therapies.

“The importance of this report … lies far beyond its immediate conclusions,” Dr. Ben-Hur wrote. “The special design of the present study sets new standards for further clinical translation in regenerative medicine for neurological diseases.”

The stem cells for Phase 1 and Phase 2 clinical trials were developed by Neuralstem, Inc., a company whose patented technology enables the production of neural stem cells of the brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glial cells. Neuralstem, Inc. is a publicly traded company based in Rockville, Md. Dr. Feldman is an unpaid consultant to Neuralstem, Inc.