Two patients with amyotrophic lateral sclerosis (ALS) have received stem cell injections to their spinal cords at the University of Michigan Health System. They are the first to receive the injections in what is part of a national clinical trial. The Phase II trial is approved by the U.S. Food and Drug Administration and funded by Neuralstem, Inc., the Maryland-based company whose stem-cell product the trial is testing. The study seeks to discover any effect that injected stem cells might have on motor neurons—muscle-controlling nerve cells that die in ALS patients.
In a Phase l trial conducted at Emory University, Atlanta, Georgia, researchers safely delivered spinal cord injections of up to 100, 000 cells. The researchers reported possible signs that in one subgroup of participants, ALS progression may have been interrupted.
“We’re going to be permitted to give more injections and more stem cells in Phase 2, ” said Eva L. Feldman, M.D., Ph.D., director of the study. “We’re very excited that we have been able to bring this important work to the University of Michigan.”
The Phase II dose escalation trial is designed to treat up to 15 ambulatory patients in five different dosing cohorts, under an accelerated dosing and treatment schedule. The patient’s spinal column is unroofed and the spinal cord exposed to receive the cells.
The first 12 patients, divided into four cohorts, will receive injections only in the cervical region of the spinal cord, where breathing function is controlled. The first cohort of three patients will receive 10 cervical region injections of 200, 000 cells per injection. The trial will then progress to a maximum of 20 cervical injections of up to 400, 000 cells per injection.
The last three Phase II patients will receive injections in both the cervical and the lumbar spinal regions. These patients will receive 20 injections of 400, 000 cells each in the lumbar region in addition to the 20 injections they will already have received in their cervical region.
The trial accelerates the treatment schedule, and is designed to progress at the rate of one cohort per month with one month observations periods between cohorts. Researchers expect all of the patients will be treated by the end of the second quarter in 2014.
