Emory University researchers have received approval from the Food and Drug Administration (FDA) to advance to the next phase of a landmark trial to treat patients with Amyotrophic Lateral Sclerosis (ALS) using human neural stem cells.
The Phase I trial, currently underway exclusively at Emory University, is designed to assess the safety of implanting neural stem cells into the spinal cord in up to 18 people with ALS and began in January 2010. The first 12 patients received neural stem cell transplants in the lumbar, or lower, region of the spinal cord. After reviewing safety data from these patients, the FDA has granted approval for the trial to advance to the final two groups of patients (three in each group), all of who will be transplanted in the cervical, or upper, region of the spinal cord.
“This represents a major accomplishment for the trial, meaning that we have achieved our stated goal of proving safety in the first 12 patients who received lumbar spinal injections,” says Jonathan Glass, MD, Professor of Neurology, Emory School of Medicine and director of the Emory ALS Center.
“Our next objective is to demonstrate that we can deliver the cells safely to the cervical spinal cord, which is particularly important because therapy in this region may help patients better maintain their ability to breathe.”
Nicholas Boulis, MD, Associate Professor of Neurosurgery at Emory School of Medicine, performs the surgery to implant the cells. He also developed the device used to inject the stem cells into the spinal cord, which received a notice of patent allowance from U.S. Patent and Trademark Office in October.
“We hope this trial continues to foster the development of both new techniques for surgical implementation and treatments for people who are living with ALS,” says Boulis. “We are still early in this trial; however, these initial results allow us to move forward with optimism.”
Also known as Lou Gehrig’s disease, ALS is a fatal neurodegenerative disease with no known cure. It causes the deterioration of specific nerve cells in the brain and spinal cord called motor neurons, which control muscle movement. As the illness progresses, patients lose their ability to walk, talk and breathe. According to the ALS Association, approximately 30,000 Americans have ALS at any given time and patients with the disease usually die within two to five years of diagnosis.
This is the first U.S. clinical trial of stem cell injections into the spinal cord for the treatment of ALS. The study is funded by the Maryland-based biotech company, Neuralstem, Inc., which is also providing the human neural stem cells for transplantation. Neuralstem’s cells have the ability to mature into various types of cells in the nervous system, including the motor neurons that are specifically lost in ALS. However, scientists say the goal of stem cell transplantation is not to generate new motor neurons, but to protect the still-functioning motor neurons by nurturing them with the stem cells, potentially slowing the progression of the disease.