Emory Conducting Landmark Study to Treat ALS

Emory University researchers are participating in a groundbreaking clinical trial to treat patients with Amyotrophic Lateral Sclerosis (ALS) using human neural stem cells.

The Phase 1 trial, approved in 2009 by the U.S. Food and Drug Administration, is studying the safety of stem cells, and the surgical procedures and devices required, for multiple injections of the cells directly into the spinal cord.

“This is the first U.S. clinical trial of stem cell injections into the spinal cord for the treatment of ALS," says Jonathan Glass, MD, professor of neurology, Emory School of Medicine, and director of the Emory ALS Center and principal investigator (PI) of the clinical trial site.

“Our main goal in this early phase is to determine whether it is safe to inject stem cells into the spinal cord and whether the cells themselves are safe," says Glass.

Since the trial began in January, three patients with ALS have received injections. Up to 12 individuals will be enrolled in the first phase of the trial.

Nicholas Boulis, MD, assistant professor of neurosurgery at Emory School of Medicine, and a pioneer in developing surgical methods for delivery of therapeutics to the spinal cord, is performing the surgical procedures. Eva Feldman, MD, PhD, director of research at the University of Michigan Health System ALS Clinic, is the overall PI of the ALS clinical trials program.

“I am confident this study is taking therapies for the spinal cord to a new level,” says Boulis. “Depending on the success of this initial trial, there will be a follow-up phase II trial or a modified phase I trial that utilizes the techniques of surgical implementation.”

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.

The stem cells used in the study, developed by the Maryland-based biotech company, Neuralstem, Inc., were prepared from cultured neural stem cells and may have the ability to mature into various types of cells in the nervous system. This includes motor neurons, the ones that are specifically lost in ALS.

Scientists say these stem cells will not generate new motor neurons, but may help protect the still-functioning motor neurons and slow the progression of the disease.

“We will closely follow these patients and will be eager to see results,” says Glass. “We are hopeful this is the first step toward a new way of treating the people who face this devastating disease.”

Watch on an interview with Glass at http://www.youtube.com/watch?v=5tln2i19wUs.

The Robert W. Woodruff Health Sciences Center of Emory University is an academic health science and service center focused on missions of teaching, research, health care and public service. Its components include the Emory University School of Medicine, Nell Hodgson Woodruff School of Nursing, and Rollins School of Public Health; Yerkes National Primate Research Center; Winship Cancer Institute of Emory University; and Emory Healthcare, the largest, most comprehensive health system in Georgia. Emory Healthcare includes: The Emory Clinic, Emory-Children's Center, Emory University Hospital, Emory University Hospital Midtown, Wesley Woods Center, and Emory University Orthopaedics & Spine Hospital. The Woodruff Health Sciences Center has $2.3 billion in operating expenses, 18,000 employees, 2,500 full-time and 1,500 affiliated faculty, 4,500 students and trainees, and a $5.7 billion economic impact on metro Atlanta. Learn more about Emory’s health sciences: http://emoryhealthblog.com - @emoryhealthsci (Twitter) - http://emoryhealthsciences.org