Phase II of ALS stem cell trial underway at Emory and University of Michigan

Woodruff Health Sciences Center | Sep. 30, 2013

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Janet Christenbury
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jmchris@emory.edu

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Illustration of neurons functioning normally.

Amyotrophic lateral sclerosis (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.

Researchers at Emory University and University of Michigan have begun the Phase II trial of a landmark study to treat patients with Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease) using human neural stem cells. The Phase II dose escalation and safety trial is designed to treat up to 15 patients in five different dosing cohorts.

"We are excited to begin the next phase of this neural stem cell trial, further evaluating safety precautions, while increasing both the number of injections and cells transplanted in ALS patients," says Jonathan Glass, MD, professor of neurology, Emory School of Medicine and director of the Emory ALS Center. Glass also serves as principal investigator of the Emory clinical trial site.

This is the first U.S. clinical trial of stem cell injections into the spinal cord for the treatment of ALS. The study is jointly funded by a $2 million grant from the National Institutes of Health and by the Maryland-based biotech company, Neuralstem, Inc., which also provides the human neural stem cells for transplantation.

The Phase I trial, conducted exclusively at Emory University, was designed to assess the safety of implanting neural stem cells into the spinal cord in up to 18 ALS patients. The first 12 patients received neural stem cell transplants in the lumbar, or lower, region of the spinal cord. Six more patients were transplanted in the cervical, or upper, region of the spinal cord, after the FDA reviewed safety data from the first 12 patients.

"In the Phase II trial, the first 12 patients will receive injections in the cervical spinal cord only, the region that may help in preserving breathing function," says Glass, who is a longtime ALS researcher and clinician. "The final three patients will receive both cervical and lumbar injections. The dose will also increase in both number of injections and cells per injection throughout the trial."

In Phase I, the trial progressed to a maximum of 15 injections of 100,000 cells each. In Phase II, the trial will advance up to a maximum of 40 injections and up to 400,000 cells per injection. 

Nicholas Boulis, MD, associate professor of neurosurgery at Emory School of Medicine, performs the surgery by exposing the spinal cord 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 the U.S. Patent and Trademark Office in 2011.

"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. "The Phase II trial will help us better understand if more injections with more cells can improve conditions for the patient in a safe manner." 

A skilled neurosurgeon, Boulis is teaching other neurosurgeons involved in this trial the proper procedures for implanting human neural stem cells. 

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. 

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.  

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Boulis is the inventor of technology related to this study. He receives licensing fees from Neuralstem, the licensor of the technology and co-sponsor of the study. Boulis is also a paid consultant for Neuralstem, and helps organize clinical trials in Korea and Mexico. The terms of this arrangement have been reviewed and approved by Emory University in accordance with its conflict of interest policies.