The ALS Association

ALS Ice Bucket Challenge Progress

Research Advances: New Study Investigates Whether Seizure Medication Has Benefits for People with ALS

Retigabine, a drug now used to treat seizures is being studied for potential benefits to people with ALS.

“This novel study will provide us with a better understanding of neuron hyperexcitability, a potentially important disease mechanism in ALS patients,” said Lucie Bruijn, Ph.D. MBA, Chief Scientist for The ALS Association. “This powerful collaboration of leaders in the fields of stem cells, clinical neurology, ALS research and industry will be the first time that lab data from patient-derived stem cells with disease-specific properties that respond to drugs have formed the basis for a clinical trial. It is our hope that this novel approach demonstrates promising results and leads to better clinical trials for ALS patients in the future.”

ALS and Hyperexcitable Neurons

A large body of data supports the hypothesis that neurons become hyperexcitable in ALS, firing more than they normally do. Such over-activity may contribute to the death of the nerve cells, considered a root cause of the disease. In cell models of ALS, Retigabine has shown the ability to reduce excitability and prolong survival in the lab.

Blood samples from clinical trial participants will be collected and studied using stem cell technology to determine if the drug provides a response at the cellular level. This is the first time that this technology has been applied in a clinical trial and could help open new possibilities for drug research and development.

Take Part in the Clinical Trial

The clinical trial will be conducted at the following research institutions: Barrow Neurological Institute (Arizona), Beth Israel Deaconess Medical Center (Massachusetts), Cedars Sinai Medical Center (California), California Pacific Medical Center (California), Duke University (North Carolina), Georgia Regents University (Georgia), Johns Hopkins (Maryland), Hospital for Special Surgery (New York), Mayo Clinic (Florida), Massachusetts General Hospital (Massachusetts), University of Michigan (Michigan), University of California Irvine (California).

Although enrollment for the trial has not yet begun, study requirements and updates can be found online at: http://www.alsconsortium.org/trial.php?id=123. Sylvia Baedorf Kassis can also be contacted at the trial coordinating center to answer additional questions regarding the trial and other eligibility requirements.

Getting Involved in a Clinical Trial

Clinical trials are the only way to develop new treatments for people with ALS. Clinical trial participants contribute to improved health care for everyone with the disease. There is a lot to consider before deciding whether a clinical trial is right for you. Here are a few resources to help you get started:

Powered by Blackbaud
nonprofit software