The ALS Association together with the Harvard Stem Cell Institute, and the Neurological Clinical Research Institute at Massachusetts General Hospital recently announced in a press release a collaboration with GlaxoSmithKline (GSK) in a clinical trial to assess a potential therapy with an anti-epileptic drug in patients with amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative disease, characterized by the gradual degeneration and atrophy of motor neurons in the brain and spinal cord that are responsible for controlling voluntary muscles, such as the ones related to movement, speaking, eating, and even breathing. ALS patients may become totally paralyzed and the majority dies due to respiratory failure within two to five years after diagnosis. There is no cure for the disease or any life-prolonging treatment.
The clinical trial will assess the drug Retigabine (GSK), an anticonvulsant designed to calm the excitability of nerve cells thought to be linked to epileptic episodes characterized by seizures. These “hyperexcitable neurons” have also been suggested to be implicated in ALS pathology. In fact, Retigabine has been shown to reduce the excitatory state and prolong neuron survival in ALS cell models.
Besides evaluating Retigabine as a potential therapy for ALS, researchers will also, for the first time, generate cells from stem cells of each ALS patient enrolled in the trial and define whether these cells can be a helpful predictor for patients who could benefit from treatment with the drug in advance.
“This novel study will provide us with a better understanding of neuron hyperexcitability, a potentially important disease mechanism in ALS patients,” said the Chief Scientist for The ALS Association Dr. Lucie Bruijn in the press release. “This powerful collaboration of leaders in the fields of stem cells, clinical neurology, ALS research and GSK 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.”
The trial will be conducted at twelve different academic sites within the Northeast ALS Consortium, an international, collaborative consortium of researchers centered on ALS clinical research and other motor neuron diseases.