The ALS Association, Harvard Stem Cell Institute, and Massachusetts General Hospital Neurological Clinical Research Institute have announced that they are collaborating with GlaxoSmithKline on a clinical trial to evaluate the potential of an anti-epileptic drug in ALS patients. In parallel testing, brain cells will be made from each patient’s stem cells to see if they can predict which patients might respond to the medicine.
The trial will evaluate the potential of the drug, Retigabine, which has a unique mechanism of action and can calm the excitability of nerve cells that are thought to cause seizures. These “hyperexcitable neurons”are also thought to play a role in ALS. Alongside testing of the medicine, scientists will for the first time create stem cells from these patients to see if they can be used to determine in advance which patients could benefit from the medicine.
The study is being led by Brian Wainger of the department of Neurology at Massachusetts General Hospital, in collaboration with Merit Cudkowicz, chief of Neurology at MGH. It will be performed at 12 academic sites within the Northeast ALS Consortium, an international, independent, non-profit group of researchers who collaboratively conduct clinical research in ALS and other motor neuron diseases. GSK will provide the drug, and funding support will come from HSCI, The ALS Association, the MGH NCRI and GSK.
“This novel study will provide us with a better understanding of neuron hyperexcitability, a potentially important disease mechanism in ALS patients,” says Lucie Bruijn, chief scientist for The ALS Association. “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.”