Retigabine, also called ezogabine, is a drug developed by GlaxoSmithKline (GSK) under the brand name Potiga that is currently undergoing a Phase II Clinical Trial to assess it as a potential therapy for those with ALS/MND. Retigabine was originally developed and FDA approved for epilepsy to calm the nerve cells linked to epileptic seizures. Those same neurons may also be implicated in ALS pathology. Data supports the hypothesis that neurons become hyperexcitable in ALS, firing more than they normally do, which may contribute to nerve cell death. This is considered a root cause and/or effect of ALS/MND. In cell models of ALS, retigabine has shown the ability to reduce excitability and prolong survival in nerve cells.
Previous in vitro pre-clinical trial results indicate retigabine can have two primary benefits for ALS/MND patients. First, the medication may stop fasciculations, thereby alleviating, stopping, or potentially reversing certain ALS/MND symptoms. Secondly, the medication may also reduce spasticity and rigidity.
In addition to evaluating retigabine as a potential therapy for ALS, researchers will also derive stem cells from epithelial (skin) cells of ALS patients enrolled in the trial. Researchers will then use these cells as a predictor to determine which patients may benefit from retigabine prior to their actual usage of the drug.
The study is being led by Brian Wainger, M.D., Ph.D., of the department of Neurology at Massachusetts General Hospital, in collaboration with Merit Cudkowicz, M.D., Chief of Neurology at MGH, and is co-sponsored by ALSA and GSK. The trial will be conducted at twelve different academic sites within the Northeast ALS Consortium, an international, collaborative group of researchers centered on ALS/MND and other motor neuron diseases. We strongly endorse entry into the trial for those who qualify because there are strong anecdotal suggestions of the potential efficacy of retigabine and we feel this is an important, well-designed clinical trial that has significant opportunity for the patient community.
Trial entrants will be selected shortly, initial dosage will be provided in July 2015, 120 ALS patients will be selected and will be divided equally between 900mg daily, 600mg daily and placebo. To see whether you or a loved one qualifies for the trial, please visit the trial's page on the Clinical Trials website. If you do not qualify for the trial, you may wish to discuss the use of retigabine with your neurologist or physician. Potential adverse side effects of retigabine should be considered, discussed with your physician and analyzed before its usage, as should be done with all medications.
As always, please contact [email protected] for further information about this trial.