A team led by Tohoku University, in collaboration with Kyoto University, Keio University and the National Center of Neurology and Psychiatry, tested MA-5, a small molecule developed by the group itself to strengthen the functioning of mitochondria, the “power plants” of cells. Amyotrophic lateral sclerosis (ALS) destroys motor neurons, and current treatments only slow its progression.
In fruit flies carrying a human mutation associated with the disease, MA-5 improved movement and restored levels of ATP, the molecule that supplies energy to cells. In skin cells from patients and in motor neurons derived from iPS cells, the compound increased energy production and mitochondrial mobility, with similar effects across different genetic and sporadic forms of the disease.
The scientists also identified possible biomarkers to predict or monitor response to treatment. MA-5 has already completed a phase I trial in a mitochondrial disease, which provided preliminary information on its safety in humans.
The study, published in the journal JCI Insight, was carried out mainly in flies and cultured cells, so further tests and clinical trials in ALS patients will be needed.
