Amyotrophic Lateral Sclerosis (ALS)

ALS is a disastrous disease, where patients die paralysed in 2-4 years. Thus we have targeted with innovative research. Using lipidomics, and a large international consortium,  we found that glycosphingolipids (ceramide/glucosylceramide) were profoundly modified in spinal cord and muscle of different animal models of ALS, that inhibiting glucosylceramide synthase (GCS) was highly deleterious for denervation while inhibiting glucosylceramidase (GCase) was beneficial. We found that the generic drug ambroxol was a specific GCase modulator, changing ceramide/glucosylceramide ratios, preventing denervation, but also increasing re-innervation of neuromuscular junctions (NMJs). We have developed ambroxol for ALS, where it is ready for phase II in Australia, (50/50 patent with INSERM, SRS is the manager), and we have obtained orphan drug status from the EMA.

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