Arimoclomol is an investigative medicinal product, meaning it is not an approved medicine. We are conducting clinical studies to investigate how effective arimoclomol is as a treatment for a number of diseases. To be approved, all new therapies must be effective and must not cause undue harm. We have studied arimoclomol’s effects thoroughly in the laboratory as well as in clinical trials in healthy human volunteers and are now investigating its effects and safety in patients.
Lysosomal storage diseases
Many lysosomal storage diseases are caused by mutations in a protein involved in lipid digestion and transportation. This causes an accumulation of lipid in compartments of the cell called lysosomes, which in turn leads to cell stress and toxicity. When stressed cells are treated with arimoclomol, they are stimulated to produce a natural chaperone called heat-shock protein 70 (HSP70), which helps to fold the digestive enzyme into a functional conformation, allowing the cell to process the accumulated lipid.
In rare circumstances, some proteins are vulnerable to aggregation, meaning they clump together and don’t get recycled properly. Aggregations of misfolded proteins cause cell stress, and eventually cell death. When stressed cells are treated with arimoclomol, they are stimulated to produce a natural chaperone called heat-shock protein 70 (HSP70), which helps to guide aggregated proteins into the recycling pathway and prevents newly-built proteins from aggregating.