Developing Wide-Spectrum Antiproteotoxicity Agents to Treat ALS

Abstract : ALS remains a devastating neurodegenerative disease with no curative treatments available. It is becoming increasingly clear that the accumulation of misfolded and aggregated proteins underlie the pathologies of several major forms of ALS. Protein products of ALS genes, including SOD1 and TDP-43, have been involved in the pathology of protein aggregation. In the past year of research, we have made significant progress towards identifying novel therapeutic agents against protein aggregation and delivering the agents to the relevant nervous systems. Our work has revealed that inhibitors of specific targets have robust effects on protein aggregation in a protein-dependent manner. We have also demonstrated significant progress in delivering agents to in vivo animal models. Further studies are required to pursue some of the new findings that were observed.