PEPSLA

The project will evaluate in a suitable transgenic mouse model for the mutated protein SOD1G93A the efficacy of a synthetic peptide patented by the proposing research group for its potential use in the treatment of Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The action of the peptide, previously validated in an appropriate cell model, is to compete with the mutant protein SOD1 for binding sites to the mitochondrial protein VDAC1, a protein on which the correct mitochondrial metabolism depends. In ALS model cells, the peptide recovers the functionality of VDAC and consequently the mitochondrial dysfunction associated with the disease resulting in a significant increase in cell viability.

Therefore, the aims of the project are to carry out all the preclinical phases of the development of a peptide compound with pharmacological activities and to certify that it meets all the requirements of safety and efficacy in a suitable mouse model of ALS.

Research will be of considerable importance for the subsequent development of possible therapy against ALS.