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Innovative drugs against neurodegenerative disorders

Neurodegeneration impacts the quality of life of patients and their families and has wider socioeconomic implications. To discover new therapeutic approaches, a group of European researchers developed small cell-permeable peptides that specifically target signalling pathways implicated in neurodegenerative diseases.
Innovative drugs against neurodegenerative disorders
Currently, one in three people in the developed world suffer from a central nervous system disorder, and the European budget to treat brain disorders exceeds that for cardiovascular diseases and cancer combined.

The EU-funded CPADS initiative aimed to develop innovative therapeutic solutions for the most common neurodegenerative disorders. The idea behind the project was to synthesise cell-permeable peptides that can manipulate biological pathways involved in degenerative events that cause neuronal cell death.

These peptides present a powerful way of blocking intracellular signalling pathways or intracellular protein complexes. Notably, apart from their therapeutic exploitation, cell-permeable peptides are important tools for creating animal models of disease. Project results and objectives are available on the project website .

Scientists of the CPADS project focused on Alzheimer's (AD) and Parkinson's (PD) disease as paradigms for the identification and characterisation of such therapeutic peptides. More than 12 lead peptides were tested for their implication in key signalling pathways involved in neurodegeneration.

In vivo screening highlighted the D-JNKI1 peptide as a rescuing treatment in AD and GluN2A/PSD-95-competing peptide as a means of improving motor behaviour in an experimental model of PD. Researchers using cell-permeable peptides to block specific transduction pathways generated an innovative animal model of sporadic AD that could be useful in drug screening.

Taken together, the results of the CPADS study demonstrated the potential of specific cell-permeable peptides to act as experimental treatments for neurodegenerative disorders. Evaluation of these novel drugs at the clinical level will determine their efficacy as future therapeutic strategies.

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