Key players join forces to tackle unmet needs in Parkinson's disease

iStock photos

iStock photos

The laboratory of Professor Hilal Lashuel at the EPFL Brain Mind Institute, and Idorsia Pharmaceuticals Ltd – a clinical-stage biopharmaceutical company specialized in the discovery and development of small molecules – entered a three-year collaboration agreement in October 2020. Their objective: to combine and apply their unique expertise and resources to discover and develop highly needed therapies for millions of patients suffering from Parkinson’s and related neurodegenerative diseases. Leveraging strong ties between academia and industry is key to address such highly complex disorders.

“The protein alpha-synuclein is thought to play a central role in neurodegenerative diseases, and is one of the most investigated targets for developing diagnostics and therapies for Parkinson’s disease,” explains Hilal Lashuel, director of the Lashuel lab at EPFL’s School of Life Sciences. Alpha-synuclein is abundantly expressed in the brain, in particular at the tips of neurons, called presynaptic terminals. The accumulation of misfolded and aggregated alpha-synuclein in neuronal cells in the form of intracellular inclusions – called Lewy bodies – is one of the defining hallmarks and central events in the development of Parkinson’s disease.

A great match, and a potential game changer for patients

“This collaboration combines some of the best skills and know-how from academia and industry with the common objective of finding new small molecules for the treatment of Parkinson’s disease and other alpha-synucleinopathies,” says Michel Steiner, group leader of the Central Nervous System pharmacology department at Idorsia. Elodie Angot, senior scientist in the same department, is also thrilled to be part of this adventure. She started her career in academia doing research on mechanisms of alpha-synuclein cell-to-cell transfer in Parkinson’s disease, which is when she came to know about the work performed by Lashuel and his team. “I could not guess at that time that I would have the opportunity to collaborate with them on a drug-discovery program in Parkinson’s disease that has the potential to be a game changer for patients,” she explains enthusiastically.

“We are excited to embark on this collaboration with Idorsia,” adds Lashuel. “On the one hand, it will leverage the expertise, integrative experimental approaches, and disease models developed in our lab. On the other hand, it will bring in Idorsia’s unique chemical libraries, high-throughput screening, and drug discovery capabilities. We feel that we are well positioned to discover novel modifiers of alpha-synuclein aggregation and toxicity as potential therapeutic agents for the treatment of Parkinson’s disease and/or other alpha-synucleinopathies”.

Two-decade expertise in a patient-centered approach to neurodegenerative diseases

“Our group is committed to advancing translational research and drug discovery in the field of neurodegenerative diseases, and we see strategic collaborations such as this one with Idorsia as essential for translating our findings to the clinic,” concludes Lashuel. His lab is one of the leading groups working on protein misfolding and aggregation. With twenty years of experience, the group has an extensive track record in using integrative approaches to elucidate the role of alpha-synuclein in health and disease.