Scientists have just discovered a molecule that could stop Parkinson’s disease in its tracks

Alpha-synuclein is a naturally occurring protein found mainly in brain cells (neurons) where it regulates the release of neurotransmitters such as dopamine, allowing communication between neurons.

In Parkinson’s disease, this protein sticks together into toxic clumps that cause nerve cell death and cause patients to suffer from symptoms such as tremors, movement difficulties and muscle stiffness. Although there are treatments available to relieve symptoms, there is currently no cure.

Normally, the normal or “native state” of alpha-synuclein is like a flexible ribbon, but when it is active it shapes itself into a helix, which is crucial to its function of binding and transporting pieces of dopamine.

The team designed a peptide fragment that traps alpha-synuclein in its healthy form, preventing it from turning into toxic clumps that cause nerve cell death.

Laboratory tests showed that the peptide was stable, penetrated brain-like cells, and restored movement while reducing protein deposits in a worm model of Parkinson’s disease.

This breakthrough, published in the magazine JAX AUIt demonstrates the potential for rational peptide design to convert large, unstable proteins into compact, drug-like molecules.

The findings represent an important step toward developing new peptide-based therapies for currently untreatable neurodegenerative conditions. Professor Judy Mason, from the Department of Life Sciences at the University of Bath, said: “Our work shows that it is possible to rationally design small peptides that not only prevent harmful protein aggregation, but also function within living systems.

“This opens an exciting path toward new treatments for Parkinson’s and related diseases, where treatment options remain very limited.”

Dr Julia Dudley, head of research at Alzheimer’s Research UK, which funded the research, said:

“Dementia is not an inevitable part of aging; it is caused by diseases such as Alzheimer’s disease. To make progress toward treating all forms of dementia, we need research focused on developing a broad range of treatments that can slow, stop, and ultimately reverse these diseases.”

“Although this is early research in an animal model, it is exciting to see that this new molecule can prevent misfolded alpha-synuclein accumulation.

“By stabilizing alpha-synuclein in its healthy form, this could open the door to a new class of therapies that could slow the progression of diseases such as Parkinson’s disease and dementia with Lewy bodies. We look forward to seeing this research move to the next stage, potentially exploring how it works in humans.”

“We are delighted to see such promising developments from Alzheimer’s Research UK-funded work opening up new possibilities for future treatments, and the potential to transform the lives of people with neurodegenerative diseases.”

More research is needed, but the team hopes that continued progress will enable these and similar molecules to advance toward clinical testing in the coming years.