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A natural compound can block the formation of toxins associated with Parkinson's disease

University of Cambridge News Jan 30, 2017

Squalamine, a natural product studied for its anticancer and anti–infective properties, could also lead to future treatments for Parkinson’s disease.

A naturally–occurring compound has been found to block a molecular process thought to underlie Parkinson’s Disease, and to suppress its toxic products, scientists have reported.

The findings, although only preliminary, suggest that the compound, called squalamine, could be exploited in various ways as the basis of a potential treatment for Parkinson’s Disease. The compound has previously been used in clinical trials for cancer and eye conditions in the United States, and a trial in Parkinson’s Disease patients is now being planned by one of the researchers involved in the study.

Squalamine is a steroid which was discovered in the 1990s in dogfish sharks. It is, however, impossible to derive any medical benefits from shark tissue, and the form used by scientists is a safer and more reliable synthetic analogue. To date, it has been extensively investigated as a potential anti–infective and anticancer therapy.

But in the new study, researchers discovered that squalamine also dramatically inhibits the early formation of toxic aggregates of the protein alpha–synuclein – a process thought to start a chain reaction of molecular events eventually leading to Parkinson’s disease. Remarkably, they also then found that it can suppress the toxicity of these poisonous particles.

The researchers tested squalamine in both cell cultures in the lab, and in an animal model using nematode worms. While their findings therefore only represent a step towards a treatment for Parkinson’s Disease in humans, they described the results as representing significant progress.

The study was led by academics from the Centre for Misfolding Diseases, based in the Chemistry Department at the University of Cambridge in the United Kingdom, and Georgetown University and the National Institutes of Health in the United States. Scientists from the Netherlands, Italy and Spain also played key roles.

The findings were published in Proceedings of The National Academy of Sciences journal.
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