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WASHINGTON, Jan. 16 (Xinhua) -- A synthesized compound mirroring one naturally made by the dogfish shark may have the potential to treat Parkinson's disease, scientists said Monday.
Preliminary findings suggested that the compound, called squalamine, blocks a molecular process thought to underlie Parkinson's Disease, and suppresses its toxic products, according to the study published in the U.S. journal Proceedings of The National Academy of Sciences.
Squalamine, a steroid which was first discovered in the 1990s in dogfish sharks and then synthesized in a process that does not involve use of any natural shark tissue, has previously 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 study led by researchers from the University of Cambridge and Georgetown University tested squalamine in both cell cultures in the lab, and in an animal model using nematode worms that had been genetically modified to suffer from a build-up of alpha-synuclein that causes cell damage and paralysis.
"To our surprise, we found evidence that squalamine not only slows down the formation of the toxins associated with Parkinson's disease, but also makes them less toxic altogether," said study author Professor Christopher Dobson of the University of Cambridge.
"If further tests prove to be successful, it is possible that a drug treating at least some of the symptoms of Parkinson's disease could be developed from squalamine. We might then be able to improve on that incrementally, by searching for better molecules that augment its effects."
The research team also showed that squalamine could protect healthy human neuronal cells from being damaged by preventing alpha-synuclein from adhering to the outside of the neuronal cells.
One of the researchers is now planning a clinical trial with squalamine in Parkinson's disease patients in the United States.
"In many ways squalamine gives us a lead rather than a definitive treatment," Professor Dobson added. "Parkinson's disease has many symptoms and we hope that either this compound, or a derivative of it with a similar mechanism of action, could alleviate at least some of them."
"One of the most exciting prospects is that, subject to further tests, we might be able to use it to make improvements to patients' lives, while also studying other compounds with the aim of developing a more powerful treatment in the future."
