Synthesized Steroid Successfully Prevents Toxin Accumulation in Parkinson's Disease Animal Model

Results from a preclinical study conducted at Georgetown University Medical Center show that a synthesized steroid called squalamine was effective in animal models in preventing accumulation of a protein that causes Parkinson’s disease. The synthesized compound mimics a naturally occurring steroid found in dogfish sharks, and may present a promising avenue for therapeutic advance against Parkinson’s as well as other neurodegenerative conditions. The National Parkinson Foundation reports that pain is a major complaint of many Parkinson’s sufferers, and indeed is often among the symptoms prompting patients to seek medical help and receive the diagnosis. The findings are published online in Proceedings of the National Academy of Sciences, but embargoed as of this writing.

Parkinson’s and dementia with Lewy bodies are associated with a clustering of the protein alpha-synuclein (α-synuclein). The international research team responsible for the study found that squalamine “unsticks” α-synuclein from the nerve cell inner wall, thereby preventing a buildup of toxic clumps. The animal model studied was a nematode worm, C. elegans, that was genetically engineered to produce human a-synuclein in its muscles. Study coauthor Michael Zasloff, MD, PhD, professor of surgery and pediatrics at Georgetown University School of Medicine, stated, “We could literally see that squalamine, given orally to the worms, did not allow α-synuclein to cluster, and prevented muscular paralysis inside the worms.” A clinical trial of squalamine is being planned, according to Dr. Zasloff, who added, “Squalamine could be especially suited to work in the gut with the goal of treating the gastrointestinal symptoms of Parkinson’s.”

Read a news story about the findings and next investigative steps here.

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