AANS Neurosurgeon | Volume 27, Number 3, 2018

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Experimental Drug Stops Parkinson’s Disease Progression in Mice

Johns Hopkins researchers say they have developed an experimental drug, similar to compounds used to treat diabetes, that slows the progression of Parkinson’s disease itself — as well as its symptoms — in mice. In experiments performed with cultures of human brain cells and live mouse models, they report the drug blocked the degradation of brain cells that is the hallmark of Parkinson’s disease. The drug is expected to move to clinical trials this year.

“It is amazingly protective of target nerve cells,” says Ted Dawson, MD, PhD, director of the Institute for Cell Engineering and professor of neurology at the Johns Hopkins University School of Medicine.

Dawson explains that if planned clinical trials for the drug, named NLY01, are successful in humans, it could be one of the first treatments to directly target the progression of Parkinson’s disease, not just the muscle rigidity, spasmodic movements, fatigue, dizziness, dementia and other symptoms of the disorder.

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