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Medical Rounds

At the Bench: Promise for Parkinson’s

A Johns Hopkins team has developed an experimental drug similar to compounds used to treat diabetes that slows the progression of Parkinson’s disease and its symptoms in mice. In experiments performed with cultures of human brain cells and live mouse models, it reports that the drug, called NLY01, blocked the degradation of brain cells that is the hallmark of Parkinson’s.

“It is amazingly protective of target nerve cells,” says neurologist Ted Dawson, director of the Institute for Cell Engineering.

The drug is expected to move to clinical trials this year, and if those trials are successful, says Dawson, NLY01 could be one of the first treatments to directly target the progression of Parkinson’s—not just the muscle rigidity, spasmodic movements, fatigue, dizziness, dementia and other symptoms of the disorder.

According to the investigators, NLY01 works by binding to glucagonlike peptide-1 receptors on the surface of certain cells. Similar drugs are used widely in the treatment of type 2 diabetes to increase insulin levels in the blood. Though past studies in animals suggested the neuroprotective potential of this class of drugs, researchers had not shown directly how it operated in the brain.A report of the study’s results was published in Nature Medicine.

Watch a video to learn more about Ted Dawson’s work using stem cells to study Parkinson’s disease.