This drug may stop Parkinson's disease, human trials set for 2019

A newly published study details an experimental drug that may be able to stop the progression of Parkinson's disease. The study, which was conducted by researchers with Johns Hopkins University, saw positive results in lab mice tested with the drugs and similar effects were seen with tests on cultures of human brain cells. Researchers indicate that human clinical trials of the drug may start as soon as next year.

Parkinson's disease affects the central nervous system, causing tremors and muscle stiffness, among other issues. An exact cause for the disease is not known, though dopamine levels appear to play an important role in the disorder's development. Various medications are available that help control symptoms of the disease, but thus far medical science has not produced a drug that can prevent or stop it.

That may change thanks to this latest work coming out of Johns Hopkins. According to a recent announcement from the university, researchers have created an experimental drug that slows down the progression of the disease and may entirely halt the degradation of brain cells. The new drug is said to be similar to compounds that are used in the treatment of diabetes.

The drug, called NLY01, was tested on live mouse models and cultures of human brain cells, where it was observed to block brain cell degradation that results from Parkinson's disease. Unlike existing drugs, which target various symptoms of the disease, NLY01 targets the damage to cells that result in the symptoms.

The drug may work by blocking the conversion of astrocytes, a type of cell, into "aggressive activated" astrocytes, which attack the connection between brain cells, ultimately resulting in neuronal death. Researchers say human clinical trials of the drug are anticipated to start in 2019, enabling experts to test for both efficacy and safety in human patients.

Given NLY01's similarity to existing diabetes medication, researchers are hopeful that the drug will have a relatively simple path toward eventual clinical use.

SOURCE: Johns Hopkins University, Nature