Drug for Rare Disorder Shows Promise for Parkinson's

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A drug under development for Gaucher disease may also work in the treatment of Parkinson’s disease, based on results in mice.

Marie-Francoise Chesselet, MD, PhD, of the of the UCLA Center for the Study of Parkinson’s Disease, and colleagues say that there is a potential close relationship between Gaucher — a rare genetic disorder —  and Parkinson’s over an enzyme known as GCase. People that have Gaucher are unable to produce enough of that enzyme, and mutation of the GCase gene is a genetic risk factor for Parkinson’s.

The drug, AT2101, improved motor function, stopped inflammation in the brain and reduced levels of alpha-synuclein, a protein involved in Parkinson’s that destroys brain neurons responsible for making the neurotransmitter dopamine, the researchers reported in the journal Neurotherapetics.

Dopamine plays a key role in motor functions and dopamine deficiency is associated with Parkinson’s.

"The promising findings in this study suggest that further investigation of this compound in Parkinson’s disease is warranted,” Chesselet said in a statement.

The research was funded in part by Amicus Therapeutics, the developer of AT2101.

Drug for Rare Disorder Shows Promise for Parkinson's
Drug for Rare Disorder Shows Promise for Parkinson's

A new study from UCLA found that a drug being evaluated to treat an entirely different disorder helped slow the progression of Parkinson's disease in mice. The study, published in the October edition of the journal Neurotherapeutics, found that the drug, AT2101, which has also been studied for Gaucher disease, improved motor function, stopped inflammation in the brain and reduced levels of alpha-synuclein, a protein critically involved in Parkinson's.

Although the exact cause of Parkinson's is unknown, evidence points to an accumulation of alpha-synuclein, which has been found to be common to all people with the disorder. The protein is thought to destroy the neurons in the brain that make dopamine, a neurotransmitter that helps regulate a number of functions, including movement and coordination. Dopamine deficiency is associated with Parkinson's disease. 

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