(RxWiki News) Researchers from the Scripps Research Institute have demonstrated the first known compound to show considerable effectiveness in protecting brain cells affected by Parkinson's disease.
Research leader Philip LoGrasso said the study provides the first compelling data on an oral, brain-penetrating inhibitor to demonstrate this effectiveness in Parkinson's disease, a progressive, neurodegenerative disease that causes tremor and coordination problems from the loss of a group of neurons in part of the midbrain involved in motor control.
The compound -- a molecule known as SR-3306 -- impairs a class of enzymes called c-jun-N-terminal kinases (JNK, pronounced "junk"). JNK enzymes play a vital role in the survival of neurons (nerve cells) and have become a target for drugs to treat Parkinson's.
The SR-3306 molecule performed well in animal models and especially in cell culture, where the compound showed greater than 90 percent protection against neuron death. The compound performed with 72 percent efficacy in animal models.
The next step will be to conduct clinical trials in humans if an application for an investigational new drug (IND) is accepted by the Food and Drug Administration.
The National Institutes of Neurological Disorders and Stroke (NINDS) funded the study, part of a $7.6 million multiyear grant.
According to the Parkinson's Disease Foundation, the disease afflicts as many as one million Americans -- more than the combined number of people diagnosed with multiple sclerosis, muscular dystrophy and amyotrophic lateral sclerosis (ALS).