With assistance from a high tech robot, NIH researchers have identified and tested a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson’s disease. "Until now, drugs used to treat Gaucher disease have not been able to enter the brain and reach those neurons that are affected in the most severe forms of Gaucher disease or in Parkinson’s disease," said Ellen Sidransky, MD, a senior investigator with NIH’s National Human Genome Research Institute (NHGRI). "It’s really exciting to have found a molecule that theoretically could be widely available to treat people with these diseases." To better understand the connection between Gaucher and Parkinson’s diseases, NHGRI researchers used a labor-intensive technology to develop pluripotent stem cells (unspecialized cells that can develop into various specialized body cells). In a process known as high-throughput drug screening, researchers at the National Center for Advancing Translational Sciences (NCATS) Chemical Genomics Center identified a promising molecule, NCGC607. In the patients’ stem cell-derived neurons, NCGC607 reversed the lipid accumulation and lowered the amount of alpha-synuclein, suggesting a possible treatment strategy for Parkinson’s disease. Researchers will next test the new molecule to see if it might be developed into an appropriate prototype drug for patients with Gaucher disease and Parkinson’s disease.