The Parkinson’s Institute
Sunnyvale, California

The Parkinson Institute has formed a collaboration with Neuraltus Pharmaceuticals to develop a small molecule compound for the treatment of dyskinesias caused by the drugs that are taken by most Parkinson’s sufferers. Founded in 1988, The Parkinson’s Institute and Clinical Center (PI) is America’s only independent non-profit organization that provides basic and clinical research, clinical trials and a comprehensive movement disorder patient clinic for Parkinson’s disease and related neurological movement disorders. The Institute’s mission is to find the causes, provide first class patient care and discover a cure. Research at the Parkinson’s Institute involves a multidisciplinary and interactive approach toward the overall goal of understanding the natural history of Parkinson’s disease (as well as related neurological disorders), finding its cause and developing better treatments. Specific lines of inquiry into the cause, cure and prevention of the disease are more effectively pursued through the coordinated efforts of investigators with different scientific background and expertise (e.g. molecular biology, pharmacology and toxicology), and the use of state-of-the-art experimental tools at the molecular, cellular and whole organism levels. The close interaction between scientists and clinicians provides a direct mechanism for translational laboratory-to-patient research. One key area of research is the mechanism of dyskinesias.

The Hospital for Sick Children
Toronto, Canada

Neuraltus is collaborating with Dr. Clifford Lingwood, Senior Scientist in the Molecular Structure & Function program at the SickKids Research Institute on the development of a novel compound for the treatment of lysosomal storage disorders. In a healthy human, cell structures called lysosomes process and breakdown unwanted substances inside the cell. Individuals suffering from a lysosomal storage disorder have a build up of the unwanted substances within the cells. This can lead to any of approximately 40 known lysosomal storage disorders with a wide variety of symptoms, including developmental delays, muscular disorders, seizures, deafness and blindness, and can end in death. Neuraltus and Lingwood will continue to perform research in hopes of developing the compound as a treatment for a number of these disorders. The compound, collaboratively developed by Lingwood and Neuraltus, affects lysosomal storage disorders by reducing the rate at which a molecule called a glycolipid is created within the body. Overproduction of glycolipids can interfere with how the cells grow and mature, how the cells adhere to each other and their ability to prevent tumours from forming. This can lead to serious disorders like Tay-Sachs, Gaucher’s, and Fabry’s diseases.