Isolating a problem\u2019s cause means one can more easily develop treatments to address it \u2013 that\u2019s pretty straightforward. So, it\u2019s not surprising that a pharmaceutical therapy that targets a gene associated with hereditary Parkinson\u2019s disease (currently in development) may translate into a treatment. What researchers didn\u2019t expect was that this therapy may also help people with the non-hereditary form of the disease as well.\nShining a Light on the Problem\nMutations in the gene that encodes a protein called LRRK2 may account for 3 to 4 percent of cases overall, making this mutation the most common cause of hereditary Parkinson\u2019s disease. But LRRK2 is found in extremely small amounts in affected nerve cells, making it difficult to study. A team at the University of Pittsburgh School of Medicine and UPMC engineered a molecular beacon that attached to LRRK2 and glowed red under a microscope only if the protein were active. This showed them where LRRK2 was active in the brain.\nThe mutation overactivates the protein and sets the course of the disease. But researchers always thought LRRK2 would cause Parkinson\u2019s only in people with the mutation. The researchers tested postmortem brain tissue donated by Parkinson\u2019s patients (none of whom had the mutation) and healthy individuals. As it turns out, non-mutated forms may also be responsible for the more common non-hereditary form.\nThe test indicated that in the dopamine neurons \u2013 the brain cells most commonly affected in Parkinson\u2019s \u2013 LRRK2 was highly active in the brains of people with the disease but not in healthy people. This suggests that LRRK2 overactivity may be important in all people with Parkinson\u2019s \u2013 not just in those who have a mutation in the gene.\nQuestioning the Status Quo\nResearchers at the University of Pittsburgh School of Medicine and UPMC have a tendency to approach problems from a different angle. J. Timothy Greenamyre, MD, PhD, a professor of neurology at the University of Pittsburgh School of Medicine and chief of the Movement Disorders Division at UPMC (as well as director of the Pittsburgh Institute for Neurodegenerative Diseases), led the study (published in July 2018 in Science Translational Medicine) that details how his team discovered the potential role of LRRK2 in non-hereditary Parkinson\u2019s disease.\n\u201cThis discovery is extremely consequential for Parkinson\u2019s disease because it suggests that therapies currently being developed for a small group of patients may benefit everybody with the disease,\u201d said Dr. Greenamyre.\nThe Long View\nFor the approximately 10 million people worldwide with this progressive disorder of the nervous system, there is no cure \u2013 at least not yet. Nobody knows for sure what causes Parkinson\u2019s in about 90 percent of cases. But with this new understanding \u2013 and with ongoing study \u2013 the world may someday see a dramatic decrease in the devastation caused by this disease.