Neuromuscular Disease Expert Robert H. Baloh, MD, PHD, Joins Cedars-Sinai Medical Center To Advance Lou Gehrig’s Disease Research
Baloh studies molecular mechanisms of Lou Gehrig’s disease, muscular dystrophies, spinal muscular atrophies and other nerve and muscle diseases
Baloh, a prolific research scientist who has published groundbreaking discoveries in genetics and molecular biology, is the principal investigator of five projects examining the molecular and cellular basis of neuromuscular disorders, primarily focused on ALS, but also on muscular dystrophies and inherited peripheral nerve and muscle disorders including Charcot-Marie-Tooth disease, one of the most common inherited neurological disorders, named for the three doctors who identified it in 1886. Two projects are funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke and others are supported by grants from the Muscular Dystrophy Association and Burroughs Wellcome Foundation.
He has published articles on the role that mitochondria – components of cells that “digest” nutrients and produce energy – play in peripheral nerve disorders. His research group was one of the first to discover that mutations in a gene (TDP-43) cause inherited forms of ALS and frontotemporal lobar degeneration, a type of dementia that can accompany ALS.
He led studies that in 2009 produced a mouse model of ALS based on a mutation in the TDP-43 gene – the first new mouse model of ALS in 14 years – that now is employed by researchers worldwide to study the disease. His group recently identified new genetic causes of both spinal muscular atrophy and limb girdle muscular dystrophy, degenerative illnesses that typically afflict young children.
“The Regenerative Medicine Institute continues to make progress in developing new stem cell treatments for Lou Gehrig’s disease. Dr. Baloh will be a critical part of translating these innovative treatments to the clinic and providing new insights into the mechanisms of how cells die in these disorders,” Svendsen said. “Our laboratory has been working on ALS for more than 10 years and we are very excited to have Dr. Baloh as a new neighbor.”
Lyden noted that discoveries like Baloh’s are changing the way scientists view the fundamentals of these disorders and the possibilities for creating new treatments. In many neurodegenerative diseases, clumps of abnormal proteins disrupt cell-to-cell signaling and kill neurons; different diseases target different types of nerve cells. Baloh’s research focuses on understanding the genetic abnormalities that cause disease; these genes provide a roadmap of molecules that are possible drug targets in diseases that have few if any treatments.
“Neurodegenerative diseases cause a loss of function: memory in the case of Alzheimer’s disease, motor control in the case of Lou Gehrig’s disease, and balance and coordination in the case of Parkinson’s disease, to name a few,” Lyden said. “We now know that these diseases are all linked, with common underlying biochemistries. From a treatment perspective, as we gain an understanding of one, we gain the principles to treat others. Having Dr. Baloh’s expertise contributing to our research will be invaluable.”
Before joining Cedars-Sinai, Baloh was an assistant professor of neurology at Washington University School of Medicine in St. Louis, where he earned his MD and PhD after graduating from Brown University. He completed an internship at Brigham and Women’s Hospital in Boston and a neurology residency at the Massachusetts General Hospital/Brigham and Women’s Hospital combined neurology program, serving as chief resident his final year. He continued his training with a fellowship in neuromuscular diseases at Washington University in St. Louis, and is board certified in both neurology and neuromuscular medicine.
Source: Cedars-Sinai Medical Center
