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Robert H. Baloh, MD, PhD, Wins PRISM Award


Robert H. Baloh, MD, PhD

Robert H. Baloh, MD, PhD, associate professor and director of Neuromuscular Medicine at the Cedars-Sinai Department of Neurology, is the recipient of the 2016 Prize for Research in Scientific Medicine (PRISM) award. The $10,000 prize recognizes outstanding scientific or medical breakthroughs achieved in the preceding five years by Cedars-Sinai faculty members.

Baloh received the honor June 15 at the commencement of the Cedars-Sinai Graduate Program in Biomedical Science and Translational Medicine in Harvey Morse Auditorium. The award citation stated that he and his colleagues "have exemplified the finest examples of biomedical discovery by digging ever deeper into the pathophysiology of an all-too-common set of devastating neurological diseases."

The Ben Winters Chair in Regenerative Medicine is renowned for his research on amyotrophic lateral sclerosis (ALS), a fatal, incurable muscle-wasting disorder that is also known as Lou Gehrig's disease. His award was for a series of discoveries involving an inherited form of ALS and frontotemporal lobar degeneration, a type of brain dementia that can accompany ALS.

The winning work examined a genetic mutation that is shared by these two disorders. The mutation causes the gene to both suppress production of a protein it normally makes and produce a toxic chemical. Baloh and his team wanted to know exactly how these twin processes worked to produce ALS and dementia.

To answer the question, they began with pluripotent stem cells, which have the power to create any type of cell. They used stem cells from a patient with ALS and used them to grow motor neurons in a laboratory dish. Then they watched how the mutated gene damaged the neurons.

In a remarkable accomplishment, the researchers were able to reverse or prevent damage to the lab-grown neurons by using chemicals to block the actions of the mutated gene. The next step was to create a mouse model of the disease, which showed that the processes demonstrated in the lab dish also damaged the nervous system in living animals and could be reversed.

Oddly, despite the damage at the cellular level, the mice did not show symptoms of ALS within their lifespan. This unexpected finding spurred the researchers to delve more deeply. They discovered that the same mutated ALS gene was critical for the function of the immune cells in the brain, and led to altered inflammatory responses. This new finding led them to propose that it is the combination of altered immune function and the toxic effects of the gene that act in concert to cause ALS and frontotemporal dementia.

Based on these discoveries, a multicenter consortium, which includes Baloh's team, has been formed to perform a clinical trial on a potential treatment for ALS and frontotemporal lobar degeneration.

"The PRISM prize is not the end of the story," Baloh said in an interview after the June 15 commencement. "It's the beginning of a story. I'm excited to see where this research might lead in the development of therapeutics and drugs for these two disorders."

Baloh joined Cedars-Sinai in 2012. He is the recipient of numerous awards, including the ALS Association Golden West Chapter Commitment to a Cure Award in 2012.

This is the second year that Cedars-Sinai has awarded the PRISM prize. The 2015 prize went to Stanley C. Jordan, MD, professor of Medicine and director of Kidney Transplantation and Transplant Immunology. Winners are chosen by an external committee of prominent scientists.