Targeting Mitochondrial Dysfunction in Neurodegenerative Disease

The Dana Foundation. By Kayt Sukel, August 15, 2017

It’s long been known that the brain is an energy glutton: Most estimates suggest that it requires about 20 percent of the body’s total energy stores to work effectively. That energy is produced by mitochondria, specialized rod-shaped organelles in every cell in the body. So perhaps it is no surprise that problems with mitochondrial function in neurons have been linked to a host of progressive neurodegenerative disorders—one of which is Friedreich’s ataxia, a rare but devastating disease that progressively kills off nerve cells in the brain and spinal cord. Now, scientists at the University of California, Davis have identified how frataxin, the gene that causes Friedreich’s ataxia, affects mitochondrial function. That understanding may lead to new, more effective potential treatments for Friedreich’s ataxia as well as other forms of neurodegenerative disease.

Cortopassi hopes to take dimethyl fumarate (DMF) to clinical trial for Friedreich’s ataxia patients soon. He also thinks measuring mitochondrial number could be an important biomarker to test the efficacy of treatments for other neurodegenerative disorders as well. Manfredi agrees.



 Targeting Mitochondrial Dysfunction in Neurodegenerative Disease