Neurodegenerative Disease Insights Can Advance Precision Medicine

Rutgers researchers discovered some of the first molecular insights into how toxic proteins are regulated in neurodegenerative disease, potentially advancing precision medicine efforts.

The most common neurodegenerative diseases are Alzheimer’s and Parkinson’s. According to the Alzheimer’s Association, more than 6 million people in the United States live with Alzheimer’s. Additionally, the disease kills more people than breast and prostate cancer combined and contributes extensively to high healthcare costs. 

Parkinson’s Disease also reflects high healthcare costs and impact. Nearly one million people in the US live with the disease, and 60,000 more are diagnosed each year. According to the Parkinson’s Foundation, that’s more than the combined number of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig’s disease.

Within the next ten years, cases of Alzheimer’s and Parkinson’s disease are expected to climb, continuing to strain the healthcare system. However, researchers can develop therapeutic targets to treat the disease by gaining molecular insight into how toxic proteins impact neurodegenerative diseases.

Although there is no cure for either Alzheimer’s or Parkinson’s Disease, creating chronic disease management treatments can potentially improve the quality of life for people living with neurodegenerative diseases.

While cells naturally grow older and die, proper regulation of cellular proteins is important in maintaining a healthy brain as individuals age. In neurodegenerative diseases, however, protein aggregates will spread to neighboring cells. Unfortunately, despite knowing that the toxic material does spread, it remains poorly understood how it happens.

The Rutgers research team studied roundworms whose stressed nerve cells can extrude neurotoxic proteins in large packets called exophers and how certain stresses impact this extrusion. The researchers discovered that specific cellular signals are necessary to form exophers and that fasting significantly increases exopher production.

“In establishing an initial molecular model for trans-tissue requirements for fasting-induced exopher elevation in neurons, we report molecular insights into the regulation of aggregate transfer biology relevant to the fundamental mysteries of neurodegenerative diseases,” the study’s first author Jason Cooper, a postdoctoral research fellow in the Department of Molecular Biology and Biochemistry at Rutgers University-New Brunswick, said in a press release.

“In neurodegenerative diseases, toxic proteins spread to neighboring cells to promote cell death. Given the importance of managing protein aggregates in aging and neurodegenerative diseases and the poorly understood biology of how those aggregates are transferred, a detailed understanding of the transfer mechanism may reveal previously unrecognized therapeutic targets.”

Researchers can turn their efforts to precision medicine development by identifying therapeutic targets, improving patient outcomes and care.