Cells prompted to "eat" Huntington proteins

Scientists have developed a novel strategy for tackling neurodegenerative diseases such as Huntington's disease: encouraging an individual's own cells to "eat" the malformed proteins that lead to the disease. Professor David Rubinsztein, Wellcome Trust Senior Clinical Fellow at the University of Cambridge, has been studying the molecular biology underlying Huntington's and other neurodegenerative diseases. Huntington's occurs when a protein known as huntingtin builds up in the brain cells of patients, mainly in neurons in the basal ganglia and in the cerebral cortex. Normally, cells dispose of or recycle their waste material, including unwanted or mis-folded proteins, through a process known as autophagy, or "self-eating".

"We have shown that stimulating autophagy in the cells--in other words, encouraging the cells to eat the malformed huntingtin proteins -- can be an effective way of preventing them from building up," says Professor Rubinsztein. "This appears to stall the onset of Huntington's-like symptoms in fruit fly and mice, and we hope it will do the same in humans."

- read the report on the research study

Suggested Articles

Compass' CD137 agonist cleared large tumors in mice that other I-O agents had failed to treat. It's advancing the drug into phase 1 human trials.

UPMC researchers are planning clinical trials of a COVID-19 vaccine that uses pieces of the virus' spike protein to create immunity.

Treating mice with niacin increased the number of immune cells in glioblastomas, reducing tumor size and extending survival.