The drug, a type of histone deacetylase (HDAC) inhibitor being evaluated in numerous ongoing clinical trials, has been shown to reverse the harmful effects of autophagy in heart muscle cells of mice. Autophagy is a natural process by which cells eat their own proteins to provide needed resources in times of stress. The new study appears in Proceedings of the National Academy of Sciences.
The researchers engineered mice with overactive autophagy and induced hypertrophy leading to heart failure. Scientists then gave the mice an HDAC inhibitor known to limit autophagy.
“The heart decreased back to near its normal size, and heart function that had previously been declining went back to normal,” Dr. Hill said. “That is a powerful observation where disease regression, not just disease prevention, was seen.”