Mitochondria are membrane-bound organelles in cells that produce adenosine triphosphate (ATP), the chemical providing the energy that powers most of a cell’s functions. Mitochondrial membranes regularly fuse or divide to maintain mitochondrial function and in response to a cell’s shifting metabolic needs, with greater energy production usually requiring membrane fusion—a process regulated by key proteins called mitofusins. Disrupted mitofusin activity can lead to health problems including type 2 diabetes, Charcot-Marie-Tooth type 2A neuropathy, neurodegeneration, and cancer. But until now, researchers have been unable to alter mitofusin activity and therefore couldn’t study how to prevent mitofusin dysfunction and treat those diseases.
In a study published online on July 7 in Nature Communications, Evripidis Gavathiotis, Ph.D., and colleagues report their development of novel small molecules that can target mitofusins to control the function of mitochondria by either activating or inhibiting mitochondrial fusion. The scientists used those small molecules to gain insights into how mitofusins affect metabolism, apoptosis (programmed cell death), and DNA damage and to unravel how mitofusins control these processes. Since the small-molecule drugs can modulate mitofusin function, they have the potential for treating mitofusin dysfunction-related diseases.
Dr. Gavathiotis is professor of biochemistry and of medicine at Einstein, co-leader of the Cancer Therapeutics Program at the NCI-designated Montefiore Einstein Cancer Center, and corresponding author on the paper. The paper’s first author is Emmanouil Zacharioudakis, Ph.D., associate in the department of biochemistry at Einstein and in the laboratory of Dr. Gavathiotis.
Albert Einstein College of Medicine has filed patent applications related to this research and is seeking licensing partners able to further develop and commercialize this technology. Interested parties can contact the Office of Biotechnology and Business Development at email@example.com
Posted on: Thursday, July 07, 2022