Two drugs designed by researchers at Albert Einstein College of Medicine preserved vision in mice that developed retinitis pigmentosa (RP), an inherited eye disease that can lead to blindness and for which no treatments currently exist. The findings, published online on July 21 in Nature Communications, suggest that such drugs might also work against more common retinal diseases.
The drugs were specifically designed to boost chaperone-mediated autophagy (CMA), a vital “housekeeping” process that disposes of unwanted proteins in cells but becomes less efficient as people age. Ana Maria Cuervo, MD, PhD, co-corresponding author of the paper, discovered CMA in 1993 and named it in 2000. Dr. Cuervo, who has published more than 200 papers on CMA, is Professor of Developmental and Molecular Biology, of Medicine, the Robert and Renée Belfer Chair for the Study of Neurodegenerative Diseases and Co-Director of the Institute for Aging Research at Einstein.
Dr. Cuervo’s research has shown that the decline in cellular cleaning is linked to Alzheimer’s, Parkinson’s, atherosclerosis and other age-related diseases. For more than seven years, she has worked with her Einstein colleague Evripidis Gavathiotis, PhD, to develop drugs that can treat these and other diseases by revving up CMA. Dr. Gavathiotis, Professor of Biochemistry and of Medicine at Einstein, and Co-Leader of the Cancer Therapeutics Program at the NCI-designated Montefiore Einstein Cancer Center, uses structure-based drug design and medicinal chemistry to create new drug compounds.