An experimental drug that reversed key symptoms of Alzheimer’s disease in mice was released by researchers at Albert Einstein College of Medicine. It’s working by reinvigorating a cellular cleaning mechanism that gets rid of unwanted proteins by digesting and recycling them. There is a truth: “Discoveries in mice don’t always translate to humans, especially in Alzheimer’s disease,” said co-study leader Ana Maria Cuervo, M.D., Ph.D., the Robert and Renée Belfer Chair for the Study of Neurodegenerative Diseases, professor of developmental and molecular biology, and co-director of the Institute for Aging Research at Einstein. But there is a hope, too: „We were encouraged to find in our study that the drop-off in cellular cleaning that contributes to Alzheimer’s in mice also occurs in people with the disease, suggesting that our drug may also work in humans.”
Alzheimer’s and all other neurodegenerative diseases are characterized by the presence of toxic protein aggregates in patients’ brains. Researchers discovered that proteins called chaperones bind to damaged or defective proteins in cells of the body. Dr. Cuervo’s team reported in Nature Communications that, for the first time, they had isolated lysosomes from the brains of Alzheimer’s disease patients and observed that reduction in the number of LAMP2 receptors causes loss of CMA in humans, just as it does in animal models of Alzheimer’s. Treatment with CA did not appear to harm other organs even when given daily for extended periods of time.