Preventing Alzheimer’s disease, gene therapy in the era of precision medicine, and using stem cells to treat CNS disease
Alzheimer’s Disease is Preventable?
To aducanumab or not—that is the question. This year was wrought with much back and forth, from BACE inhibitors to potentially NEW targets for Alzheimer’s disease. The year ended with a nice panel discussion at the New York Academy of Science’s: AD Therapeutic Alternatives to Amyloid where the top echelon of AD researchers in the field came together and discussed a plethora of new targets such as the gut microbiome, neuro-immune cells like microglia or other protein tangles like tau. The most surprising comments, though, came from MD, PhD’s articulating the need for preventative medicine. Yes, that is right. Data suggest treating the vasculature health of individuals early on in life via healthy food and lifestyle choices may prevent if not eliminate the onset of AD by reducing the inflammation that precedes disease pathology. How is this possible? Next year we may start to hear more inconvenient truths behind, “you are what you eat.”
Gene Therapies: ASO’s for precision medicine
Gene therapies are rapidly emerging as a platform for the treatment of neurological disease. We will expect to see more preclinical work in the next year that focuses on transgenes encoding therapeutic proteins, microRNAs, antibodies or gene editing machinery like CRISPR or anti-sense oligonucleotides (ASO’s). At this year’s Society for Neuroscience Meeting a scientific roundtable talked about the relevance of preclinical animal studies for refining gene therapy methods and learning more about the most effective ways to deliver genes to the central nervous system. One of the most novel applications discussed was the concept of in vivo cell conversion, where gene therapy is used to reprogram glial cells into functional neurons in the CNS, such as astrocytes reprogrammed into functional motor neurons in an SOD1G93A ALS mouse model. Also, gene therapies like ASO’s, are being used in a precision medicine-like format for rare neurodegenerative disorders like Batten’s disease.
Stem Cells in Regenerative Medicine
Recent developments in stem cell biology have provided new hope for treatments of neurological diseases and disorders. Due to their self-renewal capability, induced pluripotent stem cells are being used and tested to generate functional neurons for various neurodegenerative diseases. We may see more and more progress in the coming years where studies from the successful implantation and regeneration of dying neurons in animal models translates to humans! Can you imagine using your own adult skin cells to generate healthy new neurons in your brain? Stem cell technology is a rapidly evolving field yet it should be noted that there still lies many challenges as scientists try to reproduce published results and struggle with the differentiation potential of each cell line for robust protocols as well as tackling the ever-changing dynamic of 3D organoids to recapitulate the human brain, in a dish.