Neural Stem Cell Institute, Scientific Director, Principal Investigator and Co-Founder
Albany Medical College, Professor of Neuroscience and Experimental Therapeutics (DNET)
Age-related Macular Degeneration (AMD) is the leading cause of blindness affecting 1 in 5 people over the age of 75. As AMD progresses, patients lose the ability to recognize faces or read, and they become increasingly dependent and experience deteriorating quality of life. Dr. Temple and her colleagues discovered that adult eyes already contain stem cells that can be used to make new retinal pigment epithelial cells, in order to effectively treat the currently incurable disease. Thanks to NYSTEM funding, she’s preparing to launch the first clinical trials to transplant this patented adult retinal stem cell.
Michal K. Stachowiak, PhD
Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, Professor of Pathology and Anatomical Sciences
Symptoms of schizophrenia usually appear in adolescence or young adulthood, but new research by Dr. Stachowiak and colleagues reveals that the brain disease likely begins much earlier, toward the end of the first trimester of pregnancy. The finding expands our understanding of this devastating disease and opens up the potential for early detection and treatment in utero. The research uses recently developed technology called cerebral organoids, in which stem cells are grown into “mini-brains” that resemble the developing human brain in its earliest stages.
Todd Evans, PhD
Weill Cornell Medicine, Associate Dean for Research, Peter I. Pressman, M.D. Professor in Surgery
In 2017, Dr. Todd Evans and his colleagues published pioneering research on their development of the first-ever human tissue platform to test drugs for colon cancer. Using a “disease in a Petri dish” model derived from stem cells, his team identified a targeted drug treatment for a common, inherited form of the disease. This technique of screening drugs using cultivated cells with certain mutations is at the forefront of precision medicine, developing targeted treatments for patients’ individualized cases. It also has the potential to change the future of healthcare from one-size-fits-all treatment to personalized treatment strategies.
NYU Langone Medical Center, William Goldring Professor of Medicine; Director, Leon H. Charney Division of Cardiology
Sudden cardiac arrest kills 300,000 Americans every year. That’s why Dr. Fishman and his colleagues are using stem cells to study the electrical activity that keeps the heart beating. They do this by directing stem cells to develop into the specific subtypes of heart cells to study, which allows the researchers to better understand the specialized heart cells at a genetic level, and look into why they fail. The ultimate goal is that this research will lead to improved treatment to keep the heart beating in rhythm, up to a few billion beats over a lifetime.
University of Rochester Medical Center, Professor of Genetics and Neuroscience
Director, UR Stem Cell and Regenerative Medicine Institute
For families facing diagnoses of lysosomal storage disorders – a group of about 50 devastating inherited diseases affecting a patient’s enzymes, including Krabbe, Gaucher and Tay-Sachs diseases – there are virtually no useful treatments. Dr. Noble’s lab is developing new therapies, in the hopes of reducing the tremendous cost of these degenerative diseases to the patients, their families and the health care system at large. NYSTEM-funded research has already led to the discovery of entirely novel approaches to treating some of these disorders, by finding new therapeutic avenues and uncovering previously unsuspected properties of existing drugs. Dr. Noble is also exploring the application of this research to Parkinson’s Disease.