The NYSCF scientific team is leveraging recent advances in stem cell research to study multiple sclerosis through the creation of disease-specific stem cell lines

For patients with MS, the cells of the immune system attack and destroy myelin, a protein that normally insulates nerves. Multiple sclerosis There are about 2.5 million people worldwide with MS and estimated that 400,000 in the United States.

Multiple sclerosis is very difficult to study in people. Using embryonic stem cells and human iPS cells, disease models “in a dish” can recapitulate what happens in the central nervous system and provide insights into how to treat this disease.

NYSCF-Helmsley Investigator Dr. Valentina Fossati is modeling multiple sclerosis (MS) in the NYSCF lab using patient-derived pluripotent stem cells. These cell lines will enable NYSCF to overcome the fundamental challenges that have kept scientists and clinicians from fully understanding this devastating autoimmune disorder. This greater understanding will in turn lead to the discovery of cell-based treatments and new drugs for MS patients.

Stem cell research holds the promise for scientists to overcome one of the most challenging problems in studying human disease: the fact that by the time the disease is diagnosed, many of the events that led to it have already occurred, preventing scientists from understanding its precise origin. It also gives us an unprecedented opportunity to create the cells needed for cell replacement therapy and for screening drugs on the actual cells that are getting sick in diseases.

The problem encountered by scientists attempting to study disease or injury is analogous to that faced by investigative teams trying to understand the causes of a plane crash before the advent of the “black box,” when understanding the often subtle events that led to the incident was nearly impossible. The cell-based models of disease created in the NYSCF laboratory will serve as “data recorders” for the study of disease. Just as flight recorders allow the Federal Aviation Administration to replay air accidents over and over again to understand the underlying causes of a crash, these cell models will enable us to repeatedly replay the development of disease until it is fully understood.

Scientific research has been further hampered by the inability to obtain adequate numbers of the actual type of cells needed for research in specific diseases or other medical conditions. As a result, scientists have traditionally turned to the study of rodent models, which often poorly recapitulate human disease. NYSCF scientists have shown that human pluripotent stem cells carrying the genes causing a specific disease can replicate many of the degenerative processes that occur in humans with that disease. The ability of these cells to grow indefinitely in culture, while retaining the capacity to differentiate into all the cells of the body, will allow us to produce a limitless quantity of these degenerating cells for use in disease studies and drug identification.