The NYSCF scientific team leverages recent advances in stem cell research to study multiple sclerosis through the creation of disease-specific stem cell lines.
NYSCF-Helmsley Investigator Valentina
Fossati speaking at the 2010
Druckenmiller Fellows Reception
For patients with multiple sclerosis (MS), the cells of the immune system attack and destroy myelin, a protein that normally insulates nerves. There are about 2.5 million people worldwide with MS and an estimated 400,000 MS patients 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” recapitulate what happens in the central nervous system and can provide insights into how to treat this disease.
NYSCF - Helmsley Investigator Dr. Valentina Fossati is modeling multiple sclerosis in the NYSCF lab with 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 will in turn lead to the discovery of cell-based treatments and new drugs for MS patients.
Why Stem Cells?
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.
Recent Multiple Sclerosis Research:
“Multiple sclerosis in 2010: Advances in monitoring and treatment of multiple sclerosis
Currently, MS patients can only choose between drugs that are administered by injection. The new medications represent a new generation of treatment for MS, as they can be taken orally. Gilenya (fingolimod) is the first oral medication on the market designed to reduce relapses and slow down the progression of the disease. Dalfampridine, an extended-release drug designed for walking improvement, has been approved based on a double-blind trial with MS patients. Furthermore, Laquinimod, a novel oral immunomodulary drug, has been developed to reduce MRI lesions in MS patients.
“A reversible form of axon damage in experimental autoimmune encephalomyelitis and multiple sclerosis”
Using in vivo imaging, researchers have shown that axon demyelination is not a prerequisite for axon damage. This suggests that inflammatory axon damage and the death of oligodendrocytes might be spontaneously reversible and thus a potential target for therapy. These results also show that neurodegeneration itself, rather than the traditional damping of the immune system, could be a new target for therapy
“Multiple Sclerosis and Vitamin D: A Review and Recommendations”
A relationship between vitamin D and several diseases, including multiple sclerosis, has received interest in the scientific community. Risk of development of MS, as well as disease severity, has been associated with vitamin D deficiency in a variety of studies.
“Chronic Cerebrospinal Venous Insufficiency in Patients with Multiple Sclerosis”
A theory introduced in 2008 suggests that pathological venous flow is the original cause (or has a strong correlation with) the severity of multiple sclerosis. While the early findings suggest that MS can possibly be treated with a liberation procedure, the research is still in its preliminary stages; therefore the only safe route to explore this branch of research is through the several clinical trials going on in America and Europe that should yield results in one to two years.