Bernard Spitzer, a civil engineer and real estate developer, will give Columbia $8 million to support a stem cell research program to develop new treatments and therapies for Parkinson's disease and other neurological disorders. The gift will establish the Bernard and Anne Spitzer Fund for Cell and Genetic Therapy at Columbia.
"Parkinson's disease is one of the best examples of a disease well suited to stem cell-based therapy," says Gerald Fischbach, executive vice president for health and biomedical sciences and dean of the Faculty of Medicine. "This gift will enable our researchers to advance the effort to halt the progression of Parkinson's disease and, in doing so, translate what they've learned to other neurological disorders such as Alzheimer's disease and ALS, and other conditions such as type 1 diabetes."
"We are delighted to provide a great research institution like Columbia with the means to make substantial progress in this important area of research," says Spitzer.
Parkinson's disease is a disorder of the central nervous system that affects more than one million Americans. Symptoms of the disease result predominantly (but not exclusively) from the loss of a single population of brain cells, the dopamine neurons situated in the midbrain. Scientists have developed cell-based therapies over the past two decades that replace the lost dopamine for Parkinson's disease and have evidence that the therapy might work. In a recent study published in the journal Nature, scientists from the National Institutes of Health isolated stem cells from mouse embryos, which developed into brain cells in a rodent model of Parkinson's disease. The cells formed new connections and reduced symptoms of the disease in the animal. However, the complex nature of the human brain means more research must be done before cell replacement strategies are translated into clinically effective remedies.
"Our challenge is to optimize the conversion of stem cells into functional, appropriately connected dopamine neurons," says Stanley Fahn, H. Houston Merritt Professor of Neurology and director of the Center for Parkinson's Disease and Other Movement Disorders. "The Spitzer gift will greatly assist us in achieving this goal more quickly."
Stem cells, derived either from early embryos (embryonic stem cells) or from the adult brain (neuronal stem cells), are primitive or "immature" cells that have the potential for developing into functional, appropriately connected dopamine neurons. Although stem cells are a promising source of future cell-based therapies, scientists need to overcome the challenges of producing adequate numbers of the right kind of cells and the ultimate survival of the replacement cells.
The Bernard and Anne Spitzer Fund for Cell and Genetic Therapy will allow researchers to develop mouse embryonic and neuronal stem cell lines that produce dopamine neurons that remain "off" during the stem cell stage but are turned "on" by dopamine once the neuron has matured. This experimental process will allow researchers to use cell cultures to characterize the development, function, and survival of stem cell-generated dopamine neurons. Using genetically modified mouse models of Parkinson's disease, researchers will be able to perform and study dopamine neuron transplants generated from the mouse stem cells. Eventually, what researchers learn from the animal models will be translated to humans using human embryonic and neuronal stem cell lines.
"We are deeply committed to stem cell research because of its promise for developing revolutionary new and effective treatments for Parkinson's disease and other neurodegenerative diseases that are currently untreatable," says Timothy A. Pedley, Henry and Lucy Moses Professor and chairman of neurology. "We look forward to the collaborative efforts a gift like this will make possible and which are essential if we are to translate hope into reality."
Columbia has emerged recently as a leader in stem cell research. In April, Columbia established the Charles and Jean Brunie Fund for Cell Therapy in Brain Disease with a $3 million gift from investment banker Charles Brunie.
Fischbach is forming a Neural Stem Cell Scientific Advisory Committee to oversee the research, which will be chaired by Thomas Jessell, professor of biochemistry and molecular biophysics, who is recognized worldwide as a preeminent scholar in the field of nerve cell development.