NEWYORK, NY – The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced today that it has awarded grants totaling nearly $1 million to four researchers through its Cell Line II program, “Cell Replacement Therapy: Developing Dopaminergic Cell Lines With Long-Lasting Functional Impact in Transplant Models of Parkinson’s Disease.”
The program seeks to address current obstacles in achieving the therapeutic potential of cell replacement, including poor survival and function of stem cell-derived dopaminergic neurons after transplantation into the brain. Leading researchers have identified cell replacement therapy as one of the most promising potential treatments for Parkinson's, capable of halting disease progression and even restoring lost function.
“Focused work in this area is an essential next step for stem cell research in Parkinson’s disease,” said MJFF president and CEO Deborah W. Brooks. “We’ve made cell transplantation a top priority because we view it as a high-potential avenue of Parkinson’s disease research that is currently underfunded by the federal government.”
In 2002, the Michael J. Fox Foundation launched its first cell line initiative focused on generating a dopamine-producing cell line, which represented the next critical step in advancing the field.
With this support in place, several scientists were able to isolate and grow the cells in the laboratory, as well as to induce differentiation into dopamine neurons. However, the cells quickly lost their dopaminergic characteristics and had overall poor viability after transplantation into animal models of Parkinson’s disease.
Some of the research funded under the Cell Line II program will seek to better understand the molecular mechanisms that regulate the generation of dopaminergic neurons and to map the complex cell signals that direct stem cell differentiation and maturation (into dopaminergic neurons). The next step will be to test the approach in animal models of Parkinson’s.
One grant recipient will try to replicate earlier success of transplanting monkey embryonic stem cells into monkeys, instead using human embryonic stem cells. If long-term viability of the transplanted cells is achieved, the researchers hypothesize that their strategy will work in humans.
Another project will take a novel approach to minimize the immune or inflammatory response that results from implanting stem cells. The transplantation site essentially will be prepped to create a scar-free space that is ready to host subsequent transplanted cells. The hope is that this strategy will improve the likelihood of neuronal survival.
“The manipulation of stem cells into dopamine neurons that survive and flourish after transplantation could have tremendous therapeutic impact on the lives of Parkinson’s patients,” said J. William Langston, MD, chief scientific advisor to the Foundation and CEO of the Parkinson’s Institute. “Additionally, such modified stem cells would provide an invaluable research tool which would allow scientists to truly investigate the cause, prevention and improved treatment of the disease.”
The following is a complete list of researchers who were awarded grants for the Cell Line II initiative:
Ernest Arenas, MD, PhD
Novel Protocols for the Differentiation & Functional Integration of Embryonic & Neural Stem Cell-derived Dopaminergic Neurons in Rodent Models of PD
Jun Takahashi, MD, PhD
KyotoUniversityGraduateSchool of Medicine
Transplantation of Primate ES Cell-Derived Dopaminergic Neurons Into the Brains of MPTP-treated Monkeys
Xuejun Wen, MD, PhD
Improving the Long-term Survival and Functionality of the Transplanted Human Dopaminergic Neurons Based Upon a Novel Tissue Engineering Approach
Su-Chun Zhang, MD, PhD
WaismanCenter, University of Wisconsin-Madison
Combined Stem Cell Transplant and Growth Factor Therapy for Parkinson’s Disease