The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced today the launch of a new program designed to advance the field of cell replacement therapy in Parkinson’s disease. The initiative, “Cell Replacement Therapy: Developing Dopaminergic Cell Lines with Long-Lasting Functional Impact in Transplant Models of Parkinson’s Disease” follows upon the Foundation’s groundbreaking 2002 cell line effort. The effort is funded, in part, through a major gift from an anonymous donor.
“It is clear that human embryonic stem cells can produce abundant populations of dopaminergic neurons in the lab,” said J. William Langston, MD, chief scientific advisor to the Foundation and CEO of the Parkinson’s Institute. “Yet preliminary work shows that issues of long-term cell survival, loss of functionality and even cell overgrowth continue to hamper the success of transplants as therapy. This program seeks innovative solutions to remove these roadblocks,” Dr. Langston stated.
The program will focus on projects that develop methods to:
- improve the functional characteristics of new dopaminergic neuron cell lines, or improve differentiation of existing cell lines in a way that improves transplant outcomes;
- facilitate functionality or connectivity of transplanted cells, and
- prevent deregulated cell growth resulting from transplant.
The Foundation seeks to encourage highly innovative solutions, as well as proposals targeting the practical aspects of rapid and efficient clinical translation. All projects will focus on embryonic stem cell lines, working with either NIH- or non-NIH approved lines. Cell lines must be of a uniform type, free of contamination and potentially appropriate for use in humans, as defined by the U.S. Food and Drug Administration (FDA).
“As a Foundation committed to curing Parkinson’s, we believe it is our job to take the next step on the pathway toward cell replacement as a viable patient therapy,” stated Deborah W. Brooks, MJFF executive director.
Two years ago, the Foundation awarded more than $4 million to nine researchers to pursue the development of a cell line specifically designed to advance the study and treatment of Parkinson’s disease. The grants awarded covered several research avenues: from human adult to fetal cells and embryonic stem cells. Results from that program indicate that given current technology, embryonic stem cells are the most promising for use in cell replacement therapy for Parkinson’s disease.