Public and Private Funds Working Together to Support Stem Cell Research

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The Parkinson Alliance pioneered the private support awarded to Dr. Ron McKay in 1998. The $180,000 of private funds were leveraged into a multi-million dollar study at the National Institutes of Health for stem cell research. Stem cells, which are self-renewing elements that can generate many cell types in the body, may one day be harnessed for tissue repair in degenerative disorders such as Parkinson's, heart disease, and diabetes.

In a letter to Martin Tuchman (Chairman of the Board of The Parkinson Alliance) dated May 11, 2001, Dr. McKay states, "Stem cell biology is one of the most exciting areas of bio-medical research." As a result of this research, two major advances in the concept of using embryonic stem cells to regenerate human tissues were recently reported in major newpapers and scientific journals. In one report, biologists at the NIH used mouse embryonic stem cells to generate insulin-producing organs resembling the islets of the pancreas, an accomplishment that holds potential for treating Type 1 diabetes. In a second report, biologists proved therapeutic cloning works in mice by cutting off a small portion of the tails of mice and converting the tails' skin cells into embryonic stem cells. From those stem cells, the NIH’s colleagues at the Memorial Sloan-Kettering Cancer Center then changed them into dopamine-producing cells of the brain that are lost in Parkinson's disease.

"In animal studies, the transplanation of cells into the diseased or injured brain shows that both cellular repair and behavioral recovery are possible," states McKay. While animal studies and the limited use of cell transplant technology in human trials has revealed the potential for behavioral repair, there is a greater "need for a much more detailed understanding before stem cell therapy can be a viable therapeautic option for Parkinson’s patients."

He continues, "For example, what controls cell differentiation pre- and post-transplantation? What factors allow cells to survive and integrate into host tissue? What controls synapse formation and dopamine release? To answer these questions, we need a routine source of dopamine neurons. In the last year, we have demonstrated that embryonic stem (ES) cells can generate unlimited numbers of dopamine neurons. We have also used ES cells to generate functional pancreatic islets. In another study we have regenerated heart muscle in a model of a heart attack. These experiments unequivocally demonstrate that stem cell technology has clinical potential across many areas of medicine.

Although stem cell technology is often seen as the basis for cell transplants, information derived from stem cells will be critical for any new therapy. For example, the potential use of gene therapy to control the survival of dopamine neurons will be greatly enhanced by a detailed understanding of the action of these gene products on dopamine neurons. So stem cell technology and gene and gene delivery technology are not in opposition, but are complementary strategies. Recent work in gene and cell therapy provides a strong foundation for further advances in understanding and treating Parkinson’s disease."

Funds provided by The Tuchman Foundation (the backbone of The Parkinson Alliance) supports scientists and clinicians working on several applications of stem cells. McKay concludes, "Access to private support is an important mechanism, giving flexibility that complements the more restricted uses permited for government funds. This combination of federal and private support has generated a research program that is recognized as world leading in Parkinson’s disease, diabetes, and heart disease."

For more information on how The Tuchman Foundation is the backbone of The Parkinson Alliance, see our About Us page. For more information on the National Institutes of Health, contact its Web site.

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