Page 43 - Gates-AnnualReport-2018

Parkinson’s pioneer researcher Curt Freed, M.D. received funding for a clinical trial and student stipend from the Glendorn Foundation.
GLENDORN FOUNDATION SPURS RESEARCH AND ENABLES INTERNSHIP PROGRAM
Four years ago, a gift from the Glendorn Foundation toward the emerging field of ocular stem cell research helped kick off the Gates Center’s collaborative effort with the Depart- ment of Ophthalmology to raise $10 million for the launch of Cellsight, the ocular stem cell program. In 2017, a subse- quent gift from the foundation in support of research and stipends for Gates Center interns helped fuel the continued growth of this program, as well as training of the next gener- ation of scientists and clinicians under the charismatic lead- ership of world-class researcher Valeria Canto-Soler, Ph.D., and her colleagues.
Similar to previous efforts to kick off new research and to enable a Gate Center intern to be involved in cutting-edge investigations, the Glendorn Foundation awarded a grant in 2018 to Curt R. Freed, M.D. (see photo). The grant was imme- diately matched by Mrs. Hoehle, a long-standing supporter of Dr. Freed’s work, to initiate a study to see if a simple fatty acid sold as a food supplement had the potential to stop the progression of Parkinson’s disease. The fatty acid is named butyrate. The idea stemmed from Dr. Freed’s 2011 discovery that a related drug called phenylbutyrate could keep Parkin- son’s from developing in mice that had been genetically pro- grammed to get the disease. Adding phenylbutyrate to the animals’ drinking water starting in mid-life, before Parkin- son’s developed, stopped the disease from ever occurring. Dr.
Freed’s group found that the drug worked by turning on a pro- tective gene called DJ-1. Higher DJ-1 activity in all brain cells prevented deposits of abnormal proteins. Instead of forming deposits in the brain, misfolded forms of the alpha-synuclein protein were pushed out into the bloodstream, where they could be eliminated. Testing the same drug in people with Parkinson’s disease showed the same benefit.
Because phenylbutyrate is an expensive drug, Dr. Freed’s group tested similar but cheaper alternatives. They dis- covered that the fatty acid butyrate was just as effective as phenylbutyrate. The aim of the project funded by the Glen- dorn Foundation and Mrs. Hoehle has been to see if butyrate taken orally by people with Parkinson’s disease will lead to an increase in the plasma concentration of the alpha-synu- clein protein. While the study is not yet complete, prelimi- nary evidence indicates that butyrate is working in people as it had in mice.
Success in these human experiments could lead to larger hu- man clinical trials to see if butyrate can stop the progression of Parkinson’s disease. The food supplement is more than 100 times less expensive than phenylbutyrate. If butyrate can stop Parkinson’s, it would be a cheap and widely available drug to prevent this disabling disease and a major contribu- tion to American medicine.
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