NEWYORK, NY — The Michael J. Fox Foundation for Parkinson’s Research today announced the launch of a new funding program, Improving Delivery of Parkinson’s Disease Therapeutics to the Brain. This $2-million initiative aims to stimulate research addressing two major challenges to the development of transformative treatments for Parkinson’s disease. The first challenge is the need for non-invasive drug delivery technologies allowing therapeutics to cross the blood-brain barrier. The second challenge is the need to more precisely understand how molecules move inside the central nervous system in order to target treatments to specific regions of the brain.
The blood-brain barrier, a thin membrane of tightly packed cells, creates a wall between most parts of the brain and the rest of the circulatory system. While this barrier is critical for protecting the brain by keeping out bacteria and other harmful agents, it also prevents entry by therapeutic molecules that could potentially impact the symptoms and progression of PD and other neurological disorders. Research is required to find new delivery technologies that would safely and reliably allow drugs to pass into the brain.
Even when a molecule of interest is able to pass the blood-brain barrier, researchers face an enormous obstacle in targeting the treatment to the proper area of the brain. The Foundation therefore aims to spark research to improve understanding of the movement and diffusion of molecules inside the brain.
“Advances in molecular biology and our understanding of Parkinson’s disease continue to open new therapeutic avenues for investigation,” said Katie Hood, CEO of The Michael J. Fox Foundation. “But every therapeutic, no matter how promising, currently faces one major challenge: how do we get it into the targeted region of the brain, at appropriate levels, in a controlled and reproducible fashion? Improving on this status quo would be a major leap forward in the discovery of life-changing treatments for Parkinson’s disease.”
The difficulties posed by the blood-brain barrier affect not only “small-molecule” therapeutics — the chemical compounds that make up the majority of drugs brought to market by big pharma — but also create an obstacle to the delivery of “large-molecule” treatments involving the use of proteins, including trophic factors such as GDNF, and other biologics being developed by biotechs. Genetic material, used in gene silencing or gene therapy approaches, also cannot cross the blood-brain barrier and currently can be delivered only via surgical intervention.
Molecular size may also affect movement inside the brain, where molecules face an obstacle course of both physical impediments and chemical limitations that impact their ability to get where they need to go. Factors that may be at work include brain vasculature (arrangement of blood vessels), the structure of the brain’s white matter (the tissue through which messages pass between different areas within the nervous system) and even brain changes brought on by Parkinson’s disease itself.
This program is open to academic and industry investigators alike for research projects of up to two years’ duration. Pre-proposals will be reviewed by the Foundation’s scientific staff and a panel of scientific experts and must be submitted online by May 15, 2008. Information about submitting pre-proposals online can be found on the Foundation’s Web site (www.michaeljfox.org). Applicants whose pre-proposals meet the review criteria will be invited to submit full proposals. Funding is anticipated by October 2008.
A conference call with MJFF Research Programs staff to further clarify the aims and goals of this initiative will be held April 7, 2008, at 12 p.m. U.S. Eastern Time. Researchers wishing to participate in the call must RSVP to email@example.com and will receive an e-mail reply with call-in details.