The Michael J. Fox Foundation for Parkinson’s Research has awarded a $187,500 grant to a Northeastern pharmacologist to further study a new treatment against Parkinson’s disease. With her colleagues, Professor Barbara Waszczak developed the idea to use an intranasal delivery method to deliver a protein called GDNF (glial cell line-derived neurotrophic factor) to the brain, which, in previous studies, has shown promise as a means to stop the progression of the disease in early stages.

GDNF has long been known to rescue and protect dopamine neurons, which degenerate as Parkinson’s progresses, but researchers have not yet successfully introduced GDNF into the brain. GDNF cannot be given via a systemic route of administration since it does not pass the blood-brain barrier. Previous attempts to deliver the protein via injections to the brain have proven ineffective.

“Intranasal drug delivery has been widely used to treat many other diseases, and we are tapping into that potential for Parkinson’s,” said Waszczak, professor of pharmacology in Bouvé’s College of Health Sciences at Northeastern. “This therapeutic method, if proven safe, could benefit millions of people worldwide with the disease.”

Waszczak received a grant from The Michael J. Fox Foundation in 2007 to investigate the efficacy of delivering this protein through the nose, and the findings have proven positive. When given intranasally, GDNF reaches the brain in sufficient quantities to protect dopamine neurons from an experimental neurotoxin. The next steps, funded by this new grant, will allow Waszczak to determine if this delivery method results in nasal toxicity and how much GDNF actually reaches the target area of the brain.

If the findings in this new project prove safe and effective in lab animals, the intranasal delivery of GDNF could possibly translate into human clinical trials.

“Being able to bring this treatment potential to reality for people with Parkinson’s is the ultimate goal of this research project,” added Waszczak.

Parkinson’s disease is a degenerative disorder of the central nervous system characterized by the impairment of motor function. As the disease progresses, dopamine neurons degenerate, resulting in low dopamine levels. Disease symptoms, including slowness of movement, rigidity and tremors, emerge as this degeneration occurs, and symptoms worsen over time with or without treatment.

Robert A. Schatz, associate professor of pharmacology and toxicology at Northeastern, and Mattia M. Migliore, assistant professor of pharmacology and toxicology at Massachusetts College of Pharmacy and Health Sciences, are co-principal investigators on this project.

This research was possible with the generous donation of GDNF from Amgen.