News Releases
IGERT sponsored research leads to MJFF grant
Date: 01/18/2008
The following article was featured in the January 10 2008 edition of The Northeastern Voice.
By Susan Salk
A pharmacology professor's attempt to breathe new life into the battle against Parkinson's disease has attracted the attention of sufferer and activist Michael J. Fox.
On Dec. 1, the Michael J. Fox Foundation for Parkinson's Research awarded Barbara Waszczak a $75,000 Rapid Response Innovation Award for her work on an intranasal treatment for Parkinson's disease, a degenerative disorder of the central nervous system which is characterized by impairment of motor function and speech.
Waszczak's work will focus on a novel method for delivering the therapeutic protein GDNF (glial cell line-derived neurotrophic factor) to a region of the brain affected by the disease. Previous attempts at GDNF therapy required highly invasive methods today deemed too high-risk, she explained.
Instead of relying on direct delivery of GDNF into the brain via injections, Waszczak hopes her research may bring about the development of a nasal treatment that would be so safe and easy to use that a patient could self-administer the treatment at home.
The goal is to deliver the GDNF factor to the brain's nigostriatal pathway, which contains dopamine, a neurotransmitter essential for normal movement. As the dopamine neurons degenerate in Parkinson's, dopamine levels fall and motor symptoms emerge, such as slowness of movement, rigidity and tremor. GDNF has long been known to rescue and protect these damaged dopamine neurons, but the challenge has been getting it into the brain.
Intranasal drug delivery has been effective with other medications, but this is the first time the concept has been applied to Parkinson's disease treatment, she said.
"To date, there have been no attempts to administer GDNF by this route, or to evaluate its efficacy in a model of Parkinson's disease," Waszczak wrote in her grant application to Fox, the former film and TV actor.
In her application to the Fox foundation, Waszczak struck a hopeful note: "Such a strategy could afford a means of arresting disease progression in early-stage Parkinson's disease, and provide a therapeutic option for those not candidates for surgical approaches."
She credited pharmacy professor Mansoor Amiji with the idea of intranasal delivery, and assistant pharmacy professor Robert Campbell with his design of a novel "cationic liposomal" delivery system in the effort, which, she added, got a major boost with the grant.
"One thing that's so exciting about the Michael J. Fox grant is that it will help us leverage our work to apply for more research funding in the future, either through Fox or the NIH (National Institutes of Health)."
Noting that the Fox Foundation is "very goal-directed in what they fund," she added that she needed to show that intranasal delivery already held promise and "utility."
Her earlier work with intranasal delivery of ovalbumin, a protein derived from chicken egg whites, helped prove the merits of what she is attempting here, Waszczak said. "We were very successful in showing that intranasally administered ovalbumin entered the brains of rats," she said.
She is hoping for the same success with GDNF. "It has been known that GDNF has the potential to treat Parkinson's disease, but nobody has been able to convert that potential to reality," she said.
Waszczak came to Northeastern in 1983, after working five years at NIH. She received her doctorate in pharmacology in 1978 from the University of Michigan.
Throughout her professional career, Waszczak has focused her research on dopamine system neuropharmacology and disorders of the brain's basal ganglia with special interest in Parkinson's disease.
She has more than 50 publications, and has mentored five doctoral students. In this latest research, she said, the work of Ph.D. candidate Mattia Migliore and undergraduate Robin Ortiz has been invaluable.
She decided to pursue a career in academia, rather than "working in industry," for the freedom to pursue research projects like this, she said.
http://www.northeastern.edu/voice/pdfs/080110/080110.pdf