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.