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Researchers reverse Parkinson's damage with nasal gene delivery

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Researchers at Northeastern University have developed a treatment for Parkinson's disease meant to revive dying neurons in the brain via delivery through the nose.

The Boston researchers were able to treat the symptoms of Parkinson's--a decrease of motor function that results in tremors and slowed movements--with a protein called glial cell line-derived neurotrophic factor, or GDNF. The protein acts directly on the motor area of the brain, the substantia nigra, preventing the degeneration and death of the neurons there. Other drugs mimic or replace lost dopamine in the brain, according to a report in Business Standard, but the GDNF protein stops the degenerative quality of the disease.

However, GDNF doesn't easily cross the blood-brain barrier, so the therapy called for a new drug delivery method. And rather than surgically placing the proteins in the brain, the scientists opted for intranasal delivery, allowing direct transport of the GDNF to the necessary area.

What's more, the team, led by Barbara Waszczak and Brendan Harmon, concocted a genetic treatment that uses the same delivery method. Using nanoparticles developed by Copernicus Therapeutics, they delivered not only the GDNF but also a gene that codes for the protein into the brain, enabling the production of GDNF and allowing more time between doses. In studies on rats, the new gene protected the dopamine neurons and tempered the symptoms of Parkinson's, according to the report.

The Michael J. Fox Foundation and Northeastern University both funded the research, which Waszczak and Harmon presented this week at the Experimental Biology 2013 conference in Boston.

- here's the Business Standard report

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