Noninvasive Avenue for Parkinson's Disease Gene Therapy
Apr. 21, 2013 — Researchers at Northeastern University in Boston have developed a gene therapy approach that may one day stop Parkinson's disease (PD) in it tracks, preventing disease progression and reversing its symptoms. The novelty of the approach lies in the nasal route of administration and nanoparticles containing a gene capable of rescuing dying neurons in the brain. Parkinson's is a devastating neurodegenerative disorder caused by the death of dopamine neurons in a key motor area of the brain, the substantia nigra (SN). Loss of these neurons leads to the characteristic tremor and slowed movements of PD, which get increasingly worse with time. Currently, more than 1% of the population over age 60 has PD and approximately 60,000 Americans are newly diagnosed every year. The available drugs on the market for PD mimic or replace the lost dopamine but do not get to the heart of the problem, which is the progressive loss of the dopamine neurons.
The focus of Dr. Barbara Waszczak's lab at Northeastern University in
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Boston is to find a way to harvest the potential of glial cell line-derived
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neurotrophic factor (GDNF) as a treatment for PD. GDNF is a protein
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known to nourish dopamine neurons by activating survival and growth-
0
promoting pathways inside the cells. Not surprisingly, GDNF is able to
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protect dopamine neurons from injury and restore the function of damaged and dying neurons in
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many animal models of PD.
Parkinson 'S
However, the action of GDNF is
Brain Injury
limited by its inability to cross the
Stem Cells
blood-brain barrier (BBB), thus
Cancer Research
requiring direct surgical injection into the brain. To circumvent this
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problem, Waszczak's lab is investigating intranasal delivery as a
Health & Medicine
way to bypass the BBB. Their
Parkinson's
previous work showed that
Research
intranasal delivery of GDNF protects
Nervous System
dopamine neurons from damage by
Gene Therapy
the neurotoxin, 6-hydroxydopamine
Mind & Brain
(6-OHDA), a standard rat model of
Disorders and
PD.
Syndromes
Taking this work a step further,
Brain Injury
Brendan Harmon, working in
Parkinson's
Waszczak's lab, has adapted the
Reference
intranasal approach so that cells in
Sensory neuron
the brain can continuously produce
Dementia with Lewy
GDNF. His work utilized
bodies
nanoparticles, developed by
Dopamine
Copernicus Therapeutics, Inc., which
hypothesis of
are able to transfect brain cells with
schizophrenia
an expression plasmid carrying the
Astrocyte
gene for GDNF (pGDNF). When given intranasally to rats, these pGDNF nanoparticles increase GDNF production throughout the brain for long periods, avoiding the need for frequent re-dosing. Now, in new research presented on April 20 at 12:30 pm during Experimental Biology 2013 in Boston, MA, Harmon reports that intranasal administration of Copernicus' pGDNF nanoparticles results in GDNF expression sufficient to protect SN dopamine neurons in the 6-OHDA model of PD.
Waszczak and Harmon believe that intranasal delivery of Copernicus' nanoparticles may provide an effective and non-invasive means of GDNF gene therapy for PD, and an avenue for transporting other gene therapy vectors to the brain. This work, which was funded in part by the Michael J. Fox Foundation for Parkinson's Research and Northeastern University, has the potential to greatly expand treatment options for PD and many other central nervous system disorders. Share this story on Facebook, Twitter, and Google:
New findings !!
Getting stuff across the BBB & exactly where you want it in the brain - through the nose, with ultrasound (in mice)
