About 764,000 children and adults in the U.S. have cerebral palsy, according to the CDC. The difficulty in getting drugs across the blood-brain barrier has been vexing scientists trying to treat the condition. But new research from NIH and the Wayne State University School of Medicine could hold promise for the early detection and treatment of CP.
Researchers from NIH's Perinatology Research Branch, housed at Wayne State and the Detroit Medical Center, believe using a nanodevice to deliver drugs to the brain could be the key to preventing the neuroinflammation that leads to CP. And they've got early results from animal trials to back that up.
The scientists loaded tiny nanodevices with a dendrimer coupled with the anti-inflammatory compound N-acetyl-L-cysteine (D-NAC). They administered the treatment to newborn rabbits with CP, releasing the D-NAC once the nanodevices had crossed the blood-brain barrier. And the results they got were thrilling: Rabbits treated with the D-NAC showed dramatic improvement in motor skills on the first day. By day 5, they were able to walk and hop, unlike those given a placebo.
"The key finding of this work is that early identification of neuroinflammation allows postnatal treatment," said Roberto Romero, chief of the Perinatology Research Branch of the NIH. "This suggests that there is a window of opportunity to prevent cerebral palsy."
The effectiveness of the delivery method is two-pronged, the researchers said. First, the nanodevice allows for the transport and delivery of the compound into the brain. Then, the dendrimer in the compound helps pinpoint inflamed cells in the brain, allowing the NAC to reach its target.
The researchers also pointed out the delivery method could be useful in fighting other neurological disorders, including autism and Alzheimer's disease. NAC is in clinical trials to test its potential as an Alzheimer's treatment.
The researchers published their results in Science Translational Medicine.
- read the release from NIH
- here's the journal article