UI Scientists Use Blood Brain Barrier as Therapy Delivery System

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By Aaron Hepker

Story Created: Sep 26, 2009 at 2:49 PM CST

Story Updated: Sep 26, 2009 at 2:49 PM CST

IOWA CITY — University of Iowa researchers have discovered a way to turn blood vessels surrounding brain cells into a production and therapy delivery system.

The blood brain barrier is generally considered an obstacle to delivering therapies from the bloodstream to the brain.

Working with animals of a group of fatal neurological disorders called lysosomal storage diseases, the UI team found the diseases cause unique and disease-specific alterations to the blood vessels of the blood brain barrier.

The scientists used the distinct alterations to target the brain with gene therapy, which reversed the neurological damage caused by the diseases.
Findings, published Sept. 13 in Nature Medicine’s Advance Online Publication, could lead to a new approach for treating neurological damage caused by lysosomal storage diseases.

“This is the first time an enzyme delivered through the bloodstream has corrected deficiencies in the brain,” said lead investigator Beverly Davidson, UI professor of internal medicine, neurology, and molecular physiology and biophysics. “This provides a real opportunity to deliver enzyme therapy without surgically entering the brain to treat lysosomal storage diseases.”

The study was funded by grants from the National Institutes of Health and from the Batten Disease Support and Research Association.

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