Study Promises Better Drug Delivery to Fight ALS

(HealthDay News) -- Drugs to treat amyotrophic lateral sclerosis (ALS) can be delivered to the brain and spinal cord through cerebrospinal fluid, U.S. researchers report.

Working with rats, researchers delivered therapeutic molecules called antisense oligonucleotides to the brain and spinal cord through cerebrospinal fluid. They were able to do so at doses that slowed the progression of ALS, also known as Lou Gehrig's disease.

This method may provide an alternative to trying to deliver drugs via blood across the highly impermeable blood-brain barrier, which prevents substances in the blood from entering brain tissue, explained researchers at the University of California, San Diego.

The study was published online Thursday in the Journal of Clinical Investigation and was expected to be in the August print issue.

People with neurodegenerative diseases such as ALS, Alzheimer's disease, Parkinson's disease, and Huntington's disease have a buildup of toxic proteins in the brain and central nervous system.

A number of different approaches for removing these excess proteins have been tried, but few have been successful, due to the blood-brain barrier, the researchers said.

Injecting drugs into cerebrospinal fluid, which circulates from the brain to all areas of the central nervous system, may be an effective way of treating the buildup of toxic proteins associated with neurodegenerative diseases, they said.

More information
The Muscular Dystrophy Association has more about ALS.