In the long, difficult struggle to understand--and do something about--the brain ailment called Huntington's disease, scientists have decided the best approach may be to go fishing.
The target is a strange jellyfish that has a natural ability to glow in the dark when pestered, showing its irritation in eerie green light.
The glow, they hope, will lead toward a cure for Huntington's disease, a fatal brain disorder first noted among people living on the eastern tip of Long Island, N.Y., in 1872 by Dr. George Huntington. It was the first completely dominant genetic disease--meaning anyone who inherits the faulty gene gets the disease--described. It has always been untreatable.
In the new research, the glowing protein taken from the jellyfish will become a marker, allowing scientists to see whether, and how well, candidate drugs might work. It may allow them to find a few drugs among thousands that could treat the disease.
In 1993, a research team led by Dr. James Gusella, at Massachusetts General Hospital in Boston, found the gene that causes Huntington's.
But what they have not been able to isolate and study is the whole protein made by the gene, a substance called huntingtin. Without the entire protein to study, scientists find it hard to decipher what happens in the brain as a result of the mutation; why nerve cells in the basal ganglia gradually die off. The brain damage occurs because the protein made by the mutant gene is altered or disabled, setting it up to kill off nerve cells. Similar die-off of specific nerve cells, but for different reasons, is seen in other brain disorders such as Alzheimer's disease and Parkinson's disease. Wexler said that even though they haven't isolated the intact protein, the researchers are not slowing down their work. Their new strategy is to leapfrog ahead and begin an intensive search for drugs that can treat the disease. Their ultimate goal, of course, is to find a cure.
What they're setting up is an assay system that can quickly analyze the potential effectiveness of drugs among the many thousands of chemicals that exist. They expect the drug search to be speeded up by use of the glowing substance from jellyfish, which they call green fluorescent protein, or GFP. This test system was devised by a small biotechnology company in San Diego, Aurora Biosciences Inc.