Researchers Make Discovery That May End Lou Gehrig's Disease


Doctor Says Much More Work To Do To Prove Discovery

POSTED: 3:26 a.m. EDT August 31, 2003
UPDATED: 4:07 a.m. EDT August 31, 2003

DALLAS -- There is a new medical discovery at the University of Texas Southwestern Medical Center at Dallas, according to a report.

The Dallas-based researchers, working on amyotrophic lateral sclerosis, have discovered something that may soon end Lou Gehrig's disease, another name for ALS.

"People are very excited about this in the lab, and I am too," Dr. Jeffery Elliott told KXAS-TV.

The team of highly skilled researchers, led by Elliott at UT Southwestern, doesn't give into excitement easily.

Elliott's team discovered bright red spots, or aggregates, in a mutant protein called SOD1, trigger the death of motor neurons in spinal cells of mice, the hallmark of ALS.

"And if we are able to find what exact portion of the molecule induces aggregate formation, that is one of the important things to help find a cure for the disease," UTSMC researcher Dr. Uma Krishnan said.

What they discovered next gave Elliot's researchers a clue of where to look. UTSMC researcher Dr. Krishnan Puttparthi says they found when the SOD1 protein mutates, all other organs in the body can handle it except the spinal cord.

"The liver has a high capacity to get rid of this thing rather than the spinal cord, so we are looking into why the liver is not affected ... why only the motor neurons," Puttparthi said.

Puttparthi and the rest of Elliott's team focused on finding out how the other organs process the mutant SOD1 protein.

A few months ago, after trying chemicals found in other organs and not found in motor neurons, Elliott's researchers made a startling discovery in the cultures of mice spinal cords growing in their labs.

"We are causing the aggregates in these slices and then we see we are able to remove those aggregates by treating them with other chemicals," Puttparthi said.

Test after test proved the discovery, the chemicals removed the aggregates in the mutant protein known to cause ALS in mice.

"It's a reversible process," Elliot said.

"I think that tells us that the nervous system can repair itself, in a sense that it can remove these protein inclusions, and that is very exciting,"
Puttparthi said.

While extremely successful in the lab culture dish, Elliott says there is still much work to do to prove their discovery will reverse ALS in humans, KXAS-TV reported. His team is now preparing for the next and possibly the most important step in that research.

"To take it to a live animal, to see whether there it can be reversed, not only reverse the aggregates, but, of course, reverse the disease itself," Elliot said.

If the experiments in live mice prove successful, human trials could be under way in less than two years, but there is more. In addition to ALS, researchers at UTSMC have discovered the mutant SOD1 protein is also found in Parkinson's disease, Huntington's disease and Alzheimer's disease.

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