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TGen finds genetic links to Lou Gehrig's disease
The Business Journal of Phoenix - 2:10 PM MST Wednesday, August 1, 2007by Angela GonzalesThe Business Journal


Researchers at the Translational Genomics Research Institute in Phoenix have discovered 10 genes connected to Lou Gehrig's disease.

TGen scientists screened DNA samples from more than 12,500 people with the disease and more than 2,000 people without the disease from across the country within the past six months. The progressive neurological disorder, which leads to paralysis and death in three to five years, has baffled researchers for nearly 140 years.


"We were able to sift through the entire 3 billion letter blueprints and identify these 10 genes which seem to be misbehaving in people who eventually get Lou Gehrig's disease," said Dietrich Stephan, director of TGen's neurogenomics division and the study's principle investigator.

His study was published in Wednesday's online issue of the New England Journal of Medicine.

Known in the medical field as sporadic amyotrophic lateral sclerosis, or ALS, the disorder quickly leads to complete paralysis.

"This is the first study to find consistent genetic changes that predispose to this disorder, giving researchers new leads for the development of therapeutics," Stephan said.

He is talking to pharmaceutical firms to partner on clinical trials to study existing drugs that may impact the disease. Because TGen owns the intellectual property surrounding these discoveries, a portion of the revenue from the drugs would go to TGen to support more research in the area.

Stephan said that would take at least a couple of years. He didn't want to say what type of drugs are being tested because people with the disease might try it on their own, which can be dangerous. However, 30 people with the disease will be needed to start a clinical trial.

TGen is working with Phoenix Neurological Associates to begin enrolling patients soon.

"The hope is to slow down the progression of the disease," Stephan said. "Of course, it would be wonderful if we could stop the progression of the disease. Of course, the holy grail would be to reverse the progression of the disease. We're very cautious in claiming that we think we can do that. We're just looking to slow down the progression right now."

The study was funded by a $652,000 grant from the Muscular Dystrophy Association's Augie's Quest, a fast-track ALS research program, in collaboration with TGen. Blood donated for the study came from various MDA/ALS centers throughout the country.

"We're beginning to see such major genomic discoveries from TGen on a steady, repeatable basis," said Saundra Johnson, executive vice president with the Flinn Foundation. "Combined with the economic impact that TGen is beginning to demonstrate, it's apparent that Arizona's investment in TGen five years ago is paying off handsomely."

For more: www.tgen.org.