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ALS breakthrough at the U. could be a game-changer for patients

By Heather Simonsen  |  Posted Apr 12th, 2017 @ 7:00pm


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SALT LAKE CITY — Seth Christensen's computer tracks his eye movements so he can type like he used to, before ALS, commonly called Lou Gehrig's disease, began stealing his abilities seven years ago. After an active life with four kids, the Christensen family's youngest is just a baby. The diagnosis was devastating.

"We had to practice breathing in and out," said Amy Christensen, Seth's wife, who lives in Holladay with him and their children. "It really rocked our world."

ALS is taking away his ability to move and think.

Jean Corlett of Sandy has a similar brain disease called ataxia. "I was always losing my balance and I had a pain in my back," Corlett said.

Once an avid hiker and skier, Corlett can barely walk with a walker. And there are other problems. "Swallowing, choking, I'm always choking," she said.

Scientists at the University of Utah believe they can help people like them. "We treated the mice at eight weeks which is right when we consider disease symptom onset," said Daniel Scoles, PhD, associate professor of Neurology at the University of Utah.

They developed a therapy that slows the progression of the diseases in mice by targeting the ataxin-2 gene. The scientists injected the rodents with small snippets of manufactured, modified DNA called antisense oligonucleotides.

Dr. Stefan Pulst, chair of the Department of Neurology at the University of Utah, said, "These are molecules, pieces of DNA, that target a specific message in the cell and through complex mechanisms destroy that message. In many ways like a magical bullet or a seek-and-destroy kind of approach to diseases."

The Christensens are cautiously optimistic about the discovery. "It's hard to put your heart and your hope out on the line again," Amy Christensen said. "I do know that there's a little boy at my house who prays every night for those doctors and researchers to find a cure."

Along with alleviating ALS and ataxia symptoms, researchers say it could reverse or bring back some abilities. "We saw that we could halt the progression of the motor deterioration and we could also revert the changes in the specific proteins and, even more amazingly, in the firing of the nerve cells," Pulst said.

A breakthrough at the University of Utah could be a game-changer for deadly brain diseases like ALS. The findings, published today in "Nature," the top journal in science, are the first of their kind. This discovery means new hope for patients. (KSL TV)

The hope is that in time the compound will be given as spinal injections in patients. It stays in the brain for a long time. Scoles said, "We can treat the mice one time and for weeks and weeks, we see a therapeutic effect."

That's good news for Corlett, who misses her active life in the outdoors, but whose disease is advanced. "I'm hopeful, not for me, but for other people," she said.

Seth Christensen has already surpassed ALS' average 2-5 year lifespan. The Christensens are not giving up hope. "Of course we want that, and it would be amazing," Amy Christensen said. Life for them now, with Amy as Seth's caregiver, is all about endurance. And cherishing every precious moment.

Scientists say the discovery may lead to advances in other neurodegenerative diseases like Alzheimer's and Parkinson's. They hope to start clinical trials in 5 years.

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