When Lou Gehrig played first base for the New York Yankees 100 years ago, he was by any era’s standards a physical specimen, eventually playing in what then was a record 2,130 consecutive ball games and earning the nickname “Iron Horse.”

Capping his baseball career with his iconic 1939 “Luckiest Man on the Face of the Earth” speech at Yankee Stadium, even he could not stop the physical ravages brought on by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a chronic and always fatal motor neuron disorder in which patients’ progressively lose control of their voluntary muscles.

Like other neuro-degenerative conditions, by the time ALS patients are clinically diagnosed, it is very difficult to reverse the damage. “The brain cells have already begun to deteriorate,” explains Todd Levine, MD, the principal investigator for the HonorHealth Research Institute’s ALS clinical trial, the first in the world aimed at preventing ALS.

“Once nerves are lost, all we can do is try to stop the remaining nerves from dying,” said Dr. Levine, who is also HonorHealth’s medical director of Neurology. “Our best option to ‘cure’ these diseases is to prevent them.”

First ALS prevention study

HonorHealth Research Institute is one of 30 sites worldwide participating in the first ALS prevention study, ATLAS. The focus of the study is to identify individuals who are genetically predisposed to acquiring ALS, specifically those who carry the SOD1 gene but who don’t yet show any symptoms.

The study aims to determine whether pre-symptomatic intervention with a drug called Tofersen (BIIB067) can delay clinical onset, or at least slow the progression of disease.

One of the participants in the clinical trial is Charlie Hargett, 47, who heads a business consulting firm in Denver. Charlie agreed to participate in the trial after he found out he has a mutation in the SOD1 gene associated with ALS, the same gene mutation carried by his mother, Debra “Dee” Hargett, who passed away from ALS in 2018.

“After my mom died, I really felt like I wanted and needed to do something to bring awareness of this disease. My hope is to help find treatments, and perhaps a cure,” said Charlie, who so far has no ALS symptoms. “I’m really hoping that this study is successful and proves that this drug can keep the SOD1 gene from producing proteins and block the progression of ALS.”

The study includes a sub-set of adult participants who carry certain rapidly progressive SOD1 gene mutations.

“These are people who have a genetic mutation that causes ALS. Many of them have already watched multiple family members die of the disease,” said Dr. Levine. “They know what they are facing. They know what’s in their DNA.”

“Previous drug trials have shown little clinical benefit in patients with neuro-degenerative conditions,” Dr. Levine explained. “That’s because we’re identifying the patients after they already have lots of nerves that are lost. We want to identify who’s likely going to get ALS before they get it, and then potentially prevent them from getting the disease.”

For more information about how to refer your patients to this study, or to learn more about other neuroscience studies, please email or call the HonorHealth Research Institute Neuroscience Research Team at 602-258-2863.