A first-of-its-kind clinical trial is underway at Honor Health Research Institute in the battle against amyotrophic lateral sclerosis (ALS).
“The things that we take for granted every day are robbed from people with ALS,” said Charlie Hargett.
Hargett watched it firsthand with his mother, Debra. It stole her physical abilities one by one. She died on December 18, 2018.
“The feeling of helplessness is just overwhelming and that it’s happening to a loved one it just multiplies it exponentially,” said Hargett.
Before she passed, a genetic test revealed she had a gene called SOD1; A mutated gene that signals a strong possibility the person will develop ALS in their lifetime.
“We were obviously concerned, distraught that my mom passed so terribly, but knowing that might be in our DNA makeup, we sought out some help,” Hargett said.
He and his brother sought out their own test. The gene didn’t show up for his brother, but Hargett wasn’t so fortunate.
“To say it was a gut punch would be grossly understating it,” said Hargett.
The father of three immediately thought of his kids and his soon-to-be-born first grandchild. Hoping to make a difference in his life or at least in his children’s, he joined a new trial taking place at Honor Health Research Institute.
“This new trial that we’re doing is really the first in the world to see if we can prevent the disease from starting,” said Dr. Todd Levine.
Dr. Levine is the principal investigator for the study. He says those with the SOD1 gene who have not begun showing symptoms of ALS make up the trial.
Working with BIOGEN, they’re delivering a drug that blocks toxic proteins produced by the gene in hopes of keeping the disease at bay. Previous drug trials have shown little clinical benefit in patients with neurodegenerative conditions.
“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,” said Dr. Levine. “The goal really for them is to try to find this major breakthrough, and it’s not very often in ALS there’s a legitimate chance to find that major breakthrough.”
A number of different mutated genes have been found to cause increased odds of developing ALS. By focusing the study on one, if successful, the hope is to use that strategy to attack others.
For Hargett, every second counts. The study is providing him hope for a future filled with adventures, laughter and love.
Doing it by submitting himself to science, he’s determined to help make a difference for generations to come.
“Any small contribution that I can have to researching a cure or even a treatment that’s somewhat effective I’m all for,” said Hargett.