COLUMBUS, Ohio — Amyotrophic Lateral Sclerosis, or ALS, is a debilitating disease that affects people’s nervous system causing them to lose function of their muscles.

What You Need To Know

• Tens of thousands of Americans are affected by ALS 

• There is no cause linked to the disease 

• In April, the FDA approved a new gene therapy infusion

• The therapy is to slow and/or stop the progression of the disease

Andrew Wurm was known to be the most active person in his family. He ran marathons, strength-trained and loved to hike and go on trips with his family, but all of it took a hard stop in 2021 when he began struggling to keep his balance.

“You go from living a normal life, if you will, to all of a sudden some sort of potential end that's in the near future,” said Wurm. 

He received back surgery to address the issue, but after strength symptoms continued he learned that his back wasn’t the issue at all.

“I noticed my leg is really not strengthening, post the surgery,” said Wurm. "It all just didn't make a whole lot of sense and basically in the middle of 2022, me and my wife started to ask questions for ourselves, like, 'Something's not right here.'”

In Oct. 2022, Wurm was diagnosed with ALS, a disease that affects the nervous system and progressively weakens muscle function. People diagnosed with the disease usually only live for two to five more years.

“We sort of tried to understand the timing of when I may have started to see these problems arise, to understand when I started to have issues,” said Wurm, “That's why the first thing is really trying to figure out the timeline, because we really try to figure out where you stand in your life and sort of what's what's left.”

Wurm and his family began preparing for the worst until he learned about a recently FDA approved gene therapy that would target his specific mutation. The infusion is designed to slow if not stop the progression of motor neuron loss.

“We now can deliver a piece of it (gene) that's sort of like RNA that finds that bad RNA, binds to it and then makes it go away,” said Stephen Kolb, who serves as a Professor of Neurology and Biological Chemistry and Pharmacology at OSU Wexner Medical School. 

Wurm received his first spinal treatment last week and will continue receiving treatments until doctors see results. The therapy has seen success anywhere from six months to a year of use. When he thought all hope was lost, Wurm said this therapy can give him more time to make memories with those he loves.

“All of a sudden you have a real hope that you might be able to sort of maintain a lifestyle over an extended period of time that you otherwise wouldn’t have thought was possible,” said Wurm. 

This gene therapy is created for a small pool of ALS mutations, but the hope is that scientists and researchers can expand the patients who can receive the potentially life-saving care.