Ohio State working toward cure for type of ALS through ‘transformative’ gene therapy

The Lantern
Andrew Wurm, 53, before his first injection of the ALS treatment at Ohio State. ALS is a condition that progressively destroys nerve cells and reduces muscle function. Credit: The Ohio State University Wexner Medical Center

Some say it’s best to attack a problem at its core — in science, that could mean altering a disease’s genetic makeup.

New developments at Ohio State could help researchers find a cure to a specific form of ALS — a condition that progressively destroys nerve cells and reduces muscle function — caused by a mutation in a certain gene. Dr. Andrew Thomas, chief clinical officer for Ohio State, said the research puts Ohio State on the map as one of the few institutions conducting gene therapy, which involves the insertion of different gene codes into a patient’s brain tissue.

This could be a permanent treatment for this type of ALS, also known as Lou Gehrig’s disease, according to Dr. Russell Lonser, director of Ohio State’s Gene Therapy Institute.

“It’s transformative,” Lonser said. “Every couple of generations in medicine, we see something that really will transform therapies and help reduce the burden of disease, and I think most people would agree that this is really a transformative therapy.”

Lonser said some of the most transformative research is coming from Dr. Stephen Kolb in the Wexner Medical Center’s Neuromuscular Disorders Center, who is looking at new ways to deliver this treatment to targeted disease areas through a new drug called QALSODY, which was approved by the FDA in April to treat ALS patients with a mutation in the superoxide dismutase 1 (SOD1) gene.

“We were the first in this area, this region, to offer that to patients who have ALS with a specific genetic form of that,” Kolb said.

Typically, the fluid with the treatment code is injected directly into brain tissue, but Kolb is looking at ways to change this by injecting it into spinal cord fluid instead, which could provide more potential avenues for treatment, according to Lonser.

“When we do it directly to the brain, we’re looking at very focused, targeted spots. By changing the route of delivery, he can potentially impact a larger area of the whole nervous system,” Lonser said.

Kolb said the projected effectiveness of QALSODY lies in its targeting of genes that may be responsible for the disease in patients with mutations, which it attacks at its core.

“If you know the gene, you can actually try to develop a way to alter the expression of that gene in patients,” Kolb said. “I think that this is a milestone in the treatment of ALS because now instead of medications that we don’t really know the mechanism — they seem to work in some of our preclinical models, but not that well — now, we are targeting specific genetic alterations.”

Gene therapy research has been conducted for more than 20 years, but only in the last decade has it begun to become more popular as a legitimate avenue for treatment, Lonser said.

“For nervous system disease and neurologic disorders, we had to figure out a way to deliver it effectively into the brain in various brain targets, and so that took time,” Lonser said.

Thomas said this research emerged earlier this month and Ohio State is one of the only institutions to be conducting it.

“We’re excited to be one of the few in the country to be doing this therapy for what is a uniformly fatal disease, so anything we can do to control or stem the progression of that disease is incredibly important,” Thomas said.

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