Potential treatment may help patients with muscular dystrophy

Medical Watch

There’s news of a potential treatment that may help patients with muscular dystrophy and it’s giving hope to those with a debilitating genetic disease. 

There are more than 50 forms, but not one cure-all treatment for the disease that causes progressive muscle weakness. Patients lose strength in their limbs, core and even their heart and breathing function suffers. For decades, doctors have been searching for a way to stop the damage.

Now local researchers hope a lucky discovery in the lab will lead to better function for patients.

Dr Elizabeth McNally is a cardiologist and genetic researcher at Northwestern Medicine.

“One of the things that really goes wrong in muscular dystrophy is that the muscle gets replaced by scar tissue, she said. “That’s really the definition of dystrophy.”

It’s a process McNally and her research collaborators have been trying to stop for 20 years.

Scientists made the discovery early on — a potential target called TGF beta.

McNally said the scaring process is driven by the TGF beta molecule.

“It was one of those long-shot things,” McNally said.  “When we got this first tip it was one of those, ‘Well that was kind of lucky.’” 

As luck would have it, TGF Beta plays a critical role. Too much of it causes scar to form. From there, the researchers went to work on an antibody — a drug/protein designed to lessen the amount of TGF Beta in the muscles — thus reducing the amount of damage and weakness.

“Not to make so much scar, and the interesting part we found it stabilizes the muscles and makes them less vulnerable to injury,” McNally said. “And it also helps the muscles go bigger.”

They tested the treatment in mice with the disease.  

Alexis Demonbreun, PhD is a  genetic researcher at Northwestern Medicine.

“In these mice, they were treated early on in the disease and then they were treated for six months,” Demonbreun said. “So we’re actually slowing the progression of the disease. And now we’re currently looking to see if we can actually reverse the scarring.”

The team is optimistic their antibody will work in human patients — even those with the most severe form known as Duchenne Muscular Dystrophy. Still, there are limitations when it comes to applying a treatment that works in mice to human medicine.

“That is true and we know that but we have some faith in this pathway that we are modifying,” McNally said. “We actually think this therapy will be useful for many different forms of muscular dystrophy.”

McNally hopes to produce more of the antibody and test it for safety before taking it to human patients. She’s hopeful the next steps will move quickly much like the process to produce Covid-19 antibodies.  

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