Ever wonder why steroids are sometimes used with gene therapy? In this short video, part of our GT-FAQ video series, John, an experienced nurse on our Duchenne Nurse Educators team, explains how steroids help lower the body’s immune response to help the therapy work as intended. He also talks about when steroids are used and why doses may differ from person to person. Take a moment to watch and learn the basics.

Read the video transcript below.

Video Transcript

Why are steroids used with gene therapy?  

Steroids are medicines that reduce inflammation in the body. They work by slowing down or stopping parts of the immune system that cause inflammation.  

What does that have to do with gene therapy?

Some gene therapies use a viral vector – that’s an engineered version of a naturally occurring virus – to carry new genetic material to specific cells. 

But when this viral vector is introduced, the body might mistake it for a naturally occurring virus – like the flu virus – and try to fight it off. That immune response can stop the therapy from working as intended.

Steroids help temporarily lower the body’s immune response so that the gene therapy has time to do its job.

When are steroids given?

Steroids are given before, during, and sometimes briefly after gene therapy treatment.

Why are high doses of steroids needed with gene therapy?  

The amount of gene therapy a person needs depends on the condition being treated. For neuromuscular diseases, for example, the gene therapy must reach many muscle cells throughout the body, so a bigger dose may be needed. But a bigger dose means more viral vector and that may trigger a stronger immune reaction. To help manage this, doctors may give a high dose of steroids.  

How long are high doses of steroids required?

These high doses are typically temporary. Doctors will monitor a patient before, during and after gene therapy treatment. The doctor will create a plan to slowly withdraw steroids or adjust levels back to the patient’s normal treatment plan.  

Let’s review!

  • Gene therapy uses a viral vector.
  • Viral vectors can trigger the body’s immune system.
  • Steroids reduce the body’s immune response.
  • Steroids are given before, during and after gene therapy ...  
  • To help gene therapy work as intended.

If you have questions about gene therapy, talk to your healthcare provider or email our Patient Affairs team at [email protected].