Backed by funding from the Cure JM Foundation, scientists at the UK’s Nucleic Acid Therapy Accelerator (NATA) are creating tiny medicines that press the “quiet” button on the immune system’s mistaken alarm that causes myositis.
How Is the Immune System Misbehaving in JM?
Think of your immune system like a safety alarm. Interferon beta is the warning that blasts when a virus shows up. It tells the immune system to wake up and get to work. With JM, sometimes that alarm gets stuck on even when there is no danger. A loud, nonstop alarm makes the immune system overreact. In juvenile myositis that constant signal can inflame muscles and make kids feel weak, sore, and tired.
What Did the Scientists Do?
With grant funding from Cure JM, scientists tested tiny “off switches” that quiet the interferon beta alarm at its source. They tried 27 versions and found two that worked best. Those two switches turned the immune system alarm way down, which reduced inflammation. Just as important, safety tests showed that this approach was also safe.
Why Does this Matter?
If the immune system “alarm” can be quieted, the immune system may calm down and muscles, skin, and blood vessels may be protected. This is an early lab step, not a treatment for people yet, but it points toward a new way to help without heavy steroids.
What’s next?
The team plans animal studies to confirm functional benefits (like stronger muscles and less inflammation) and has already shared results at major scientific meetings. These are clear signals that this Cure JM–supported work is moving toward real-world impact.
The Bottom Line
Cure JM-backed researchers have found two tiny medicines that aim right at the problem genes in JM. They could be the first of their kind and a new way of treating JM. This is the kind of progress that your support makes possible.
__________________________________________________________
Tell Me More about the Science
What is the NATA?
The Nucleic Acid Therapy Accelerator (NATA) is a UK national initiative dedicated to developing nucleic acid therapeutics and technologies that support them. By bringing together scientists, clinicians, charities, and industry partners, NATA provides the expertise and resources needed to turn discoveries into real treatments. They are helping promising new treatments be designed, tested, and refined all in one big space. This collaboration allows promising new science to flourish.
What is a Nucleic Acid Therapy?
Nucleic acid therapy is medicine made of genetic instructions that tells cells exactly what to do differently, so the cells are no longer misbehaving.
In the case of JM, we know that the immune system alarm is driven by a chemical called interferon-beta which drives inflammation. A nucleic acid therapy is a tiny piece of genetic code that can tell cells to make less interferon-beta, which may calm the immune response without heavy steroids.
