New study shows resolving inflammation could prevent early heart damage in Duchenne muscular dystrophy
DMD is a genetic condition that causes progressive muscle weakness, including in the heart.
For boys with Duchenne muscular dystrophy (DMD), heart damage can begin early in life, often before symptoms are noticeable. A new study from Children’s National Hospital sheds light on why this happens and points to a promising therapy that could change how doctors protect the hearts and muscles of children living with DMD.
The findings, published in Cell Death & Disease, laid the groundwork for a newly awarded Department of Defense (DoD) clinical and translational research award that will launch in August 2025. This grant will support critical studies needed to advance this therapy toward clinical trials.
The challenge: Protecting growing hearts and muscles
DMD is a genetic condition that causes progressive muscle weakness, including in the heart. Steroids remain the standard treatment for inflammation, but long-term use can slow growth in children, contribute to muscle loss and even interfere with newer gene therapies as boys get older.
“Treating chronic inflammation is essential, but it has to be done in a way that supports cardiac muscle repair and works in concert with other emerging therapies,” says Jyoti Jaiswal, PhD, professor at Children’s National and senior author of the study.
As boys with DMD live longer thanks to exon skipping and micro-dystrophin gene therapies, there is an urgent need for treatments that keep their hearts and muscles healthy.
Understanding early heart damage
In this study, researchers focused on how and why heart damage begins in the absence of dystrophin using a severe model of DMD. They found that the problem is not just inflammation itself, but the failure to turn it off. When inflammation lingers instead of resolving naturally, it leads to scarring and tissue loss in both heart and skeletal muscle.
Instead of relying on steroids to block inflammation, the research team tested a pro-resolution therapy that works to clear inflammation through the natural healing process. The therapy activates formyl peptide receptors, which play a role in naturally switching off inflammation once tissue repair begins.
This approach successfully reduced inflammation, limited scarring and preserved healthy heart and skeletal muscle tissue. Importantly, it may also complement gene therapy approaches, potentially boosting their effectiveness.
“This is a physiological way to help the body finish the healing process,” says James Novak, PhD, associate professor at Children’s National and the lead author of the study. “It allows inflammation to do its job to help repair the tissue and subsequently clear out to prevent chronic inflammation and tissue damage.”
Building toward clinical trials
These findings helped secure a DoD grant through which, the team will pursue investigational new drug (IND)-enabling studies; a critical step toward bringing this therapy to clinical trials for children with DMD.
“This line of research represents a new way of thinking about treating Duchenne,” says Dr. Jaiswal. “We are excited to take the next step in moving it closer to patients.”
Read the full study in Cell Death & Disease.
