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A phase 3 clinical trial found that an experimental cell therapy called deramiocel slowed the loss of arm and hand function in children and young adults with Duchenne muscular dystrophy (DMD). The therapy also showed benefits for heart function.
At the American Academy of Neurology (AAN) Annual Meeting, researchers reported that deramiocel reduced the rate of DMD disease progression by 54 percent over one year compared with placebo (inactive treatment).
These findings are important because people with DMD often lose arm and hand function over time. This can make everyday activities harder, such as eating, dressing, and using a wheelchair.
Heart problems are also a major cause of serious complications in DMD, so treatments that may help preserve both muscle and heart function are closely watched by families and healthcare providers.
Deramiocel, also known as CAP-1002, is an experimental cell therapy. The treatment uses donor heart cells called cardiosphere-derived cells. Researchers believe these cells may help reduce inflammation and scarring in muscle tissue.
Instead of replacing dystrophin, the protein missing in DMD, deramiocel is designed to help protect skeletal and heart muscle from ongoing damage. The therapy works partly by releasing tiny particles called exosomes. These particles may help lower inflammation and support healing.
The phase 3 HOPE-3 study included 106 males ages 10 and older with DMD. Most participants could no longer walk independently. Everyone in the study continued standard corticosteroid treatment while receiving either deramiocel infusions every three months or a placebo (inactive treatment).
Researchers measured upper limb function using the Performance of Upper Limb 2.0 (PUL 2.0) scale, which tracks shoulder, elbow, and hand movement. After 12 months, people receiving deramiocel showed 54 percent less decline than those receiving a placebo.
The study also found:
Researchers also reported that deramiocel showed benefits when they looked at movement, heart health, and overall disease severity together.
According to investigators presenting the study results at the AAN Annual Meeting, deramiocel was generally well tolerated. The most common treatment-related side effects were temporary flu-like symptoms after infusions, typically resolving within 24 to 48 hours.
Investigators reported one serious adverse event (an unexpected health event) in the deramiocel group (1.9 percent) compared with five in the placebo group (9.6 percent). They said the overall safety profile was similar between the groups.
On myMDteam, people share their experiences with muscular dystrophy, get advice, and find support from others who understand.
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