Caleb Sizemore says growing up with Duchenne’s Muscular Dystrophy (DMD) was tough.

Caleb’s story

The disease is a rare genetic disorder that slowly destroys a person’s muscles, impairing their ability to walk or breathe. Eventually, it attacks the heart, leading to premature death.

Caleb says the disease meant “I was limited in what I could do, where I couldn’t play sports and where I was teased and bullied sometimes for being different.”

In the past, people with DMD – almost all of whom are boys – lost the ability to walk by the age of 12, and many died in their 20s. But a new treatment originally funded by CIRM is showing promise. It’s helping reverse some of the damage caused by the disease.

New research

Published in the journal The Lancet, the results of a clinical trail showed that the therapy helped halt the decline in muscle strength in the arms and hands. I also appeared to improve heart function.

In a news release, Dr. Craig McDonald, a UC Davis professor and the study’s lead author, said th results were promising.

“The trial produced statistically significant and unprecedented stabilization of both skeletal muscle deterioration affecting the arms and heart,” he said.

The CAP-1002 therapy uses cells derived from the human heart. These are cells that have previously demonstrated the ability to reduce muscle inflammation. They’ve also been shown to enhance cell regeneration. The clinical trial is called HOPE-2 (Halt cardiomyopathy progression in Duchenne).

Dr. McDonald says that, with current treatments having only a limited impact on the disease, CAP-1002 may have a big impact on the people affected by DMD and their families.

“The trial showed consistent benefits of this cell-based therapy. It suggests that this infusion may be an important treatment option for the boys and young men who have this debilitating disorder.”

The team now hopes to apply to the Food and Drug Administration for permission to start a larger clinical trial involving more patients.

Caleb Sizemore took part in an earlier clinical trial involving this approach. He says MRI’s showed that the therapy appeared to reduce scarring on his heart and gave him greater energy.

In 2017, Caleb talked to the CIRM governing Board about DMD and his part in the clinical trial. You can see that video here.