New stem cell therapy shows promise in Duchenne trial

Published Date
Beatriz Bustillo Ramirez
24 wells cell culture plate

Capricor Therapeutics has recently published the final results from its HOPE-Duchenne clinical trial. This study evaluated the safety and efficacy of a cell therapy called CAP-1002 in people with Duchenne muscular dystrophy.

CAP-1002 consists of ‘cardiosphere-derived cells’, a type of stem cell obtained from donated human hearts. Animal studies have shown that these cells can generate new muscle and reduce muscle scarring (fibrosis). They also encourage other cells to grow and repair by releasing tiny packages called exosomes, which carry important proteins and signalling molecules from one cell to another.

The HOPE-Duchenne study was a phase I/II randomized, controlled, open-label trial. Patients older than 12 years old with myocardial fibrosis (scarring of the heart tissue) were treated with either CAP-1002 or standard of care. 25 patients took part, of which 17 were non-ambulatory. CAP-1002 was delivered with a procedure called ‘intracoronary infusion’, where the cells were pushed directly into the heart through a nearby blood vessel.

To measure the efficacy of the treatment, the investigators used the Performance of the Upper Limb (PUL) test and heart Magnetic Resonance Imaging (MRI). The PUL test is a standardised way of measuring a patient’s mobility of the shoulder, arm and hand. It involves a number of different exercises such as lifting and moving objects of different weights and sizes.

After twelve months, myocardial fibrosis was reduced in treated patients when compared to those in the standard care group. Muscle function also improved, especially in the patients that were initially doing worse in the PUL test. There were no major safety concerns associated with the treatment.

Capricor is now conducting a Phase II, double-blind, placebo-controlled study in the USA. This trial delivers CAP-1002 using a drip connected to a vein – a method that is less invasive and potentially safer.

This cell therapy is in its first stages of development, but the results so far are nevertheless exciting. They show that it is possible to deliver cardiosphere-derived cells to Duchenne patients and that this could have potential benefits in the heart as well as in other muscles of the body.

Michael Taylor, one of the authors of the study and Director of Advanced Imaging Innovation at The Heart Institute (Cincinnati Children’s Hospital, Ohio, USA), said in a press release:

These findings suggest that CAP-1002 could be an option for older patients who have scarring in the heart as a result of the underlying disease. As many of these patients are in later stages of the disease, the improvement in upper limb strength is important in order to maintain independence.

The study was published in the scientific journal, Neurology.

Further information

Find out more about the Phase II HOPE 2 clinical trial.

Read Capricor’s press release.

Learn more about stem cells and their potential as a treatment

If you have any questions about this news story or any other DMD research, please contact the MDUK Research Line on 020 7803 4813 or email

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