We receive many questions from families who are interested in stem cell therapy as a potential treatment for Duchenne. Today, Treat-NMD (an organisation that represents neuromuscular clinicians and researchers from around the world) has released a statement about the “treatments” that some clinics abroad might offer to people living with Duchenne:
“There is a great unmet need for identifying new therapies for Duchenne muscular dystrophy. At the same time, it is critical that potential treatments be studied and tested in clinical trials in order to ensure their safety and efficacy.
It has come to the attention of TREAT-NMD that families of patients with Duchenne muscular dystrophy are raising money to fund a ‘lifesaving’ stem cell treatments. The TREAT-NMD executive committee would like to issue the following statement:
There is currently no clinically approved stem cell therapies for Duchenne, and there is no evidence that stem cell therapy is safe or effective for Duchenne muscular dystrophy.
Stem cell therapy is in early clinical development for Duchenne muscular dystrophy, including an ongoing clinical trial sponsored by Capricor Therapeutics. This trial is designed to test safety and potential effectiveness of this stem cell therapy product. Participants in the trial receive the treatment as part of the trial, and not by independently paying for the medicine. The information regarding safety, adverse events, and efficacy undergoes careful assessment by monitors and regulators and the results are presented at international meetings and in the peer reviewed literature.
Importantly, clinics selling stem cell treatment as a therapy make unsubstantiated claims about safety and efficacy of this approach. There is no evidence that stem cell treatment offered by these clinicals is effective and there is no evidence that it is safe.
Selling this as a treatment is a not an acceptable clinical practice, and furthermore carries significant risk for potential harm to patients using such unproven and unregulated therapies. We therefore strongly recommend against pursuing such treatments, and advocate against the raising of money to support obtaining non-regulated substances or biologics.
As physicians and researchers working on Duchenne muscular dystrophy, and in partnership with families and advocacy groups, we are fully aware of the need to find effective therapies for this condition; nevertheless we caution against any unproven initiative which could put the health and potentially the lives of boys and young man affected by Duchenne muscular dystrophy at risk.
