The World Duchenne Organization is expressing its concerns regarding an organization that claims to be raising money for a ‘lifesaving’ stem cell treatment. We are supporting and sharing this statement from Treat-NMD, the organization that represents doctors and scientists working in neuromuscular disease. Please read the statement below carefully.
Statement from TREAT-NMD
“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 treatment. The TREAT-NMD executive committee would like to issue the following statement:
There are 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.”
Considerations for stem cell therapy in Duchenne
To summarize, there are three important points to consider when researching treatments:
- Currently, there are no approved stem cell therapies for Duchenne muscular dystrophy
- You never pay for a clinical trial
- If a ‘trial’ is not on clinicaltrials.gov, it is not a legitimate trial
For any questions regarding this issue, please reach out to [email protected]