PAEDIATRIC NORTH STAR
NEWS
The NorthStar network has fully endorsed the TREAT-NMD Statement on Stem Cell Tourism.
'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 Carpricorn 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.
'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 Carpricorn 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.
- We are excited to announce the Adult NorthStar Database launched in December 2020 and it is now live. Over 20 adult Trusts are part of the Adult NorthStar Network and contribute to the adult database. Adults with DMD can join the registry from the age of 16 years.
If you have any queries related to the Adult NorthStar Network or Database and/or your Trust would like to participate in this project, please contact:
Adult NorthStar Network Coordinator
Aleksandra Pietrusz
a.pietrusz@ucl.ac.uk
Project Lead
Prof Ros Quinlivan
r.quinlivan@ucl.ac.uk
- The following paper has recently been awarded the RCPCH 2020 Lorber award for the best scientific paper in paediatrics at the RCPCH Conference which will be held on Tuesday 28th April 2020. This paper was made possible thanks to the collaboration with the North Star Clinical Network. "Fractures and Linear Growth in a Nationwide Cohort of Boys With Duchenne Muscular Dystrophy With and Without Glucocorticoid Treatment. Joseph S, Wang C, Bushby K, Guglieri M, Horrocks I, Straub V, et al." JAMA Neurology. 2019.
- The following poster was highly commended for the Best Poster Presentation by a Trainee Prize at the BPNA 2020 conference. "The role of dystrophin brain isoforms on early motor development and motor outcomes in young children with Duchenne muscular dystrophy. M Chesshyre, D Ridout, L Abbott, V Ayyar Gupta, K Maresh, A Manzur, M Scoto, G Baranello, F Muntoni." Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; 2Population, Policy and Practice Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, UK. Developmental Medicine & Child Neurology, 62(S1), 15–75.
EVENTS
- The Annual North Star National Meeting for network members was held virtually in July 2021, London.
- North Star and SMA-REACH consultants met at the 46th BPNA meeting which was held on the 30JAN2020, Belfast.
