A new report has highlighted how NHS referral pathways and an approved treatment for gene therapy trials for Duchenne muscular dystrophy (DMD) need to be agreed.
The report is particularly important for patients and families living with DMD as it sets key recommendations that, if implemented, will transform access to potentially life-changing treatments.
DMD is a genetic disease that causes muscle weakness and wasting. It eventually affects all the muscles in the body, including the heart and lungs.
No cure, but promising treatments
Unfortunately, there is currently no cure, but there are treatments and therapies that can slow down the progression and improve quality of life.
Gene therapy is one of the most promising new treatments for DMD. It involves delivering new genetic material to cells to overcome errors (or mutations) on the dystrophin gene.
A gene therapy was approved as a DMD treatment in the US in June. This was the first DMD gene therapy to be approved anywhere in the world.
While not yet available as an approved treatment in the UK, there are DMD gene therapy clinical trials taking place – including in Newcastle – giving eligible patients the opportunity to access these potentially transformative treatments.
Gene therapy on the horizon
Michela Guglieri, professor of neuromuscular disorders at Newcastle University and honorary consultant in human genetics at Newcastle Hospitals, said:
“As gene therapy is on the horizon for Duchenne muscular dystrophy as a licenced product, we considered how we can prepare for gene therapy licenced products in Duchenne muscular dystrophy as standards of care, using learnings and shared experiences from approved gene therapies in other neuromuscular disorders.
“A set of recommendations for improved, standardised future practice is proposed, as well as referral pathways for gene therapy clinical trials and licenced products for Duchenne muscular dystrophy.”
Equitable access to research
The report is the result of a collaborative effort of the DMD Hub (Duchenne UK and the John Walton Muscular Dystrophy Research Centre, a partnership between Newcastle University and Newcastle Hospitals), Cell and Gene Therapy Catapult, and the Northern Alliance Advanced Therapy Treatment Centre.
Emma Heslop, DMD hub manager, added:
“By highlighting best practice from across the UK, we propose a referral pathway for gene therapy clinical trials in the UK, to allow effective and equitable access to research for Duchenne muscular dystrophy patients”.
Commenting on how important it is that these recommendations are adopted, Alessandra Gaeta, Director of Research at Duchenne UK, said:
“Now that these recommendations have been developed, we can use them to drive best practice in Duchenne muscular dystrophy through their adoption in the NHS.”
The report can be viewed here.
- Pfizer Ltd provided financial support for this project, to Cell Therapy Catapult Ltd, in the form of a grant. Pfizer Ltd has had no influence in the initiation, development or delivery of this project, nor have they influenced the development or content of any materials produced as an output of this project.
