Great Ormond Street Hospital (Gosh) is taking the unprecedented step of attempting to license a gene therapy for ‘bubble baby’ syndrome itself, after the pharmaceutical company that planned to bring it to market pulled out. The therapy had a more than 95% success rate in trials for ADA-SCID, a life-threatening genetic condition that leaves children with no immune system.
Professor Claire Booth, a principal investigator on the trial from Gosh and University College London, said: “We’ve been developing the treatments, seeing the amazing results in trial and then we hit this roadblock. We’re seeing more and more companies pulling out of the field. So we needed to find a way to get it to patients.”
The hospital plans to apply to the UK regulator, the Medicines and Healthcare products Regulatory Agency, next year for a licence to offer the treatment on a non-profit basis. The one-off therapy involves harvesting stem cells, genetically reprogramming them and reintroducing them to the patient, growing a new immune system that should last for life.
Currently, the treatment is only available on a compassionate use basis, which Booth described as unsustainable. The move comes amid concerns about the affordability of gene therapies; for example, the spinal muscular atrophy drug Zolgensma costs £1.6 million. Booth said these treatments “don’t literally cost £1m” and could cost one-tenth of that under the new approach.
Gosh has received £350,000 from the medical charity LifeArc and its own charitable arm to prepare the application. Dr Catriona Crombie, head of rare disease at LifeArc, said: “It is not acceptable to us that there are proven treatments failing to reach patients due to commercial challenges. If successful, we hope that this might be a proof of concept that would then allow other gene therapies to be made available for rare genetic diseases.”
