GOSH team begins development of oesophagus replacement therapy for infants with birth defects

4168A technique for replacing oesophageal tissue in babies who have not developed the organ properly is currently being established by surgeons at Great Ormond Street Hospital, in collaboration with teams from UCL (University College London) and Royal Free Hospital.

The work could prove to be the first step towards a clinical trial of a tissue engineered oesophagus in infants. As the lack of a complete oesophagus leads to severe feeding problems, this technique could provide effective swallowing for children as they grow.

Infants born with oesophageal atresia lack a complete oesophagus, resulting in an inability to feed, long-term health issues and often requiring full-time care. Around 250 babies are born yearly with this condition in the UK – a large number of which are treated at GOSH. Current surgical repair approaches for this condition are generally ineffective therefore alternative techniques are greatly needed.

Building on previous work, the team are set to develop their approach using stem and other cells taken from amniotic fluid in pregnancy, which are then grown on a donor ’scaffold’ to create a new oesophagus. Development and analysis of this technique is expected to lead to a clinical trial in newborns in 2016.

This work is possible thanks to funding by the UK Stem Cell Foundation and support from UCL Business PLC.

Dr Paolo de Coppi, consultant paediatric surgeon at GOSH & clinical senior lecturer at UCL Institute of Child Health, said: ‘At Great Ormond Street Hospital we treat some of the most severe cases of oesophageal atresia, witnessing the difficult long-term health problems these children experience. This funding from the UKSCF, for which we are very grateful, will help us develop this technology, ultimately providing patients with an entirely new oesophagus rather than trying to repair an inadequate one. This is part of our wider research into regenerative medicine, in which we are aiming to engineer rejection-free organs and tissues for transplant.’

The Specialist Neonatal and Paediatric Surgery (SNAPS) department at Great Ormond Street Hospital provides specialist surgical treatment for newborn babies (neonatal) and children (paediatric) with congenital (present at birth) conditions as well as diseases of the gastrointestinal tract.

The department has an excellent international reputation in clinical and basic science research and develops new ways of treating newborn babies and children, such as oesophageal replacement surgery and minimally invasive (keyhole) surgery. Expertise in stem cell therapy and regenerative medicine also exists in the surgical research department.

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