Team led by UCL & GOSH to develop infant oesophageal replacement product First joint project for Cell Therapy Catapult & UK Stem Cell Foundation.

A team from UCL (University College London), Great Ormond Street Hospital (GOSH) and Royal Free Hospital is collaborating with the Cell Therapy Catapult on the development of a tissue repair product for babies in whom the oesophagus has not developed properly. The project, funded by the UK Stem Cell Foundation and supported by UCL Business PLC, builds on work of UCL, GOSH, Royal Free Hospital and University College London Hospital. It is expected to result in the first clinical trial of a tissue engineered oesophageal replacement in infants, providing effective swallowing 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, and as a leader in child health, GOSH treats a large number of these patients. With current surgical repair approaches generally ineffective, oesophageal atresia is an area of clear unmet medical need. The market for this condition is thought to be worth more than £70m pa, and a successful product would also have potential in oesophageal repair or replacement following trauma or disease in adults and children.

Using the expertise developed in previous studies, the project team will develop its oesophageal repair approach using stem and other cells taken from amniotic fluid in pregnancy, which are then grown on a donor ‘scaffold’ to create a new organ. Development and analysis of the prototype is expected to lead to a clinical trial in neonates in 2016. The Cell Therapy Catapult will be responsible for project management, as well as providing input around non-clinical, clinical and regulatory matters.

Dr Paolo De Coppi, consultant paediatric surgeon at GOSH & clinical senior lecturer at UCL Institute of Child Health, said: ‘At Great Ormond Street 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.’

Keith Thompson, CEO of the Cell Therapy Catapult, said, ‘The Cell Therapy Catapult is involved with several innovative scaffold-based tissue repair projects, and it’s clear that this is an important source of new products. We’re delighted that our first joint project with UKSCF is in this area of high unmet medical need, where our expertise can bring real patient benefit.’

Lil Shortland, CEO of UKSCF, said, ‘The UK Stem Cell Foundation is pleased to fund this important and ground-breaking research in the area of oesophageal atresia. With our focus on progressing the development of stem cell-based therapies for devastating conditions, this project is fully aligned with our objectives.’