New funding to support pioneering thymus transplant research at GOSH

11 Jul 2018, 2:19 p.m.

Professors Graham Davies and Adrian Thrasher have been awarded funding from Great Ormond Street Hospital (GOSH) Charity to further develop a cutting-edge new treatment for children with the rare immunodeficiency condition complete DiGeorge syndrome (cDGS).The condition results from an absence of the thymus - a gland responsible for producing the T-cells that help fight infections. Without treatment children with cDGS are likely to die before the age of 2.

The new research project will build on a previous trial led by Professor Davies which showed that transplant of donor thymus tissue restored T-cell numbers in 75 percent of patients with cDGS. Patients developed the ability to fight common infections and should have a much longer life expectancy.

The funding will support the LetterOne GOSH Charity Research Group in Thymus Transplantation and will enable researchers to look at how to maximise the number of T-cells produced after thymus tissue is transplanted into children with cDGS.

In the previous study, thymus tissue for transplant was obtained from children undergoing surgery for inherited heart defects as the thymus is normally removed and discarded to allow access to the heart. This does not affect the donor as their immune system has already developed by the time the thymus is removed.

Led by Professor Graham Davies, GOSH Consultant Paediatric Immunologist and Honorary Clinical Professor of Paediatric Immunology at UCL Great Ormond Street Institute of Child Health (ICH), the new research will investigate how the technique can be enhanced to reduce the autoimmune complications that can occur in some patients.

GOSH is one of only two centres across the world offering thymus transplant for cDGS. A longer term aim is to refine this treatment further so it might eventually be used to treat children with other immune system disorders.

This new award builds on earlier work supported by the National Institute for Health Research (NIHR) GOSH Biomedical Research Centre (BRC), EU FP7, GOSH Charity, Mason Medical Research and the Wellcome Trust.

NHS eye gene therapy restores Saffie's sight

Saffie has had her sight restored thanks to life-changing eye gene therapy for rare blindness at GOSH

£3M study led by patient voices targets pain in inflammatory arthritis

A new £3 million research programme involving Great Ormond Street Hospital and University College London researchers aims to tackle one of the largest unmet clinical needs in inflammatory arthritis – pain reduction.

GOSH joins European health leaders to shape the future of paediatric data sharing

More than 50 clinicians, data scientists, digital health innovators and industry leaders came together in Barcelona last month to tackle one of the biggest challenges in paediatric healthcare: how to share health data safely across borders to improve care

Engineered tissue offers hope for children born with ‘missing’ food pipe

Scientists from Great Ormond Street Hospital (GOSH) and University College London (UCL) have created the first lab‑grown oesophagus - the food pipe - shown to safely replace a full section of the organ and restore normal function, including swallowing, in