Director Professor Thomas Voit said
“GOSH BRC are at the forefront of translational research and continue to bring novel drugs to patients who need them, develop new medical approaches such as organ reconstruction, and push forward new platform technologies like AAV vectors for gene therapy. We were delighted to see so many people at our showcase event as we shared some of our recent research highlights and innovations in children’s medicine”.
During the day there were updates from each of the BRC Theme Leads who outlined key exemplar projects to date and aspirations for the remaining three years of the current funding term. Professor De Coppi introduced our new theme Advanced Treatments for Structural Malformations and Tissue Damage covering recent successes in stem cell therapies for retinal conditions and gut disorders as well as oesophagus tissue engineering and the UK’s first fetal surgery for spina bifida, carried out in collaboration with UCLH. The audience also heard from Gene, Stem and Cellular Therapies Deputy Theme lead Waseem Qasim who covered some of the GOSH BRC’s current and planned gene therapy portfolio and highlighted successes in cell therapy for Acute Lymphoblastic Leukaemia (ALL) using both edited T-cells from the patient and universal CAR-T cells from healthy donors.
Professor Paul Gissen updated on the Novel Therapies for Translation into Childhood Diseases theme highlighting recent successes in trials for Lennox-Gastaut syndrome, Spinal Muscular Atrophy, CLN2 disease and Duchenne muscular dystrophy. Prof Gissen also covered the development of a highly specialised cell and tissue repository enabling biomarker discovery for conditions such as hypertrophic cardiomyopathy, and described the GOSH BRC’s new wave of gene therapy studies using adeno-associated virus (AAV). Representing the Genomics and Systems Medicines theme was Professor Lyn Chitty who outlined the huge success of the 100,000 Genomes Project at GOSH and described the development of rapid paediatric sequencing, a method that can help establish a genetic diagnosis in critically ill children, as an exemplar of translational research in action.
Supporting the next generation
There were also short talks from the GOSH BRC’s Junior Faculty: a committee established to encourage early career researchers to develop leadership skills and ensure their voices are represented at all levels of BRC decision making. This included early career researchers Dr Paola Bonfanti, Dr Julien Baruteau and Dr Luís Lacerda who have all received support through the Junior Faculty’s innovative BRC Catalyst Fellowship. The scheme was established to encourage early career researchers to develop careers in translational research by offering funding and support to enable them to develop independent research proposals for external fellowship schemes.
Attendees also heard from radiographer Ian Simcock and physiotherapist Emma Shkurka who are carrying out PhDs supported by the NIHR’s Clinical Academic Fellowships scheme. Both Ian and Emma were were supported in their career development and fellowship applications by GOSH’s Centre for Outcomes and Experience Research in Children’s Health, Illness and Disability (ORCHID). Part of the GOSH BRC’s Experimental Medicine Academy, ORCHID aims to support nurses and allied health professionals to develop careers in research by providing guidance, training and protected time to focus on research.
The importance of patient and public involvement
The importance of PPI/E was addressed throughout the afternoon’s talks, with Professor De Coppi highlighting the crucial relationship between GOSH BRC researchers and oesophagus atresia patient groups. He explained how this two way dialogue helps shape research into new treatments for structural malformation, ensuring it is relevant, accessible and important from the point of view of patients with the condition.
Closing the day, was Dr Lola Solebo who described how patient and public involvement has played a vital role in shaping her research into the childhood eye condition uveitis. NIHR Clinician Scientist and Consultant Ophthalmologist Dr Solebo shared her experience of consulting with our Young Persons’ Advisory Group and how rewarding it was to discover how strong their opinions were on her research study design and direction. Dr Solebo also emphasised the importance of engaging with patients and the public at an early stage.