Twelve days of Research and Innovation at GOSH 2024

23 Dec 2024, 1:51 p.m.

set of 3 colourful test tubes in a lab

It’s been a busy year for Research and Innovation at GOSH, with a number of ground breaking research trials, advanced data projects and technology pilots.

To round off the year, here are just twelve of the stories (and amazing staff and researchers) that we shared this year.

We’re looking forward to seeing what 2025 will bring!

1. GOSH announces collaboration in pioneering PHEMS project to revolutionise use of health data across Europe

GOSH announces it is one of six leading European children’s hospitals to make up the PHEMS project consortium. PHEMS (short for ‘Paediatric Hospitals as European drives for multi-party computation and synthetic data generation capabilities across clinical specialities and data types’) project will revolutionise how children’s health data is managed and used across Europe and advance research and innovation in healthcare for children, while ensuring patient’s rights and data privacy remain protected.

GOSH collaborating in pioneering PHEMS project to revolutionise use of health data across Europe | Great Ormond Street Hospital

2. Stem cells collected in late pregnancy herald advances in prenatal medicine

A new pioneering approach, developed by researchers at UCL and Great Ormond Street Hospital, means human development can be observed in late pregnancy for the first time, raising the possibility of monitoring and treating congenital conditions before birth.

Stem cells collected in late pregnancy herald advances in prenatal medicine | Great Ormond Street Hospital

3. First-of-its-kind study into the Ketogenic diet

A ketogenic diet, which is low in carbohydrates and high in fat, shows promise as an alternative treatment option for infants with drug-resistant epilepsy.

The new study, led by researchers at GOSH and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH), was the first in the world to compare the impact of a ketogenic diet to further anti-seizure medication for infants aged one to 24 months with drug-resistant epilepsy.

The study found both treatment options to be effective for reducing seizures in infants.

Ketogenic diet effective treatment option for infants with drug-resistant epilepsy | Great Ormond Street Hospital

4. New plan announced to get more children access to gene therapy treatments

GOSH, supported by LifeArc and Great Ormond Street Hospital Children’s Charity announces plans to revolutionise how children living with a rare disease can gain access to life-changing treatments that have been proven to work.

A big challenge with developing treatments for rare diseases is that due to small patient numbers they are often not commercially viable for companies to invest in and keep on the market, even if the treatment has been proven to cure a condition.

GOSH, LifeArc and GOSH Charity hope to address this through the plan which would be the first of its kind in the UK and the first time an NHS Trust would hold the market authorisation - or licence – for a gene therapy drug, meaning patients can directly access and benefit from the treatment.

New plan announced to get more children access to cutting-edge, proven gene therapy treatments for rare diseases | Great Ormond Street Hospital

5. New treatment for brain tumour approved after over 20 years of research

The first-ever targeted treatment for brain tumours in children was approved for NHS patients, following decades of research by GOSH consultant Professor Darren Hargrave.

New treatment for brain tumour approved after over 20 years of research | Great Ormond Street Hospital

6. First UK trial of Deep Brain Stimulation for children with epilepsy begins at GOSH

Thirteen-year-old Oran made history as the first patient in the UK to participate in a clinical trial using deep brain stimulation (DBS) to treat epilepsy.

Since the procedure, his daytime seizures have decreased by 80%, having a significant impact on his quality of life.

First UK Trial of Deep Brain Stimulation for Children with Epilepsy Begins at Great Ormond Street Hospital | Oran's Story | Great Ormond Street Hospital

7. GOSH only hospital outside of North America to receive innovation funding award for Artificial Intelligence

GOSH was awarded the Amazon Web Services IMAGINE Grant: Children’s Health Innovation Award, to support AI development and drive progress for children’s healthcare.

GOSH was the only hospital outside of North America to be successful in receiving this funding.

GOSH only hospital outside of North America to receive innovation funding award for AI | Great Ormond Street Hospital

8. The GOSH Clinical Informatics Research Programme (CIRP) receives funding for another year from GOSH Charity

In partnership with Higher Education Institutes across the UK, CIRP funds data scientists, clinical and non-clinical PhD fellowships, internships, and other activities that enable health informatics research.

By offering the opportunity to work alongside clinician specialists and other hospital staff, CIRP projects focus on developing the use of data and technology to transform delivery of care, for the benefit of patients at GOSH and the wider NHS.

Academia | GOSH DRIVE

9. New clinical trial at GOSH gives hope to children with aggressive blood cancer

Researchers at GOSH and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) are collaborating on a novel approach to clinical trials to give hope to children with an aggressive type of blood cancer, T-cell acute lymphoblastic leukaemia (T-ALL).

New clinical trial at GOSH gives hope to children with aggressive blood cancer | Great Ormond Street Hospital

10. GOSH pilots AI tool to give clinicians more quality-time with patients

Patients and clinicians at GOSH took part in the first NHS trial of a bespoke healthcare AI tool, TORTUS, to help increase face-to-face time during appointments.

Following successful early stages at GOSH, the programme led by GOSH’s Data Research, Innovation and Virtual Environments (DRIVE) unit, has secured funding to deliver the first evaluation of ambient voice technology at scale in the NHS, in partnership with AI company TORTUS.

GOSH pilots AI tool to give clinicians more quality-time with patients | Great Ormond Street Hospital

11. After a successful pilot, the GOSH Clinical Insights Unit is renewed to deliver advanced analytics for a second year

The Clinical Insights Unit (CIU) at GOSH DRIVE uses advanced data analytics to support operational decision making at GOSH.

The team is made up of data scientists and analysts who work with operational and clinical teams in the hospital to increase the accessibility and utility of Electronic Patient Record (EPR) data.

By partnering with the clinical and operational teams, the CIU can understand their needs and build digital tools to enable data-driven decision making to help run the hospital.

Clinical Insights Unit | GOSH DRIVE

12. Duchenne Muscular Dystrophy gene therapy trial highlights complexity of disease

The first large-scale trial of gene therapy for the debilitating neuromuscular disease, Duchenne Muscular Dystrophy (DMD) has been carried out by an international group of researchers, including Great Ormond Street Hospital (GOSH) and University College London (UCL).

DMD is a genetic condition which causes progressive muscle weakness, most commonly in boys.

Duchenne Muscular Dystrophy gene therapy trial highlights complexity of disease | Great Ormond Street Hospital

NIHR GOSH Clinical Research Facility celebrates patients and their families with a festive celebration

On Wednesday 18 December we celebrated the festive period at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility (CRF) on the 8th floor of the Southwood building.

Duchenne Muscular Dystrophy gene therapy trial highlights complexity of disease

The first large-scale trial of gene therapy for the debilitating neuromuscular disease, Duchenne Muscular Dystrophy (DMD) has been carried out

New findings from world’s largest study on children with Long-Covid

A new study led by clinicians and researchers at Great Ormond Street Hospital and University College London Great Ormond Street Institute of Child Health has found that 70% of young people in England with Long-Covid recover within two years.

GOSH celebrates its fifth academic training weekend

In early November 70 early career researchers came together for the fifth NIHR GOSH BRC Academic Training Weekend.