GOSH Charity National Paediatric Cancer Clinical Trial Fund

Funding to support early phase clinical trials and translational work packages to develop therapeutics for hard to treat paediatric cancers with unmet need.

The GOSH Charity National Paediatric Cancer Clinical Trial Fund is a new funding initiative that will support early phase clinical trials to advance the development of therapeutics for hard to treat paediatric cancers with unmet need.

The fund supports the GOSH Charity Cancer Research Strategy (2024-2029), which aims to invest at least £15 million in paediatric cancer research and transform outcomes for children and young people with rare and hard to treat cancers. Up to £6 million is available for the 2025/26 call for applications, to support ambitious and innovative early phase clinical trials and associated translational work packages that have the potential to accelerate the testing and approval of new therapies for paediatric cancers with limited treatment options and deliver meaningful advances in paediatric cancer research.

Funding is available for:

1. Early Phase Clinical Trials

• Clinical trials up to phase II a/b to test new biologically targeted therapies are eligible for support, which should be designed to test the safety, tolerability, pharmokinetics and preliminary efficacy of a new treatment.
• Trials should aim to identify the appropriate dosage, delivery mode and administration schedule, assess potential side effects and gather initial date on the treatment’s effectiveness.
• Treatments should be novel, advanced and targeted.
• Trials can include standalone paediatric clinical trials, a paediatric arm for an existing adult trial where the cancer type spans the age spectrum, or a new UK paediatric arm of an existing international platform trial.
• Priority will be given to first-in-child or first-in-class treatments, and in areas such as immunotherapy, precision medicine, gene and cell therapy, vaccines, novel approaches and CAR-T therapy.
  

2. Translational Work Packages

• Translational research work packages linked to an existing trial are eligible for support.
• Work packages must directly support the potential of a clinical trial to move through the research pathway.
• Proposals could include, for example:
  • Biomarker development – to identify and validate biomarkers that predict patient response to treatment, disease progression or relapse, for example to help tailor therapies to individual patients and improve trial outcomes.
  • Adaptive trial designs – using insights from translational research to inform adaptive trial designs and modifications to the trial procedures based on interim results. This can accelerate the trial results by making it more flexible and responsive to emerging data.

Proposals should focus on hard to treat paediatric cancers and those with the greatest unmet need:

Hard to Treat Cancers:

• Cancers with low or poor survival rates (ie a survival rate below 50%).
• Cancers that have returned or have not responded to initial treatment (relapsed and refractory stages).

Cancers of Greatest Unmet Need:

• Paediatric-type high grade glioma.
• ATRT, ETMR and other embryonal brain tumours.
• Osteosarcoma.
• High-risk neuroblastoma.
• Rhabdomyosarcoma.
• Ewing sarcoma.
• Desmoplasia small round cell tumour.
• AML.
• High-risk ALL.
• Ewing-like sarcoma.
• Medulloblastoma.
• Ependymoma.
• Other sarcomas.