The Paediatric Therapeutic Development Workshops are a joint initiative between LifeArc, Innovative Therapies for Children and Adolescents with Cancer (ITCC), Cancer Research UK, Cancer Research Horizons and the Cancer Grand Challenges PROTECT team. The workshops bring together childhood cancer experts, including patient advocates, to help identify the most urgent needs and promising treatment ideas. The aim is to speed up progress by prioritising promising options, allowing the research community and funders to help drive future progress in a more streamlined way.

Before the workshop, participants received a detailed document explaining what we currently know about rhabdoid tumours and the research tools available (e.g. cell lines). They also received a list of drug targets and treatments currently in development.

Rhabdoid tumours usually involve changes in molecules called SWI/SNF. SWI/SNF is involved in restructuring a cell’s genetic material, so that certain genes can be switched on and off to control vital cell functions. This is known as chromatin remodelling. When SWI/SNF doesn’t work properly it can lead to cancer.

The group selected several targets found in rhabdoid tumours that should be prioritised for potential new drugs, many linked to chromatin remodelling. These included:

1. DCAF5: this molecule helps rhabdoid tumours survive by breaking down some SWI/SNF groups. Blocking DCAF5 may allow SWI/SNF to work normally again.
2. SMARCA4: this protein is thought to play a role in helping cancer cells grow and spread. Blocking SMARCA4 has stopped rhabdoid tumour cells from growing in the lab.
3. MDM2: a protein called p53 can help suppress cancer growth, but MDM2 breaks down p53. Blocking MDM2 could allow a build-up of p53, causing cancer cells to die.  
4. PDGFR and FGFR: these targets help cancer to grow and spread. They are found in higher amounts in rhabdoid tumours. Targeting them together could stop tumour growth and help kill cancer cells.

Another well-known target called EZH2 was agreed to be important in rhabdoid tumours. The group recommended testing combinations of drugs that target EZH2 in clinical trials. These combinations may work better than using a single drug on its own.

The outcomes of this workshop can now be used by the research community and funders to help drive focused progress, together. The response to the workshop has also shown how much the community values this approach. We now believe there is appetite to build on this work moving forward, incorporating the innovative approaches and tools developed through these workshops. Investigators can apply for further support from projects like C‑Further – a partnership between LifeArc, Cancer Research Horizons, and Great Ormond Street Hospital Charity – which focuses on developing new medicines specifically for children with cancer.