Three trials due to start thanks to increase in network's funding

The extra funding that The Little Princess Trust provided to Experimental Cancer Medicine Centres (ECMCs) across the UK led to a new site being added to the network in Cardiff.

In an interview below, first published on the ECMC website, those working in the Welsh capital explained what has been achieved since the latest five-year funding period began in April of last year.

Answering the questions are Madeleine Adams, Lead; Ceri Hogg, Research Nurse and Rhian Thomas-Turner, Centre Business Lead, from the team in Cardiff.

Firstly, can you explain why early phase trials are so important for paediatric cancer patients and their families?

Early phase trials offer access to innovative medicines, giving families choices at a time when their child has relapsed, or no further standard treatments are available.

For many this can be a welcome option and whilst many trials may not offer a cure it gives children the opportunity to be involved in studies that support the progression towards better treatments and survival outcomes.

Children are offered to participate in national or international clinical trials where possible, all with the aim of improving outcomes for children.  

What do you see as the main impact of having a Paediatric ECMC in Wales?

Prior to being accepted as a Paediatric ECMC, children and families who wanted to participate in early phase research studies would invariably have to leave Wales to do so. 

As early phase trials in paediatric oncology are most commonly offered to children once all standard of care options have been exhausted, rather than a starting point for treatment, this meant that most children were having to travel outside of Wales when they were already very vulnerable. 

In addition, some families were not in a position to go to other centres due to the significant financial costs such as travel, accommodation and possibly loss of household income, associated with long distance travel and long stays away from home. 

For our families, the biggest impact will be easier access to these trials and also being able to access them closer to home.

Being recognised as a Paediatric ECMC also supports the Children’s Hospital for Wales reputationally. We have demonstrated a standard in research delivery that hopefully encourages sponsors to place studies with us, which in turn benefits our patients, increasing the trial opportunities available to patients in Wales.

What has ECMC funding meant to the activity you deliver in Cardiff?

Prior to receiving funding from the ECMC, the Welsh children’s cancer charity LATCH, supported us with short term funding to allow us to begin building the Early Phase service. 

The ECMC funding is enabling that work to continue, with the knowledge that the funding will be available for the next 5 years. 

That funding supports a designated specialist team who have expert knowledge on establishing and running trials for paediatric cancer patients. 

Being part of the ECMC Paediatric Network also opens up networking and collaboration opportunities with our colleagues across the UK.  

It allows us to work alongside experienced colleagues in paediatric early phase trials nationally to improve equity of access to trials, and also encourages pan-age collaborations with adult colleagues in the Cardiff ECMC and beyond.

Why is being part of such a collaborative network like the ECMC Paediatric Network so essential?

The ECMC Paediatric Network opens up opportunities to learn from national colleagues and to collaborate on future work. 

Due to small patient numbers, collaboration is vital in paediatric research and this network has created a space for ideas to be discussed and acted upon. 

Also, as a new member of the network, we are also provided with the opportunity to learn from larger established centres, to benefit from support when opening trials, so that high standards are maintained nationally when delivering care.

Can you let us know about a trial/some of the trials that have opened in Cardiff over the past year?

Since joining the ECMC in April 2023, we have three trials that are in set-up. These have been chosen to cover a broad range of diagnoses:

· DETERMINE, a multi-drug, precision medicine trial for adults, teenagers and children with rare cancers;

· CARE, A Phase 1/2, Tolerability, Study of Repotrectinib in Paediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK1-3 Alterations

· LIGHTBEAM, a Phase 1/2 Study to Evaluate the Antitumor Activity and Safety of Investigational Agents in Children and Young Adults with Haematologic Malignancies or Solid Tumors

We hope to be opening these trials over the next few months

What do you see as the biggest opportunity and challenge in paediatric oncology over the next 5 years?

Cardiff is in a unique position in that we sit within a health board that treats a wide age range of patients, has an onsite Teenage and Young Adults (TYA) unit and has close collaborative links with Velindre NHS Trust, where adult oncology patients are treated. 

This gives us great opportunity to open and collaborate on pan-age studies.  Now that we have better opportunities for our paediatric patients we want to ensure that we increase the accessibility of research the Welsh TYA age group. 

As with most plans, the challenge is funding adequate resources to achieve this aim but we have clear plans and ambitions set out to facilitate this.