Childhood cancer research – what are the barriers?

September is childhood cancer awareness month, an important opportunity to increase public awareness about a collection of diseases that can devastate families.

Relatively speaking compared to adults, cancers are uncommon in children – around 1,600 children are diagnosed each year in the UK. But for the parents, families and children affected by these diseases statistics like this offer little comfort.

The good news is that thanks to research, more than eight in 10 children now survive cancer for at least five years. But a sobering fact remains – even with these improvements – children’s cancers are still the most common cause of death in children and the most common cause of death by disease in teenagers and young adults. And there is an urgent need for kinder treatments, along with new ways of dealing with the long term cost of surviving cancer in childhood.

So what’s being done to change this?

Today, Cancer Research UK’s head of policy development, Emma Greenwood, will be taking part in the European Society of Paediatric Oncology (SIOPE) international conference on children with adolescent cancer, in Brussels. She will highlight some of the challenges faced by researchers in setting up clinical trials for children’s cancers, and the work we’re doing to support them.

So we thought it would be a good idea to share some of the points that she’ll be raising at the conference.

Clinical Trials Regulations

The way scientists run clinical trials is governed by certain regulations, some of which are showing real promise in the fight against children’s cancers. The EU’s Clinical Trials Regulations were adopted by the European Parliament in April 2014, and will apply from late May 2016 at the earliest. These new rules will make it easier to set up trials across different countries, making trials more accessible to wider groups of cancer patients. Important changes as a result of the new Regulations include:

• A single European computer system – This will make authorising trials more straightforward, because researchers will only have to make a single application to all member states at once.
• Enabling more research into new uses for established treatments – This would mean that clinicians could do more work to understand how best to use the treatments that we already have, or find out how to use established therapies in new areas.
• Regular reviews – The legislation will be reviewed every five years to ensure that it is fit for purpose

This is why we we’re doing all we can to make sure they’re implemented to the planned timeline.

This is particularly important for children’s cancers, as the numbers of patients affected are small, meaning that trials need to be run internationally to recruit enough participants. We are working hard with our contacts in Europe to make sure that the Clinical Trials Regulations are put into practice as soon as they apply in May 2016.

European Paediatric Regulations

One of the challenges in boosting the development of new treatments for children’s cancers are a set of EU laws known as the ‘European Paediatric Regulations’, which were introduced in 2007. They set out with the goal of increasing the development of medicines for children by drug companies.

But instead, they’ve inadvertently created a loophole that means companies can potentially avoid testing their drugs in children.

The laws state that a company developing a new drug must think about the potential use of that drug in children – completing something known as a ‘paediatric investigational plan’ (or PIP for short). But since the laws were introduced, some drug companies have put this off, particularly for drugs targeting tumours not directly relevant to children.

For example, when developing a lung cancer drug a company could defer its paediatric plan, as lung cancer predominantly affects adults. But as we’re learning more about the genetic makeup of different tumours we are beginning to see similarities emerge that may mean a treatment for adult cancer could potentially target cancers with a similar genetic makeup in children (even though they didn’t develop in the same part of the body).

Sadly, a recent study found that the practice of delaying trials of certain drugs for children’s cancers was widespread. Of 28 drugs approved for adult use in Europe since 2007, researchers found that 26 of these could potentially be relevant in children’s cancers, but half had not been pursued because the adult condition did not occur in children. This means that new treatments that could be used in fighting children’s cancers are potentially being missed.

So what’s being done about this?

We know that regulators are looking at how to resolve this issue, finding new ways to make sure that manufacturers are testing new drugs in children’s cancers that could benefit. This is something we’ll be keeping a close eye on, and we’re actively talking to decision-makers in the European Union and looking for further opportunities to apply political pressure where we can.

Data Protection Regulations

We are also pushing European Parliamentarians to improve another piece of legislation called the ‘European Data Protection Regulation’, which will affect how researchers set up studies using collections of patient data from across Europe.

At the moment, researchers can access patient data, but they must pass through a strict process – subject to robust rules about how they handle the data – and rightly so. Individuals who do not want their data used in this way also have the right to object.

But there are some proposed changes in the pipeline for these EU regulations, which could affect how scientists carry out their research – something we explore in this blog post.

This would be really unfortunate – for some studies it’s not possible to seek individual permission to use each record (again, it’s worth reading this blog post for more detail). In other cases, the study needs lots of data to generate a reliable result, which would make it difficult to gain individual permission from lots of people. Alerting groups to their involvement may also introduce a bias to the study – something researchers need to avoid if they are to gather meaningful data.

As it stands, some of the amendments being made to the regulations could undermine research of this kind, and even the data registries themselves. We’re working actively with MEPs at the European Parliament to make sure that this doesn’t happen.

Our work to beat childhood cancers

Despite some of these political challenges, Cancer Research UK’s work to fund trials into promising new cancer treatments continues.

In order to increase participation in clinical trials, our Children’s Cancer Trials Team co-ordinates groundbreaking trials in 21 centres across the UK and Ireland, bringing new treatments to the young patients who need them.

Given the limited investment from pharmaceutical companies in children’s cancers, we are a major funder of childhood cancer research in the UK, supporting 60 per cent of all children’s cancer trials.

We recently launched our new strategy that sets out our priorities for the future. In this, we highlighted our commitment to doing more to beat cancers in children, teenagers and young adults. We are also working with the children’s cancer research community to spark proposals for high-quality new research projects that we hope we can fund in the future.

Our work has played a big part in the progress made in beating children’s cancers to date. But this all requires a sharp political eye to keep things on track.

We believe that through increasing our focus on this important area, campaigning in Europe for the right regulations to make it easier to set up and recruit to trials, we will bring forward the day when all children’s cancers are cured.

And that’s a statistic that can’t come soon enough.

Phil Reynolds is a Policy Adviser at Cancer Research UK