Putting the child first when treating childhood cancers

Innovate UK’s Cancer Therapeutics programme has three key objectives: to provide support to commercially-led R&D; to provide innovative products and services more quickly and effectively into healthcare ensuring faster access to transformative solutions for patients; and to create growth in the UK cancer therapeutics sector, including childhood cancer and young persons’ cancers.

As part of the programme we have worked with the venture creation specialists Deep Science Ventures to explore the landscape of therapeutic approaches in paediatric oncology. The resulting white paper, Revolutionising Paediatric Cancer Treatment, which launched in February 2024, highlights opportunities with potential to attract the investment needed to deliver life-changing therapies for children with cancer.

Globally, around 400,000 children and adolescents are diagnosed with cancer each year and approximately 80,000 lose their life. Thanks to medical advancements, around 85% of children diagnosed with cancer now survive beyond five years. However, the physical, social and emotional impacts on patients, families and communities are far reaching and long term.

Children are not mini adults

Children and adults are very different when it comes to the effectiveness of treatments. Children’s vital organs and their neurocognitive abilities and personalities are still developing. It is a difficult balancing act to give children an adequate dose of chemotherapy to cure their cancer, while limiting toxicities. The side-effects from these harsh treatments themselves, which were mostly developed for adults, can often lead to long-term health issues, including damage to organs, hormone function and fertility. Secondary cancers may also be caused by the chemotherapy that put their original cancer into remission. All of this together can lead to profound socioeconomic problems and contributes to a reduced capacity to thrive and contribute to society.

Small patient population

While cancer is the largest cause of death by disease in children and adolescents, it still represents a relatively small patient population. Within this group, there are different types of cancer: leukaemia, central nervous system cancers, lymphomas. When you’re thinking about targeted therapies, the diversity of these cancers means clinical trials must be tailored to each specific type, further narrowing the patient populations available for these trials.

It’s notoriously difficult to get the data that you need from clinical trials with such small populations. More of an international effort is needed to generate larger trial groups and this is starting to happen. Some clinical trials can now be run in a more remote manner, so patients don’t always have to go into a centre for appointments. This should support the inclusion of children into trials as much of the monitoring can be done within the child’s home.

As well as the small patient populations leading to small clinical trial populations, the ethics of running any trials in children play a big part in the reluctance of many organisations to specifically target children.

Opportunities for UK businesses

These difficulties have inevitable consequences. Of the 300 or so cancer treatments licensed around the world, fewer than 10 are specifically designed for children. To put this into context, there are over 1,000 new oncology drugs in clinical trials at the moment, yet only a handful of these are being evaluated for the potential to treat childhood cancers.

There are huge opportunities for UK businesses. The white paper shows the scale of the opportunity to create new business models for underserved areas such as paediatric oncology by taking a child-first approach.

By detailing the mechanisms that could be explored, the white paper highlights that a child-first approach doesn’t mean a child-only approach. This consideration of the wider market for treatment options will make investment more appealing, rather than ideas stalling for a perceived lack of return.

Reasons for optimism

It feels like we’re on the cusp of building something that will be transformative for survivors of childhood cancer. With the opportunities outlined by Deep Science Ventures, new partnerships such as the C-Further consortium launched by medical charities LifeArc and Cancer Research UK, as well as the diversity of groundbreaking ideas we are funding, mean there are a lot of reasons to be optimistic.

Innovate UK’s funding is accelerating the delivery of medicines that are uniquely designed for children with cancer. It encourages the collaboration of exciting UK businesses and university spinouts with leading edge researchers and specialist paediatricians. Projects include the advancement by Medannex of a first-in-class therapy to treat a type of bone cancer known as paediatric osteosarcoma, and a novel mRNA-based cell therapy for childhood brain cancer that is being developed by the UK company Coding Bio.

As well as these remarkable approaches to deliver much needed treatment options, we are also funding projects to better tailor existing chemotherapies for children such as DosoLogic from Vesynta, a UCL spinout company.

They are developing a software platform that analyses children’s responses to chemotherapy in real time. By doing this, healthcare professionals will be equipped with the tools to prescribe safer and more effective chemotherapy doses for the individual child, improving their long-term quality of life through minimising toxicities while ensuring an effective dose to put the cancer in remission. While not a new therapy, if successful, it will have a significant positive impact on the futures of childhood cancer patients.

New ways to new ventures

Our work with Deep Science Ventures is all about taking challenging problems in areas of unmet patient need and focusing on those problems to come up with a solution that forms the basis of a commercially viable venture. By focusing on the challenge and bringing the right people to the table to create solutions we can develop something that is investable and novel. Such approaches complement more mainstream pathways in the translation and commercialisation of academic research.

We are moving towards a new era of targeted, more effective, safer medicines for everybody: children, adults, underserved populations, people of every socio-economic status and ethnic background. Cancer therapeutics are just the start…