Historically, research carried out has been down to what researchers and doctors think is important to learn more about and what funding is available. Perhaps they think patients only want new drugs or more follow-up scans to feel reassured that they are clear of cancer. However, if they don’t talk to patients and their families, it’s presumptuous to assume that they know what patients feel would help them most.
An example of this is at my former hospital. One of the rooms on the cystic fibrosis ward had a tiny, prison-like, window at the top of the wall. A generous donor understandably thought that this room would be miserable to spend much time in. So, they donated a huge backlit picture of a tropical island that nearly covered the wall. The idea was that it would brighten up the room and improve a patient’s hospital stay.
But the picture made the room sweltering, because of the backlight, and reminded me that I was stuck in a hospital room and not in any of the many other places I would rather be. I like to think that if the donor had spoken to inpatients, then they would have said what actually would have made a difference eg. a microwave to prepare food, care packs containing forgotten necessities or a good WiFi connection. I’m not sure many patients staying in that room would think that a wall-sized light-up picture is a good idea when other things are perhaps needed more.
That’s why it is so important to include the thoughts and feelings of the people you are trying to help. Many of CCLG’s cancer research projects now include patient and parent representatives, which you can read more about in my ‘Can you change the future? We think so.’ blog, but that’s normally after the project has been funded and the research question decided.
The Priority Setting Partnership
What is needed is parents and patients’ opinions right from the start, helping to shape expectations for research and decide the most important issues facing children with cancer.
CCLG and The Little Princess Trust recognised this need for input from people with lived experience of childhood cancer. They funded the Children’s Cancer Priority Setting Partnership, whose goal was to go through a rigorous process of surveys, interviews and workshops designed by the James Lind Alliance to find the top priorities for childhood cancer research.
Young patients, childhood cancer survivors, parents, carers and family members, as well as cancer professionals were invited to participate in this process and there was a special emphasis on talking to young children to make sure their opinions were heard.
We’ve now reached the end of this two-year process, and the launch event for the top ten childhood cancer research priorities was held earlier this week. For the first time, researchers know what is important to children with cancer and know where to focus their work.
However, this doesn’t mean that research projects funded before the announcement looked at non-essential issues – far from it. One of the most exciting things to me was finding that around 90% of our research projects are already researching these ten priorities in some way. The remaining ten percent of our research projects are trying to learn more about different cancer types, forming the foundations for projects that will directly address the ten priorities. It's fantastic to know that we are on the right track, and that our research is making a difference.
The top ten priorities and our research projects
I wanted to give you some examples of what some of these research priorities look like in terms of real-life research projects. All of the projects below were funded through CCLG, either through our Special Named Fund programme or our partnership with The Little Princess Trust, before the top ten priorities were announced. They all cover one (or more) of the biggest problems facing children with cancer.
Can we find effective and kinder (less burdensome, more tolerable, with fewer short- and long-term effects) treatments for children with cancer, including relapsed cancer?
Funded by the Little Princess Trust (LPT) in partnership with CCLG, Dr Carmela De Santo is looking at whether starving leukaemia cells could help fight blood cancer. Her project is called ‘Targeting nutrient use by childhood blood cancers’. Cells need nutrients, called amino acids, to survive. Previous research has shown that cancer cells use different types of amino acids than healthy cells, and that removing the supply of an amino acid called arginine could kill a range of adult and childhood cancer cells with very few side effects for the patient. In the project’s first year, Carmela has already found a medicine to stop leukaemia cells absorbing several amino acids that the cells are reliant on for growth. She is now testing the medicine to see whether it is more effective in combination with other chemotherapy drugs. Carmela hopes that using her new medicine in combination with existing chemotherapy might make lower and safer chemotherapy doses just as effective at killing cancer cells as high doses of the chemotherapy drug on its own.
Why do children develop cancer (including the role that genetics plays) and could it be prevented?
Scientists think that neuroblastoma begins in embryos in their mothers’ wombs, when a group of embryonal cells become cancerous due to changes in their DNA. Sometimes these changes cause there to be a lot of MYCN proteins in the neuroblastoma cell. It is very difficult to successfully treat patients whose neuroblastoma has a lot of MYCN proteins. The first step to creating effective treatments is learning more about the protein. Dr Helen Bryant’s LPT project, called ‘How does neuroblastoma begin and can we use this knowledge to develop new treatments?’, is working to find out more about MYCN. She is growing early embryonal cells in her lab and increasing the MYCN present until the cells become cancerous. The aim is to find out how different amounts of MYCN change neuroblastoma’s development. Helen hopes that the team can find a medicine that target’s MYCN’s effects, and that their findings might lead to better and kinder treatments.
Are the psychological, practical, and financial support needs of children with cancer, survivors, and their families being met during treatment and beyond? How can access to this support be improved and what further support would they like?
It can be difficult to know how children feel during cancer treatment. Doctors need to not only consider their physical health, which children might not know how to communicate, but also other factors like their mental health and whether they can do the activities that are important to them. Dr Madeleine Adams wants to fix this problem with her project, titled ‘Identifying the support needed by children with sarcoma’. She is developing a questionnaire that doctors can use to measure a child’s quality of life whilst going through treatment. Parents and patients have been involved at all stages to make sure that the specific challenges they faced during treatment are considered. The tool, called SAM-Paeds, is already being used as part of an international clinical trial for rhabdomyosarcoma. Madeleine also hopes that the questionnaire will be used in clinic appointments, as well as in research studies, to identify children who need extra help.
How can we speed up the process of getting diagnosed and starting treatment in the right place?
The process of making sure a patient receives the correct diagnosis in order to start the right treatment can take a while from when the child is first referred. One example of this can be seen in children with kidney tumours where some have more complicated needs. Dr Tanzina Chowdhury at Great Ormond Street Hospital spent two years working on her LPT-funded project, called ‘"Getting it right first time" for children with renal tumours’. The goal is to make it easier for doctors to see which children need stronger treatments earlier on. Her work has created a national library of children’s scans, showing when the kidney tumour was diagnosed and what happened to it after treatment. Researchers are now looking at whether artificial intelligence software can highlight the correct children, from the database of scans, who need different treatment strategies at diagnosis. Tanzina’s project also supported the development of a national advisory panel for kidney tumours, made up of kidney tumour experts who can advise local doctors on the best treatment choices for each patient at diagnosis and relapse. By the end of the project, the expert panel had helped decide the right treatments for 190 children.
Why do children relapse, how can it be prevented, and what are the best ways to identify relapse earlier?
Researcher Dr Karen Keeshan’s project is called ‘Using genetics to understand why acute myeloid leukaemia cells don’t respond to treatment’. In this LPT-funded project, she is using genetic analysis to find out why some acute myeloid leukaemia cells, called leukaemic stem cells, survive treatment and lead to relapse. Using bone marrow samples taken during a current clinical trial, she is looking at which genes are active in each leukaemic stem cell that didn’t respond to treatment. Her team will look at how the stem cell changes as the cancer progresses, then use their genetic analysis to find drugs that will kill the cells that survive treatment at each stage. Karen hopes that this will help design more precise and targeted treatments for this aggressive type of leukaemia that will better treat and prevent relapse.
If you want to learn more, you can read about the top ten research priorities for childhood cancer and take a look at our research projects.
Ellie Wilkinson is CCLG’s Research Communication Executive.
She is using her lifelong fascination with science to share the world of childhood cancer research with CCLG’s fantastic supporters.
You can find Ellie on Twitter: @EllieW_CCLG