Children’s Cancer and Leukaemia Group (CCLG) and Alice’s Arc are proud to announce a new research project that will help support families and clinicians with treatment decisions for relapsed and refractory rhabdomyosarcoma.
Rhabdomyosarcoma is the most common soft-tissue cancer in children. Although most children with rhabdomyosarcoma can be treated successfully, if their cancer comes back after treatment (relapsed) or doesn’t respond to initial treatment (refractory), the odds of survival drop considerably.
For children with relapsed and refractory rhabdomyosarcoma, further treatment may not exist and treatment offered may not be successful. This means that families face complex and difficult decisions about how to approach their child’s care.
Living-REFoRMS project
Titled ‘Living-REFoRMS’, the project aims to create a ‘dynamic’ online resource for families of children with relapsed or refractory rhabdomyosarcoma, as well as clinicians, that will be kept up to date with the latest information about new clinical trials and treatment options available. This pioneering project is funded by Alice’s Arc and CCLG Special Named Funds including Angus’ Door, The Jenni Clarke Fund, Ollie’s Star, Team Jake, Pass the Smile for Ben, Jacob’s Join, Hattie’s Rainbow of Hope Appeal and Super Ruby’s Rhabdo Raiser.
Living-REFoRMS is led by Dr Jess Morgan from the Centre for Reviews and Dissemination at the University of York. It builds upon the work of the original and ongoing CCLG-funded REFoRMS project, which investigates how families make decisions when their child has relapsed or refractory rhabdomyosarcoma and assesses the effectiveness of new treatments.
The new study takes the form of a ‘living’ systematic review where evidence is regularly searched, assessed and reported – as opposed to the usual one-off review - and is the first of its kind in childhood cancer. This regularly updated information will be available to families and clinicians through CCLG and Alice’s Arc, which will make it easier for them to keep informed.
New treatments are regularly being researched and developed for relapsed and refractory rhabdomyosarcoma. Therefore, it is crucial that the information is the most current available and evaluation of completed research and open trials around the world is an ongoing process.
Dr Morgan said:
“Living-REFoRMS is the first-ever living systematic review in children’s cancer, which is really exciting.
“Relapsed and refractory rhabdomyosarcoma is a really good subject for a living review because early phase trials in relapsed and refractory disease happen quite quickly and there’s lots of new data and findings to update.
“Having a review that updates every time something changes is very helpful to be able to make sure doctors and families can keep up to date.”
Alice's Arc
Alice’s Arc was founded by Sara and David Wakeling to raise funds for research into rhabdomyosarcoma while their daughter, Alice, was on treatment for the disease.
Alice was first diagnosed with stage 4 alveolar rhabdomyosarcoma, aged three, in March 2015.
Following 20 months of chemotherapy at Great Ormond Street Hospital (GOSH) and proton therapy in the USA, Alice spent one year in remission before her cancer returned in February 2018. She passed away, aged seven, in October 2019, over four and a half years after her diagnosis.
Sara said that part of Alice’s Arcs’s research mission is to invest in research that will lead to more treatment options at the point of relapse and to improve the experience for families when faced with the prospect of relapse.
As parents who have experienced relapsed rhabdomyosarcoma, Sara and David understand how hard it can be to make decisions about treatment at this time and believe that having a resource available to equip clinicians and parents better when making these choices will have a profound impact on families in the future.
Sara said:
“The point of relapse is really complicated, because it raises lots of questions like if my child only has this very small chance of survival, what kind of treatment do I want to put them through?
“A goal of this project is to create a high-quality, regularly updated online resource to share the findings that will be accessible to families and clinicians, helping to support decision making in relapsed and refractory rhabdomyosarcoma.
“The impact of this kind of resource could be a real innovation in the provision of information, co-development of a treatment plan and a considerable support tool for families and clinicians.
“It’s a very tangible piece of work that could be high impact for families – it’s something they can actually use, it’s visible and it’s something that is meaningful to parents and families.”
CCLG Chief Executive Ashley Ball-Gamble spoke of the importance of collaboration between organisations to ensure resources are used in the most efficient way possible, to maximise the impact of research for children and their families.
He said: “It is fantastic to be working with Alice’s Arc to fund the Living-REFoRMS project. Working in partnership with other charities helps to fund important childhood cancer research projects faster than we could do alone.
“We look forward to seeing how the project develops and seeing the difference up-to-date, accessible information can make to children with relapsed or refractory rhabdomyosarcoma.”
Sara added: “We’re delighted to be working with CCLG to ensure that vital research into rhabdomyosarcoma can forge ahead – together, we are stronger.
“We all have a shared mission and pooling funds is a great way of ensuring important research into rhabdomyosarcoma is being undertaken.”
Dr Morgan thanked all those that have fundraised to help make this research possible and said that the project team is determined to use this opportunity to make a real difference to patients and their families in the future.
She said: “We really appreciate the time and effort that people put into raising this money, and we know how important that fundraising has been.
“We hope that the research we do will honour that effort and will provide answers to questions and information for families to improve treatment and care in the future.”
You can keep up to date with the research team’s work on Twitter @REFoRMS_Rhabdo