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Neuroblastoma progress – outcomes from our research symposium

Friends of Rosie, now known as the Children’s Cancer Research Fund (CCRF), runs an international research symposium in alternate years. In September 2024, over 40 people gathered together in Manchester for the two-day scientific events titled, Theragnostics for neuroblastoma: making molecular radiotherapy work better. Here our Scientific Advisory Board Chair, Dr Mark Gaze, shares a summary of the day and what it has achieved.

The high-risk form of neuroblastoma is a cancer of unmet need, as despite improvements in outcome in recent years, around half of those affected die of this cruel disease. Radioactive drugs have an established place in both the diagnosis (nuclear medicine scanning) and treatment (molecular radiotherapy) of neuroblastoma. The word theragnostics unites these two things, which in a way are like two sides of a coin.

The symposium attracted a wide range of individuals, including paediatric oncologists, nuclear medicine physicians, clinical scientists, radiologists, therapy radiographers, cellular, molecular and radiation biologists, surgeons, and importantly, parents of children with neuroblastoma. They came not just from the United Kingdom, but also the United States, Sweden, France and the Netherlands. In turn, they presented the state of the art in their field of expertise, discussed how concepts presently in research might be introduced into everyday practice, and what clinical trials might evaluate innovative approaches to refine and improve treatments. Networking and informal conversations continued in refreshment breaks and in the evening. New friends were made, and new collaborations started.

There is some real progress this side of the horizon. Two new clinical trials of molecular radiotherapy for children with neuroblastoma are expected to start recruiting patients in the UK in 2025. One is MINT, a trial to see whether the addition of a drug called talazoparib can enhance the efficacy of iodine-131 meta iodobenzylguanidine (mIBG). The other is LuDO-N, a trial to see whether a more dose intense schedule of administration of lutetium-177 DOTATATE (LuDO) has better results than the less intense schedule evaluated previously.

Slightly further in the distance, it is hoped that the development of a novel imaging radiopharmaceutical iodine-124 mIBG, used with the new generation of high sensitivity scanners being installed presently in a few UK centres, will allow refinement and optimisation of mIBG therapy. Much work is underway to take this forward, and it is hoped that a trial may start in 2026.

By bringing together scientists and clinicians from different but inter-related disciplines, and linking together key players from different countries, the symposium has facilitated development work on these three projects. We anticipate they will be run internationally, thereby bringing forward the completion date of each, for the benefit of children with neuroblastoma.

A detailed report has now been published in the high-impact factor American Journal of Nuclear Medicine. If you want to read more detail, you can find it at: https://doi.org/10.2967/jnumed.124.269121

Professor Mark Gaze
Chairman, CCRF Scientific Advisory Board

Nancy Dykes

Nancy Dykes

Nancy, 11, was diagnosed with rhabdomyosarcoma when she was just 2 years old. She was treated with Proton Beam Therapy (PBT) in Germany but unfortunately relapsed less than a year later. The family were then sent to Germany for a second round of PBT. Although treatment helped to cure Nancy’s cancer, she has been left with lots of lasting effects which will require ongoing treatment throughout her life. Here Nancy and her mum, Antonia, share their story in the hope it will help others, raise awareness, and raise more money for children’s cancer research.

Prof John Hickman

Prof John Hickman

Why I am a Trustee of CCRF
 
I am a relative of the Larkin family and knew Rosie Larkin ( Friends of Rosie, now CCRF) when she had and then died of neuroblastoma, aged six. At that time, I was a scientist and professor at Manchester University, actively working on the problem of why some cancers do not die despite intensive and toxic therapies.  Seeing Rosie during her unsuccessful therapy and then attending her funeral, besides being emotional, drove me to double down on my own research as well as to support the Charity as best I could as a cancer researcher. I spent over a decade as the Chairman of the Scientific Advisory Board of the CCRF, when the Board moulded policies to ensure that funds raised for research into childhood cancers were distributed only to clinicians and researchers who had the brightest ideas that could impact of cancer medicine for children.
 
My role on the Board
 
As a Trustee,  I hope that my many decades of experience in cancer research and medicine can help the CCRF determine where best the funds that are raised can be invested in cutting edge research and medicine. I am also keen that the CCRF brings together UK and international experts in science and cancer medicine to brainstorm on how best to advance the treatment of childhood cancers.
 
My Background
 
I was a Professor of Pharmacology at Manchester University, working on drug resistance in cancer. I then moved to Paris, where I still live, to head cancer drug discovery at a French pharmaceutical company (Servier). I then headed an EU consortium for six years that brought together researchers from the pharmaceutical industry, universities and biotechnology companies, from all over Europe and the UK, to improve cancer drug discovery. I continue to be active in cancer health policy, publishing in the medical literature.
Katrina

Katrina London

Why I am a Trustee of CCRF

I have spent my professional career representing individuals and families affected by the devastating asbestos-related cancer, mesothelioma, which has no cure and for which there are only a limited number of palliative treatments available on the NHS.

I joined CCRF because I wanted to bring my understanding of the need to raise awareness of less well known cancers and passion for the need to secure improved funding for research to children facing a cancer diagnosis.

My role on the Board

I am responsible for oversight of our collaboration agreements with other charities to co-fund research into cures and treatments for childhood cancers. I also manage our challenge events and help with community fundraising. 

My Background

I am a Senior Associate at Irwin Mitchell LLP and specialise in Asbestos Disease Litigation, acting on behalf of victims of asbestos related diseases and their families.  Over the years, I have undertaken a number of fundraising challenges to raise money for cancer research, including a 1000 mile cycle ride from Glasgow to Southampton.   

Carla

Carla Nuttall

Why I’m a Trustee of CCRF
 
I became a trustee of the Children’s Cancer Research Fund because I believe every child deserves the chance to grow up healthy and with everything to look forward to in life. Like many people, I’ve seen the impact cancer can have on families and I wanted to do something that could make a real difference. This started through my work as an ambassador for Cyclists Fighting Cancer, which supported young people and their families in finding strength, confidence, and joy through movement.
 
That experience shaped my understanding of the challenges families face and the importance of community-led support – but crucially, the clear need for that to go hand-in-hand with targeted research and long-term solutions.
 
This is why I’m so passionate about the work CCRF does and proud to contribute to a cause that gives children and their families, the greatest chance of living a life without limits.
 
My Role on the Board
 
I support CCRF’s mission by bringing my experience in brand, marketing, and communications to the board. My focus is on helping CCRF tell its story in a way that resonates with people – whether that’s families, researchers, donors, or the wider public. I work alongside an incredible team to strengthen CCRF’s voice, build trust, and grow awareness so that more children and families can benefit from the breakthroughs we’re helping to fund.
 
My Background
 
I’m the Executive Director of Corporate Affairs and Marketing for The Growth Company, a social enterprise dedicated to helping people, businesses, and places to thrive through its market-leading insight, employment, skills and business support.
My passion is to help the organisations I work with to communicate with clarity, purpose, and integrity. That’s why I’ve spent my career in strategic communications supporting organisations that want to make a positive difference – CCRF could not fit my sense of purpose any better.