Research Q&A with Professor Deb Tweddle

Since 1997, we’ve supported 68 neuroblastoma projects nationwide and have invested over £7 million to research the disease. During Childhood Cancer Awareness Month 2021, we invited our supporters to share their research questions with us.

Professor Deb Tweddle is Professor of Paediatric Oncology at Newcastle University and an Honorary Consultant in Paediatric and Adolescent Oncology at the Great North Children’s Hospital, Newcastle. She’s also been a Trustee of Neuroblastoma UK since 2010.

Thank you to everyone who asked a question and to Prof. Tweddle for providing answers.

1. Is there anything "we" can do, as in we adults, i.e. as in a donation of some kind, if matched?

For most children with neuroblastoma, their own healthy stem cells are used for a stem cell transplant. But your matched stem cell or blood donation may help many others with blood cancers, blood disorders or people needing bone marrow transplants.

You can join the stem cell register or join the blood donor list.

2. Is there any evidence to show the difference in relapse rates and survival rates between those who go through protocol treatment without complication and those who have to deviate from ‘the plan’ for alternative treatments?

Interesting question. Children with refractory neuroblastoma i.e. neuroblastoma that doesn’t respond to first or second line treatments, have an increased chance of relapse.

But our own recent research suggests that at almost 50% go on to complete treatment. Around 50% of children with high risk neuroblastoma will relapse regardless of whether they needed any additional treatment.

3. Are there any new medicines or treatments discovered for higher grade neuroblastoma?

ALK (anaplastic lymphoma kinase) genetic abnormalities (mutation- mistake or amplification- lots of extra copies of the gene) are associated with poorer outcome in patients with high risk neuroblastoma, particularly  ALK amplification.

Fortunately, there are now new treatments that target ALK called ALK inhibitors and these are currently given to children with relapsed or refractory neuroblastoma and ALK gene abnormalities. In the very near future, they are going to be given to children at diagnosis in addition to standard treatment as part of the next high risk neuroblastoma trial (HRNBL2).

4. Why doesn’t neuroblastoma get more funding for research? 

Around 100 children are diagnosed with neuroblastoma every year in the UK. This makes it a ‘rare’ cancer. Because of this, sadly research into neuroblastoma doesn’t get as much funding compared to other cancers. The relatively low numbers also make it hard to conduct meaningful clinical trials.

That’s why your support is so important. Your donations have made a clinical trial for children with high-risk neuroblastoma available in the UK this year, allowed us to continue to fund vital pre-clinical studies and will go towards our latest research grant round focussing on international collaboration and immediate clinical impact studies.

5. How much money do we need to raise to find a cure? How long will this take?

Finding a cure for neuroblastoma needs billions of pounds of investment in research and will take decades. Neuroblastoma UK has provided funding of more than £7 million for neuroblastoma research over the last 25 years alone. This has made steps towards understanding this disease in more detail and made small inroads towards our goal of finding kinder treatments and eventually a cure.

But much more needs to be done. We’ve seen how significant investment in research can make a real difference, with the development of a COVID vaccine or the progress with treatment for children with leukaemia. We need continued and dedicated research funding to make breakthroughs in neuroblastoma treatment.

6. Why do families have to travel to America for a vaccine? Why isn’t it available here?

A number of research groups in the U.S. are running clinical trials to see whether giving children the bivalent vaccine or DFMO can further reduce the chance of relapse for children with neuroblastoma. Families choose to join this trial and travel overseas as it isn’t running in the UK. 

Although the use of vaccines or DFMO have been found to reduce the risk of relapse, we don’t know if children who received them wouldn’t have relapsed anyway as the trials in the U.S. weren’t randomised trials. Until we do a randomised trial where half of children receive the vaccine or DFMO and the other half don’t, we won’t know if they have made a difference to the chance of relapse.

We do know that the relapse rate for children who complete all the current treatments for high risk neuroblastoma, and are in complete remission at the end of therapy, have the same chance of relapse as patients who have had DFMO or vaccine.

Many parents find the uncertainties at the end of their child’s treatment very difficult. An information sheet has been produced to provide the best available assessment of relapse rates for children who successfully complete all standard UK treatment for high-risk neuroblastoma. It also provides more information about the DFMO / Bivalent vaccine.

Read more: Outcome after high-risk neuroblastoma treatment in Europe (opens in new window)

If you have any questions about any of this information, please do speak to your child’s consultant.