Research into neuroblastoma
Researchers are very interested in the diagnosis and treatment of neuroblastoma. There are a number of clinical trials for people to join in the UK. Many have their neuroblastoma treatment as part of a clinical trial.
Individual trials close when enough people have joined. There might then be a period of time before the results are available. New trials then open based on the lessons learned.
Amy: My daughter Poppy is a fun loving, adventurous, excitable eight year old child and she’s just lovely.
Poppy was diagnosed age three with a rare type of cancer called neuroblastoma, and we were completely devastated.
The prognosis for Poppy at that stage was a 20% chance of survival.
We were approached by Poppy’s cancer specialist to take part in a new trial. This potentially could prolong Poppy’s life.
The only thing that did sort of trouble us as a family when they did tell us was that it was possibly quite painful.
We were told at that time that they would manage the pain, that we would have somebody with us 24 hours a day.
Poppy’s treatment was an infusion into her bloodstream. With regards to pain and any symptoms that arose with Poppy, somebody was always there to make sure that she wasn’t in any pain or any suffering.
The trial for Poppy has enabled her to be a normal healthy child and without that who knows what position we would have been in now.
The doctors and the nurses and the specialists are all there to help ease any worries and concerns that you have.
If I could give anybody any advice it would be if you have any queries or concerns or worries then speak to your cancer specialist. That is their job. That is their role. That is what they do.
Diagnosing neuroblastoma
Researchers are looking into different types of scans. They are looking into which is better at showing where neuroblastoma has spread in the body.
Scientists are also studying blood samples and samples of neuroblastoma tissue. This is to look at changes inside the cancer cells. Researchers want to find out if they can choose the best treatments based on these changes.
Treating neuroblastoma
Doctors are looking into improving the treatment for high risk neuroblastoma.
This video shows one way that Cancer Research UK is supporting research into neuroblastoma.
Voiceover
Before we are born, stem cells divide and make new cells such as nerves, which can spread out from the spinal cord into the rest of the body.
In children with neuroblastoma however, they continue to divide when they shouldn’t, producing way too many cells and forming a tumour.
So what are they up to?
Meet MYCN and ALK. These two proteins have specific functions in our body and usually behave themselves.
In some cases of neuroblastoma though, these two proteins are thought to turn rogue, working together somehow to push the stem cells into making cells that can form tumours.
Professor Chesler at the Institute of Cancer Research in London, hopes that by using a model that recreates neuroblastoma in the lab, they can test new treatments which only target the excess cells created.
Across the country, Cancer Research UK is funding work like this, to better understand cancers affecting children and young people and to develop new, better and kinder treatments.
Chemotherapy
High risk neuroblastoma is difficult to treat, so researchers around the world are looking to improve treatments for children with this type of cancer. A large research study is looking at 3 main areas. The first is comparing 2 different chemotherapy regimens for induction treatment. This is RAPID COJEC and GPOH (a regimen used previously in Germany). Researchers want to know if one regimen is better than the other at helping children with high risk neuroblastoma. They will also be comparing the side effects of these treatments.
The second part is looking at consolidation treatment. The researchers want to know if it would be better to have 2 courses of high dose chemotherapy instead of one before a stem cell transplant.
The final part is looking at the amount of radiotherapy a child gets. Researchers want to see if adding a boost of radiotherapy to the normal (standard) dose of radiotherapy helps improve their outlook.
Targeted cancer drugs
Cancer cells have changes in their genes that make them different from normal cells. These changes can lead to the cell growing faster and working in a different way to normal cells. Targeted cancer drugs take advantage of this and target the specific gene changes the cancer cells have.
Researchers are looking at new targeted cancer drugs. This includes looking into different ways of giving them.
Lorlatinib is one of these new targeted cancer drugs. It is a 
. It is for neuroblastoma that has changes in the anaplastic lymphoma kinase (ALK) . Having changes to the ALK gene can cause neuroblastoma cells to grow and behave abnormally as cancer cells.
Researchers want to know:
- whether lorlatinib works best with or without chemotherapy
- the best amount of lorlatinib to give
- more about the side effects
Researchers have been looking at a drug called bevacizumab in the BEACON trial. They wanted to find out if it could help children with neuroblastoma. Bevacizumab is a type of targeted cancer drug called a monoclonal antibody. It targets the tumour’s blood supply.
Results from this phase 2 trial showed that bevacizumab with temozolomide and irinotecan may help treat children with refractory and relapsed neuroblastoma. More research is needed to understand about the different combinations of drugs with bevacizumab. Researchers are now working towards opening the second BEACON trial.
Immunotherapy
Immunotherapy uses the immune system to fight cancer.
A new treatment is looking at using anti-GD2 T-cells. T cells are part of the immune system and help fight infection. Researchers are taking immune cells from the blood. They then change them in the laboratory so they can attack the cancer cells. They change them by putting a gene into them. Genes are coded messages that tell cells how to behave. Doctors hope the new gene will help the T cells to recognise and attack the cancer cells.
Another study is looking at giving 2 immunotherapy drugs at the same time. They are looking at ones called nivolumab and dinutuximab beta with mIBG treatment. This is very early research. The aim is to help children with neuroblastoma that continues to grow during treatment. Or for those whose neuroblastoma has come back. This trial is called MiNivAN.
Researchers are also looking into a monoclonal antibody called naxitamab in combination with granulocyte macrophage colony stimulating factor (GM-CSF). GM-CSF is a type of . This early research is looking at treating children who have high risk neuroblastoma whose treatment isn’t working.
The researchers want to find out:
- how well this treatment works for this group of children with neuroblastoma
- about the side effects
- the safest amount (dose) of naxitamab to give
Radiotherapy
Radiotherapy is a common treatment for neuroblastoma. It uses high energy x-rays to kill cancer cells. Doctors plan radiotherapy treatment very carefully. They aim the treatment so that cancer cells get a high dose of radiation. The healthy cells surrounding the tumour receive a lower dose. But this can be hard to do using conventional radiotherapy.
Researchers have developed a new type of radiotherapy. It is called Intensity Modulated Arc Therapy (IMAT). This allows radiotherapy to be directed more accurately at the cancer. Doctors think that using IMAT means that they can give a higher dose of radiotherapy to the cancer. They hope that it will be better at killing the cancer cells.
Researchers are also looking into a type of radiotherapy called proton beam therapy (PBT). Proton beam radiotherapy is a type of . The proton beams are aimed precisely at the cancer. This type of radiation reduces the risk of damage to the surrounding healthy tissue and organs. They want to see how well this works in treating children with neuroblastoma.
Blood and bone marrow tests
A recent study looked at particular protein markers found in the blood at diagnosis. The study team concluded that this test can help show which children have high risk disease. They were looking at it for children with stage 4 neuroblastoma.
Children with high risk disease might not respond as well to standard treatment. Knowing this early on could help doctors decide to give newer treatments sooner. We need more research to see how helpful these blood tests are.
Other treatments
Arginine is an important protein that helps cancer cells grow and divide. Researchers are looking at a new drug called pegylated recombinant human arginase (BCT-100). BCT-100 is a drug that can lower the amount of arginine. Doctors hope this will help starve cancer cells and stop them from growing.
Research into side effects
Researchers are also looking into the long term effects of treatment. They are doing this for all types of childhood cancer. The Centre for Childhood Survivor Studies is carrying out a study. This study is called the British Childhood Cancer Survivor study.
This research is looking at children who were:
- diagnosed with cancer between 1940 and 1991 and
- lived for at least 5 years after diagnosis
This type of research takes many years to produce results. This is because the children need to be followed throughout their lives. So it will be some time before we know the results.
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