Research into neuroblastoma

Research and clinical trials

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.

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.

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.

Go to information about this clinical trial

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 ​tyrosine kinase inhibitor​. It is for neuroblastoma that has changes in the anaplastic lymphoma kinase (ALK) ​gene​. 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.

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.

MiNivAN trial

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 ​growth factor​. 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 ​external radiotherapy​. 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.