A trial of T Cells for neuroblastoma that is not responding to treatment or has come back (1RG-CART)

Cancer type:

Children's cancers
Neuroblastoma

Status:

Results

Phase:

Phase 1

This trial looked at using white blood cells called anti-GD2 T cells to treat neuroblastoma. It was for people aged 12 months or older, whose cancer had not responded to treatment or had come back after treatment.

The trial was supported by Cancer Research UK. It was open for people to join between 2016 and 2019. The team reported the results in 2021.

More about this trial

Neuroblastoma is a rare cancer. It mostly affects children, and often those under 5 years old. It usually starts in the tummy (abdomen) area.

When this trial was done, doctors often treated neuroblastoma with surgery, chemotherapy or radiotherapy.

In this trial they were looking at a type of immunotherapy called anti-GD2 T cells. 

The research team took some immune cells (called T cells) from blood samples. They added a gene to the T cells in the laboratory. This gene tells the T cells to recognise and attack cancer cells. This is sometimes called CAR T cell therapy.

Results from laboratory studies of anti-GD2 T cells looked promising. But this was the first time it was used as a treatment for people with neuroblastoma.

The main aims of the trial were to find out whether:

  • they can make anti-GD2 T cells in the laboratory
  • it is a safe treatment
  • it causes any side effects 
  • it can shrink neuroblastoma

 

Summary of results

The team found it hard to say for sure how well treatment worked because only a small number of people joined the trial.

Trial design
This trial was for people with neuroblastoma that hadn’t responded to treatment, or had come back after treatment.

Everyone taking part had a single treatment of anti-GD2 T cells. Some had a higher dose than others. And some also had cyclophosphamide and fludarabine chemotherapy.
 
Results

A total of 12 people had anti-GD2 T cells as part of this trial. They were between 2 and 15 years old. 

There were 5 treatment groups:

  • 4 people had T cells and no chemotherapy (group 1)
  • 1 person had T cells with cyclophosphamide (group 2)
  • 1 person had T cells with cyclophosphamide and fludarabine (group 3)
  • 3 people had a higher dose of T cells with cyclophosphamide and fludarabine (group 4)
  • 3 people had an even higher dose of T cells, with cyclophosphamide and fludarabine (group 5)

CAR T cells in the blood
The research team took blood and tissue samples to see if they contained any CAR T cells. 

One test showed that 6 people had some CAR T cells in their blood sample. 

A different test showed that 9 people had some CAR T cells in their blood sample. 

No one had a high enough level in their blood for 2 weeks after treatment for it to be considered a successful treatment.

No one had any CAR T cells in the bone marrow or cancer samples taken after treatment.

The team had planned to do another part of this trial. But they felt that the immune response in people taking part wasn’t strong enough. So they decided not to continue with the second part. 

Side effects
Everyone who had treatment had at least one side effect. But many were mild or didn’t last long.

The first 6 people in groups 1, 2 and 3 didn’t have any severe side effects.

One person in group 4 had two side effects that were more severe. 

One was something called cytokine release syndrome (CRS). This is when immune cells release a large number of proteins (cytokines) into the blood. It can cause symptoms such as fever, low blood pressure and breathing problems.

The other was a serious infection which caused sepsis. The team think that the this is more likely to have been caused by the chemotherapy than the T cells.

No one in group 5 had any severe side effects.

How well treatment worked
The team were able to look at how well T cells were working for 10 people, a month after treatment. They found that the cancer had:

  • stayed the same in 5 people
  • continued to grow in 5 people

Conclusion
It is hard to draw any firm conclusions about how well anti-GD2 CAR T cells worked because of the small number of people in the groups. 

The trial team felt the results showed that this treatment did affect the immune system in the short term. And that it didn’t cause too many severe side effects. They suggest more work is done.

Where this information comes from    
We have based this summary on information from the research team. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item) or published in a medical journal yet. The figures we quote above were provided by the research team. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Professor John Anderson

Supported by

Cancer Research UK (Centre for Drug Development)
NIHR Clinical Research Network: Cancer

Other information

This is Cancer Research UK trial number CRUKD/15/001.

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

9177

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Around 1 in 5 people take part in clinical trials

3 phases of trials

Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.

Last reviewed:

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