A trial looking at gene therapy for acute myeloid leukaemia and myelodysplastic syndrome

Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Myelodysplastic syndrome (MDS)




Phase 1/2

This trial looked at a treatment called T cell receptor gene therapy Open a glossary item.  It was for people with acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS). 

The trial was open for people to join between 2015 and 2018. The team analysed the results in 2019.

More about this trial

Doctors often treat acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) with a chemotherapy drug called azacitidine. But it doesn’t always work very well.

The research team wanted to find out if T cell receptor (TCR) gene therapy is useful for people in this situation.  

Doctors remove specific immune system cells (called T cells) from a person’s blood sample. Then they change (modify) the cells in the laboratory. They add a gene to the cells to help them recognise a protein called WT1. The doctors then give the person back their T cells through a drip into a vein.

Abnormal blood cells such as leukaemia cells often have a high level of WT1. Other cells in the body usually have a very low level of WT1. Researchers hoped the modified T cells would recognise the WT1 protein and attack the abnormal blood cells.

This trial was for people who’d already had azacitidine, but their AML or MDS had got worse. Everyone taking part had the same treatment.

The main aims of the trial were to find out:

  • how well T cell receptor gene therapy works
  • more about the side effects

Summary of results

As part of our editorial policy, any trial information we write is checked externally before we put it on our website. The research team have published some results for this trial. But we have been unable to find anyone involved with the trial to check the summary for us. 

This means we are not able to include a plain English summary of the results on this page.

More information
There is more information about this trial in the link to the European trials website below.  

Please note, the information we link to here is not in plain English. It has been written for healthcare professionals and researchers.

A single arm Phase I/II study of the safety and efficacy of gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML) who have failed to achieve or maintain an IWG defined response following hypomethylating agent therapy
The European Union Clinical Trials Register
Accessed September 2023

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 Emma Morris

Supported by

Cell Therapy Catapult
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer

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

Freephone 0808 800 4040

Last review date

CRUK internal database number:


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

Last reviewed:

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