
Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.
This trial looked at a drug called ponatinib for chronic myeloid leukaemia (CML) or some types of acute lymphoblastic leukaemia (ALL). It is for people whose leukaemia had not responded to, or had got worse while having, other treatments.
Most people with CML, and some people with ALL, have a change () to a gene in their leukaemia cells. Doctors call this
. It affects a protein called BCR-ABL1 which tells the body to make too many white blood cells.
Certain drugs called (TKIs) can block the BCR-ABL1 protein. But sometimes these drugs don’t work very well, or they stop working (the leukaemia becomes
to them).
In this trial, researchers looked at a new TKI drug called ponatinib. The people taking part have already had other TKIs such as imatinib, nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib but their leukaemia was not responding. Some people taking part had a genetic change in their leukaemia cells, called T315I, which makes them resistant to all other TKIs. The aim of this trial was to see if ponatinib helped people with CML or Philadelphia positive ALL who are in one of the following situations
The trial team found that ponatinib did help people with chronic myeloid leukaemia (CML) and people with Philadelphia positive acute lymphoblastic leukaemia (ALL).
When the researchers analysed the results they looked at the blood counts to see if they had gone back to more normal levels. This is called haematological response.
They also looked at the to find out how many cells containing the Philadelphia chromosomes were present. This is called cytogenetic response.
They also looked at how much BCR-ABL1 was in their blood. This is called a molecular response.
Of the 449 people who took part in this trial, the researchers were able to look at the results of 444. The researchers analysed the results according to whether the people taking part had Philadelphia positive ALL or by the stage of their CML.
Of the 267 people who had chronic phase CML
Of the 83 people who had accelerated phase CML
Of the 62 people who had blast phase CML
Of the 32 people who had Philadelphia positive ALL
The most common side effects were
The trial team concluded that ponatinib could help people with CML or Philadelphia positive ALL.
We have based this summary on information from the team who ran the trial. The information they sent us has been reviewed by independent specialists () and published in a medical journal. The figures we quote above were provided by the trial team. We have not analysed the data ourselves.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Professor Jane Apperley
ARIAD Pharmaceuticals Inc
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
Pharmanet
Freephone 0808 800 4040
Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.