Chronic myeloid leukaemia (CML) cells produce a protein called BCR-ABL that isn't found in healthy cells. BCR-ABL is a tyrosine kinase, which causes CML cells to grow and reproduce out of control. People with CML are often treated with tyrosine kinase inhibitors (TKIs), such as imatinib or nilotinib. TKIs block the action of the BCR-ABL tyrosine kinase, so signals that tell the CML cells to grow and divide are disrupted.
TKIs have improved the outcome for people with CML dramatically. However this improvement remains limited to people who receive TKI treatment whilst in the chronic phase of their disease. Unfortunately, the majority of people whose disease has progressed to blast phase have already received all the available drugs in succession during the chronic phase, so there is no TKI available when they progress. If this happens, people are usually given intensive treatment comprising high dose of chemotherapy alongside a TKI, and then a stem cell transplant.
This trial is looking at a conventional chemotherapy regimen called FLAG-IDA alongside a new TKI drug called ponatinib for people who are in the blast phase of their CML. Ponatinib works in a similar way to imatinib and nilotinib, in that it specifically targets BCR-ABL that is only found in CML cells. However, ponatinib was also designed to target mutated forms of the BCR-ABL that arise in people who become resistant to their TKI treatment, as well as other kinases implicated in blood cancer and solid tumours.
If found to be safe, researchers hope that this combination of drugs can be used in future trials to see whether we can improve the outlook for people with CML who are in blast phase.
The aims of the trial are to:
- Find the best dose of ponatinib for people having intensive treatment for CML that is in blast phase
- See how well ponatinib works for this group of people
- Learn more about the side effects
You may be eligible to join this trial if:
- You have chronic myeloid leukaemia (CML) that is in the blast phase
- You are at least 16 years old
Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>
If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>