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Clinical trial shows some patients could have treatment levels reduced

The Bloodwise logo. Bloodwise appears in black text against a white background
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26 Jan 2017

Patients with chronic myeloid leukaemia (CML) taking part in a new clinical trial looking at whether some patients can receive reduced treatment have talked about their experiences in a new BBC report.

Photo: DESTINY trial participant Jean with her family

Patients with chronic myeloid leukaemia (CML) taking part in a new clinical trial looking at whether some patients can received reduced treatment have talked about their experiences in a new BBC report. The patients interviewed were also among the first to take part in clinical trials for the groundbreaking drug imatinib in 2000. 

The introduction of targeted drugs known as tyrosine kinase inhibitors (TKIs), of which imatinib was the first of its kind, transformed the outlook of CML patients. Until then there were a limited range of treatment options available to patients. A bone marrow transplant represented the best chance of a long term cure, but the majority of patients were too elderly to undergo the gruelling treatment needed.

TKI drugs are taken daily in tablet form, enabling most patients to live a normal life. TKIs are thought to control the disease, rather than cure it however, so patients currently have to stay on treatment long term. Some patients experience substantial side effects while on treatment.

Recent results from a new clinical trial, funded by Bloodwise, have shown that some patients can stop taking TKIs altogether. The DESTINY trial investigated whether some patients with excellent responses to these targeted drugs can remain well on either a lower dose of treatment or without treatment at all. The trial is led by the University of Liverpool and involves hospitals across the country, including Newcastle upon Tyne Hospitals - where one of the original imatinib clinical trials took place in 2000. It is supported by the National Institute for Health Research (NIHR).

Results from the study suggest many CML patients with excellent responses may be able to safely reduce TKI side effects by cutting their dose in half. Of 174 study participants, the vast majority (93%) showed no evidence of leukaemia relapse one year after cutting their TKI dose. Many patients experienced a significant decrease in TKI-associated side effects within the first three months. Just 12 participants showed signs of leukaemia relapse, all of whom regained their initial remission level or better within four months of resuming a full TKI dose.

Professor Richard Clark from the University of Liverpool’s Institute of Translational Medicine, who led the DESTINY study, said: “Taken together, these findings indicate that some patients are being unnecessarily over-treated. The other important implication is that patients do not have to have extremely low levels of leukaemia on very sensitive tests in order to safely try reducing their TKI dose.”

68 year old Jean from Newcastle was diagnosed with CML in 1996 and took part in one of the original imatinib clinical trials in 2000, run by Consultant Haematologists Prof Stephen O’Brien and Dr Anne Lennard at Newcastle upon Tyne Hospitals.

Jean recalled: “I feel very blessed that I was in the right place to be on the trial the first time around, undoubtedly I would have died without it. I’m now 20 years in from diagnosis and I’m so lucky to be here. I’ve seen my sons grow up, I’ve got grandchildren, all the things I didn’t think I was going to have because there was a strong possibility I would be dead by 50.”

“To be told you’ve got CML today is very different to when I was diagnosed because it's now a chronic condition rather than a terminal condition. Over the last few years I’ve been able to tell travel insurance companies I’m in remission and of course you never could tell anybody that when I was diagnosed as there was no such thing.”

In 2015 the medical team at the Newcastle upon Tyne Hospital Foundation Trust approached Jean for a second time and asked if she would be willing to take part in the DESTINY trial looking into whether doses of TKIs could be reduced or stopped in some patients.  

Jean said: “By the second trial I was very comfortable, medically very balanced, everything was working well so in a sense it was more of a risk inasmuch as everything seemed to be working fine, whereas before the first trial I knew it [the treatment] wasn't going to work for very long. Prof Steve O’Brien and Dr Wendy Osborne gave me so many reassurances, that if at any point I wanted to or they thought I was at risk I could stop, so I always felt like I was in control of what happened. I just thought I’ve got to try because nobody knows how long being on the drug will work or what the future holds. I am please I did it because I definitely think being on half dosage has suited me better.

Watch the coverage of the story on BBC Look North here.