Some leukaemia patients may be missing out on new treatments, our research finds

11 Jan 2018

In new research, majority-funded by Barts Charity, findings show patients with an aggressive form of leukaemia, currently ineligible for any type of targeted therapy, may benefit from new drugs.

One such drug, midostaurin, was recently approved by the US Food and Drug Administration to treat this type of aggressive leukaemia, but only for certain patients – those who show mutations on a gene named FLT3.

In unexpected findings, the research team at Queen Mary University of London found that these FLT3 mutations were not a good predictor of whether the drug would be beneficial to a patient.

Instead, they found that protein markers were much more accurate in identifying which patients would respond to the drug.

The study

The researchers analysed cell samples from 36 patients with acute myeloid leukaemia (AML), an aggressive cancer of the myeloid cells – cells that help the body fight bacterial infections.

The lead researcher, Dr Petro Cutillas, commented on the importance of the findings:

“Currently about 30% of AML patients are eligible for midostaurin, and about 50% of those benefit. With our markers we expect the number of patients treated would double.

“Although our study focused on a specific type of leukaemia, the realisation that protein markers are better predictors of responses then genetic alterations is likely to have implications for the development of personalised therapies for other cancer types.”

The findings were published in the Nature journal Leukemia. These new insights help to show why some patients respond to new cancer drugs while others are resistant.

Understanding these responses at a molecular level can help both scientists and clinicians working alongside each other to more efficiently predict which drug may be appropriate to treat each individual patient.

This is the ultimate goal of personalised, precision medicine.

Our thoughts

Dr Francesca Gliubich, our Director of Grants, said: “This breakthrough from Dr Cutillas highlights the capacity of precision medicine to maximise patient outcomes and reduce the time to identify the most appropriate treatment.

“We’re delighted to support projects such as these that have the potential to make a significant step change in healthcare.”

Changing thinking about treatments

Genetic alterations are a hallmark of cancer, where errors in the DNA code often allow malignant cells to become addicted to particular cellular pathways in order to rapidly divide, grow and spread.

But findings from this research show that genetic alterations or mutations alone do not represent ideal predictors of drug response – as previously thought.

Markers of activity for kinases – a group of proteins involved in cancer progression – predicted responses to midostaurin and other new targeted cancer drugs with higher accuracy – identifying an increase in the number of patients to potentially benefit from these drugs.

What it means for the future of cancer research

Dr Pedro Cutillas added: “This study opens new opportunities in the field of personalised medicine. We expect that this work will eventually lead to treatments tailored to the patient’s tumour, so that less time and money is wasted with treatments that are not expected to work, saving funding for the NHS and benefiting patients with better treatments.”

The rest of the funding for this research was from Cancer Research UK and the Biotechnology and the Biological Sciences Research Council.

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