01 Aug 2022 Research

A research breakthrough targets cancer over healthy tissue, bringing possibility of a new treatment option for most deadly type of leukaemia

A new scientific paper published on 22nd June in Science Translational Medicine, announced a breakthrough by Leukaemia UK-funded scientist Dr William Grey that could lead to a vital new treatment option for patients with acute myeloid leukaemia (AML).

Every day in the UK 28 people – 10,000 a year – are told the devastating news that they have leukaemia, a blood cancer affecting white blood cells.

Despite decades of progress, only half of leukaemia patients live longer than five years after their diagnosis and the 5-year survival for one type of leukaemia, called acute myeloid leukaemia (AML), is just 15.3% [1].

Over 3,000 people are diagnosed with AML each year in the UK, including 100 children [2].

Despite greater understanding of the disease, the mainstream therapies for acute leukaemias like AML have remained unchanged for decades, often involving harsh treatments like chemotherapy and stem cell transplant.

In people aged 65 or older, only 5 out of 100 people (around 5%) will survive AML for 5 years or more after diagnosis [3]. Older patients with AML are often not eligible for the best performing therapies but crucially if we could bring them back into the eligibility criteria for this, there would be improved prognosis.

That is why this latest discovery by Dr William Grey and his colleagues, of a potential new targeted treatment for AML, is so important.

Dr Grey, a Leukaemia UK funded scientist working at that time as a post-doc at The Francis Crick Institute in Professor Dominique Bonnet’s team and now developing his own group at the University of York, discovered that by inhibiting the action of a particular protein, CKS1, leukaemic stem cells can be successfully targeted without causing harm to normal, healthy stem cells – making it an excellent treatment target.

The aim of Dr Grey’s work was to find ways to treat AML patients with the worst overall prognosis. This is a patient group who would usually have less than a 1 in 5 chance of surviving up to five years from initial diagnosis. Interestingly, Dr Grey’s findings suggest that by reducing the side effects of traditional chemotherapy, it may provide the opportunity to bring back elderly and clinically unfit patients into a selection criteria for intensive therapy.

Dr Grey and the team are now exploring the possibility of initiating a clinical trial with colleagues at the Royal Marsden Hospital in London. As they continue to investigate how this approach could be used to eliminate leukaemia, they are also looking at how the knowledge they have gained can be used to further improve this method of targeting leukaemic stem cells and develop new therapeutic approaches.

Dr William Grey said:

“We hope that this work will open new avenues of investigation into the protein dynamics of stem cells, and give us a better understanding of how stem cells work in our body and how they go wrong during disease. In doing so we hope that we can reveal new and more effective treatment targets that haven’t yet been discovered during the genetic revolution that has been ongoing for the past two decades.”

Fiona Hazell, Chief Executive of Leukaemia UK, said:

“There is a vital need to develop kinder and more effective treatments for acute leukaemias. By focussing on trying to improve outcomes for AML patients with the worst prognosis, research can help accelerate progress where it is most needed in order to save and improve more lives. Dr Grey’s promising discovery during his Leukaemia UK John Goldman Fellowship demonstrates the importance of continued research and could go on to provide a vital new treatment option for the most vulnerable leukaemia patients.”

Iona, who has now undergone a successful treatment programme of chemotherapy, further medication and crucially, a stem cell transplant, has been told that she has now undergone so much chemotherapy that she has reached the limit for one particular drug. This means that if Iona were to relapse, she would need a different form of treatment – hopefully one that can be as effective as the chemotherapy she has already received but without the strain that it puts on the human body.

Iona said:

“It would be amazing if less invasive forms of treatment for leukaemia can be found, which work just as effectively as chemotherapy but don’t take such a heavy toll on your body. I know that for me this would be particularly important if I ever did relapse, as I have now gone through so much chemotherapy that I have reached the lifetime limit for one of the drugs I was having.”

On Dr Grey’s research, Iona added: “I hope that Dr Grey’s research into new treatments can help improve the prognosis and experience for the next people to be diagnosed with leukaemia. Ultimately, we need to find more and more ways for people to survive this diagnosis.”

Find out more about Dr Grey’s research.

[1] Cancer Research UK, statistics for people diagnosed with AML in one area of England between 2004 and 2016, Survival | Acute myeloid leukaemia | Cancer Research UK

[2] Blood Cancer UK, Childhood acute myeloid leukaemia (AML) – what is it, symptoms and treatment | Blood Cancer UK

[3] Cancer Research UK, average survival rate for all age groups over 65 years old, statistics for people diagnosed with AML in one area of England between 2004 and 2016, Survival | Acute myeloid leukaemia | Cancer Research UK

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