SCOTTISH scientists are at the forefront of an “Achilles heel” breakthrough that could lead to a cure for chronic myeloid leukaemia (CML).

The blood cancer causes the body to make too many white blood cells, which eventually fill the bone marrow and reduce the number of healthy white cells.

Now researchers at Glasgow and Manchester universities have found an “Achilles heel” of the disease and have successfully targeted the weakness with drugs to eradicate CML in mice.

Their study, published in the magazine Nature, analysed CML and normal blood stem cells and found two proteins that were key to the survival of CML stem cells. The group, which has spent more than six years working on this research, then developed a drug combination to target these proteins and kill the cancer stem cells, while largely sparing normal cells.

The team, led by Professor Tessa Holyoake from the University of Glasgow and Professor Tony Whetton from the University of Manchester, used a range of techniques to show that these two proteins (p53 and c-Myc) acted as “gateway controllers” in CML.

Using the concept of precision medicine — the right drug, at the right time, for the right effect in the patient — they designed a new treatment to exploit the caner’s weakness. With CML cells transplanted into mice, they demonstrated that drugs targeting these two proteins killed the cells that cause the leukaemia, effectively eradicating the disease.

The results have huge potential implications for other cancers, including acute myeloid leukaemia and brain tumours. The team now want to start human trials in patients with drug-resistant CML.

Holyoake, who led the team from the Paul O’Gorman Leukaemia Research Centre, said: “We are certainly excited by the results shown in the study.

“The research – a fantastic example of precision medicine in action – is at an early stage, but the data we collected has revealed two weaknesses in CML and a potential drug approach to eradicating these key stem cells. We also could not have achieved such an excellent result without all the generous stem cell donations from both CML patients and other members of the public, so it is important to say thank you to them.”

The team used a range of techniques in their research including proteomics - the large scale study of quantities, structures and functions of proteins.

Whetton added: “We are really excited that our new proteomics approaches helped to achieve this.

“There are so many other diseases where we can use the same proteomics approach to find precision medicine solutions for patients.

“We have the largest clinical proteomics centre in Europe in Manchester so we really look forward to contributing to this work.”

The breakthrough was welcomed by Dr Áine McCarthy, from Cancer Research UK, who said: “By recognising the important roles p53 and Myc play in helping chronic myeloid leukaemia stem cells to survive, this study has identified two new ways to target and kill these cells.

“Excitingly, this early-stage laboratory work also showed that two experimental drugs which target the effects of these molecules can kill CML stem cells in mice.

“The next step will be to test if this combination works the same way in people, and if it is safe to use.”

CML sufferers survive longer — 85 per cent live for more than five years after diagnosis – and there is a growing economic cost associated with current therapy costing between €40,000 (£31,200) and €70,000 (£54,700) for one patient per year living in Europe.