Precision medicine reveals ‘Achilles heel’ in chronic myeloid leukaemia

pharmafile | September 16, 2016 | News story | Medical Communications University of Glasgow, chronic myeloid leukaemia 

Scientists at the University of Glasgow believe they have revealed an ‘Achilles heel’ of chronic myeloid leukaemia (CML), making their second significant breakthrough in the treatment of the disease by using precision medicine.

CML is a rare form of blood cancer, caused when normal blood cells become leukaemic or CSL stem cells. Sufferers are currently treated with tyrosine kinase inhibitors (TKIs) which rarely cure the disease but hold it in check, meaning they require lifetime use. 150,000 people in the EU alone rely on TKI’s at a cost of £5 billion per year.

TKI’s do not kill CML cells, but research teams led by Dr David Vetrie and Professor Tessa Holyoake at the Institute of Cancer Sciences, University of Glasgow believe they have discovered a new drug which could lead to a treatment of the disease.

The study, published in the journal Cancer Discovery, identified two proteins which are essential to the survival of CML stem cells, which led to the development of a drug combination which has been shown to target and kill over 90% of these cells, while leaving normal cells unharmed.

Dr David Vetrie, based at the Wolfson Wohl Cancer Research Centre in Glasgow, said: “This discovery, based on seven years of experimental work, is a great example of precision medicine – finding drugs that target only the CML stem cells we want to kill, while leaving normal cells unharmed.”

“Having a new drug that we can give to patients alongside a TKI is an ideal scenario in the clinic – TKIs kill most of the CML cells, and we believe that the EZH2 inhibitor will kill the remaining CML stem cells.”

The findings could potentially pave the way for advances in the treatment of other cancers including acute myeloid leukaemia and brain tumours.

Dr Mary Scott, first author on the paper, added: “Our aim is to take this drug into clinical trials with CML patients. Current clinical trials using an EZH2 inhibitor in other forms of blood cancer have shown some promising early results. The drug is taken as a pill, is very safe and has minimal side-effects, making it ideally suited for the CML clinic.”

Matt Fellows

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