Scientists have found a potential new treatment strategy that could significantly halt acute myeloid leukaemia (AML), a particularly aggressive type of blood cancer, by targeting the pathways that our cells use to respond to oxygen levels.
The research, out now in Nature Cancer, was co-funded by Barts Charity, the Medical Research Council and Cancer Research UK. The research was co-led by scientists from The Institute of Cancer Research, London, and the University of Oxford, and partly undertaken at Queen Mary University of London.
AML: an aggressive type of blood cancer
AML is an aggressive type of blood cancer that usually affects older adults, but also occurs in children and young adults and for which there are few treatment options. Patients with AML experience a dramatically increased production of immature white blood cells, called blasts. These blasts damage the bone marrow and other organs with devastating consequences to patients.
Therapies have remained relatively unchanged for the last 30 years, with most patients receiving chemotherapy and bone marrow transplants to control their condition.
Unfortunately, many treatments are not effective and can have toxic, sometimes deadly, side effects.
Preventing AML from progressing
The team aimed to understand whether enzymes called hypoxia-inducible factor prolyl hydroxylases (PHDs), which sense levels of tissue oxygen in the body, could be a target for treating AML. This builds on previous research where the team showed that active PHDs target proteins for destruction, which leads to the aggressive acceleration of AML.
The researchers now show, for the first time, that targeting the pathways that our cells use to respond to oxygen levels could provide a new avenue for treatment, without impacting the normal production of blood cells in the bone marrow.
These enzymes can already be blocked safely with existing drugs used to treat anaemia.
Excitingly, the team have developed a new first-in-class drug which more selectively blocks the enzymes than existing drugs, and so may reduce side effects.
Our £10m investment in cancer research
We are proud to have supported part of this collaborative research, which was funded as part of Barts Charity’s £10m investment into cancer research at Barts Cancer Institute in 2019. We look with anticipation to the next steps of potential clinical trials and bringing this exciting development to patients with AML.