An Addenbrooke’s consultant is spearheading research into an aggressive form of blood cancer which could advance treatments for patients with the worst prognosis.
Acute myeloid leukaemia (AML) affects people of all ages, often requiring months of intensive chemotherapy and long hospital admissions.
It normally develops in cells within the bone marrow, crowding out the healthy cells, and leading to life-threatening infections and bleeding.
Mainstream AML treatments have remained unchanged for decades and fewer than one in three people survive the cancer. The prognosis is particularly poor for patients with AML that is characterised by specific genetic mutations within the leukaemic cells, such as loss of chromosome 7 or a gene called CUX1. To date, no personalised treatments are available to target these high-risk cases.
Honorary consultant haematologist, Dr Chi Wong, is the senior author of a paper which has today (30 April) been published in Nature Communications and offers hope to those with AML.
Dr Wong, a Wellcome Clinical Fellow at Cambridge’s Wellcome Sanger Institute, has led a team to identify a vulnerability in a poor-prognosis subtype of AML which could be a target for novel treatments.
The Wellcome Sanger team used CRISPR/Cas9 gene-editing technology to show that a lack of a properly functioning CUX1 gene leads to the expansion of certain types of defective blood stem cells, culminating in the development of leukaemia.
Loss of CUX1 causes increased expression of another gene, called CFLAR, which normally stops cells from undergoing cell death, providing a way for mutated leukaemic cells to survive.
The researchers showed that targeting CFLAR could be a possible treatment, paving the way for the application of new drugs in these poor-prognosis blood cancers. Currently, there no drugs approved that target CFLAR.
Dr Wong said:
Acute myeloid leukaemia is a devastating disease, which is currently difficult to treat. This new study provides evidence that could be used to help develop new targeted treatment, offering hope for this group of patients who unfortunately are more likely to have a poor prognosis.