Dr Owen Williams, Institute of Child Health, London

The high overall cure rate for childhood leukaemia masks an extremely poor outlook for children with certain forms of the disease. Children who are diagnosed with leukaemia at less than one year of age have a particularly poor prognosis. Half of them will not reach their fifth birthday.

Working in collaboration with their colleagues at Great Ormond Street Hospital, Dr Williams and his team are making important discoveries about the genetic changes which lead to infant leukaemia.

The vast majority of infant leukaemias are caused by a change involving a break on the Mll gene on chromosome 11.

The broken chromosome fuses with other broken chromosomes (usually chromosome 4), forming a new gene at the fusion point. This leads to the production of a protein which triggers certain genes in white blood cells to be switched on in an inappropriate way, causing them to mutate into leukaemia cells.

The team is breaking down this complex series of events to understand precisely what happens at each stage. Having discovered which genes are switched on in the white blood cells as a result of the chromosome fusion, the team now has a two-pronged approach:

• To determine the effects of these genes on the development, proliferation and resilience of leukaemic cells by ‘turning on’ the genes in bone marrow stem cells.

• To define the role of the same genes in the normal function of bone marrow stem cells – i.e. what happens to cells if this gene is deleted?

The ultimate aim of this programme of work is to improve the poor outlook for infants with leukaemia by developing new treatments which can effectively block the series of events which are causing their white blood cells to malfunction.

Return to Current Treatment Projects