Cancer Research UK’s Drug Development Office has opened the first trial of a new type of drug to treat children aged from six months to 18 years with acute leukaemia, who are no longer responding to treatment.

In this first-in-child study, 15 children with acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML) will receive a treatment called AT9283. AT9283 belongs to a new class of drugs called aurora kinase inhibitors.

The trial is led by Great North Children’s Hospital, Newcastle upon Tyne and is also running at four other clinical centres.

Despite great progress in the treatment of childhood leukaemia, approximately 100 children with leukaemia under the age of 15 die each year in the UK. Leukaemia that has returned or is resistant to current treatments remains one of the key causes of death from childhood cancer. Cancer is the second most common cause of death in childhood.

AT9283 looks promising in laboratory studies and has been tested in a small number of adults (solid tumours and haematological malignancies) and children and adolescents with solid tumours. This trial will assist in establishing the correct drug dose and investigate if it can treat children and adolescents with leukaemia.

Chief investigator Professor Josef Vormoor, said: “It’s devastating to have to tell parents of a child with leukaemia that the disease has returned. Or that it’s unlikely their child can be treated with existing drugs.

“So I’m incredibly excited about the launch of this trial, to see if a new drug can treat the disease, when a child has stopped responding to current treatments."

AT9283 blocks the activity of a group of proteins called aurora kinases which control cell growth. Blocking these proteins can stop cancer cells’ ability to grow.
Around 1,500 children are diagnosed with cancer each year in the UK, with leukaemia, the most common childhood cancer accounting for around a third (31 per cent) of all cases.

The trial is funded, managed and sponsored by the charity’s Drug Development Office (DDO) and the drug is provided by Astex Pharmaceuticals.

Dr Nigel Blackburn, director of drug development at Cancer Research UK’s Drug Development Office, said: “We’ve made amazing progress in the treatment of childhood cancers: In the 1960s, only around a quarter of children with cancer survived. Today, three quarters survive. But there is much more to do.

“There’s an urgent need to develop new treatments for young people with leukaemia. The launch of this first trial of a completely new treatment for childhood leukaemia is incredible news and we’ll be watching the results with great interest.”

Cancer Research UK spent nearly £9 million last year on research into children’s cancers.

Source: CRUK