Scientist leads successful drug trial for childhood leukaemia

(PhysOrg.com) -- Research has shown that a new cancer treatment increases survival to almost 70 per cent for children whose acute lymphoblastic leukaemia (ALL) returns.

The study, carried out by Professor Vaskar Saha, who is Cancer Research UK Professor of Paediatric Oncology at The University of Manchester, is published online in The Lancet on December 4. The results were so promising that now all children with relapsed ALL are being offered the trial drug Mitoxantrone.

The trial was funded by Cancer Research UK (CRUK) and Leukaemia & Lymphoma Research. Central Manchester University Hospitals NHS Foundation Trust has sponsored the study since early 2010, providing oversight of all the clinical trial processes including the conduct, management and safety of the study.

Patients from Royal Manchester Children’s Hospital have also participated in the trial, and it is supported by the National Institute for Health Research’s Manchester Biomedical Research Centre (MBRC).

Two-hundred-and-sixteen children across the UK, Australia and New Zealand took part in the trial; 111 were given the standard treatment Idarubicin and 105 were given Mitoxantrone. After three years, 69 per cent of children given Mitoxantrone had survived the disease, compared to 45 per cent of those given Idarubicin.

Professor Saha, who is based in the School of Cancer and Enabling Sciences, said: “These striking results show just what a powerful drug Mitoxantrone is in treating children whose leukaemia has returned, offering hope to many families across the country.”

The significant increase in survival seen in this trial has resulted in Mitoxantrone being offered to all children with relapsed ALL since 2008. As well as improving survival, children given Mitoxantrone also experienced fewer side effects.

Over the last 30 years the number of children who have survived ALL has risen from 50 to over 80 per cent but similar improvements have not been seen in children whose cancer returns. It remains the leading cause of cancer death in children, and survival for children whose leukaemia returns had until now remained constant at around 50 per cent.

Professor Saha added: “As a result of this trial, Mitoxantrone is now the standard treatment for relapsed ALL, and is having a significant impact on the number of children who beat the disease worldwide. This is the first time that a trial in ALL has been stopped so early after one drug had such clear benefits for patients.”

Kate Law, director of clinical research at Cancer Research UK, said: “These exciting results highlight the impact that research is continuing to have to help more children beat the disease. Cancer Research UK is the largest funder of research and trials into childhood cancers in the UK. Today, thanks to research like this, more than three quarters of children beat cancer, compared to a quarter in the 1960s.”

Also writing in The Lancet, Professor Martin Schrappe of the University Medical Centre Schleswig-Holstein called the outcome "one of the largest improvements ever achieved by a single modification of treatment in childhood ALL".