Henry Winter
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Drug dramatically improves survival for relapsed childhood leukaemia

Henry Winter
Posted by
06 Dec 2010

A new treatment increases survival by over 50 per cent for children whose acute lymphoblastic leukaemia (ALL) returns, according to the results of a trial published online in the Lancet on 4 December.

The results of the trial – funded by Cancer Research UK and Leukaemia & Lymphoma Research - were so promising that all children were offered the trial drug Mitoxantrone.

Of the 216 children who 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 Vaskar Saha, of the Paterson Institute in Manchester, said: “These striking results show just what a powerful tool Mitoxantrone could be to treat children whose cancer has returned, offering hope to many families across the country.”

Over the last 30 years the number of children who have survived ALL has risen from 50 to 90 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 has remained constant at around 50 per cent.

Mitoxantrone was chosen for this trial because it can attack ALL cells that have survived the first round of treatment. As well as attacking rapidly dividing cells, it also targets ‘hidden’ ALL cells. These cells are able to escape the initial treatment by hiding within a protective barrier of normal cells. Scientists think that these cells re-seed the cancer, triggering the cancer’s return.

But Mitoxantrone is able to attack this cache of cancer cells and tag them so that the body’s own immune system can destroy them.

Professor Saha said: “As a result of this trial, Mitoxantrone is now becoming the standard treatment for relapsed ALL, hopefully having a significant impact on the number of children who beat the disease worldwide. Mitoxantrone is a relatively cheap and readily available drug and we now want to further explore the impact it could have for children with ALL.”

Dr David Grant, Scientific Director at Leukaemia & Lymphoma Research, said: “This trial has added to the evidence that truly curative drugs have to kill resistant or residual leukaemia stem cells. These cells are the likely source of relapse because they escape first line treatment. The remarkable improvement in survival reported in this trial is very encouraging for the testing of other leukaemia stem cell specific drugs.”

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.”