Dr J Anderson and Dr M Pule, Institute for Child Health

08 December 2008

Assessment of anti-PAX5 immunotherapy for paediatric haematological and solid cancers

Amount of grant: £200,289

Date of award: December 2008

Dr John Anderson and Dr Martin Pule, Institute for Child Health, London

This ground-breaking research aims to help children who are failed by existing treatments.

Background

Most children with leukaemia are initially treated with high doses of chemotherapy – to kill the deadly leukaemia cells and enable blood cells to restore their vital functions.

Some children respond well to chemotherapy and no further treatment is required. Some children, however, respond less well, or may relapse. The only hope for these children is a bone-marrow transplant (BMT).

The effectiveness of BMT lies in the immune response of the donated cells – the hope is that they will attack the leukaemia. But BMT is a high-risk procedure, used only as a last resort.

The research project

The research team wants to engineer the patient’s own immune cells to attack the leukaemia cells and avoid the risks of a bone-marrow transplant.

The research team wants to engineer the patient’s own immune cells to attack the leukaemia cells, and avoid the risks of BMT.

They will do this by reprogramming the patient’s immune cells to recognise a molecule called PAX5 - this is present on the surface of leukaemic cells, but not present on other cells.

Their work is still at an early stage. They are carrying out essential laboratory testing to provide data on the effectiveness of the treatment and its safety before it can be taken onto the wards for clinical trials.

If the team succeeds in their aim, high-risk leukaemia patients will be able to avoid the dangerous complications associated with bone marrow transplantation. They should also have long-term protection against the leukaemia cells returning.

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