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We will combine gene therapy with immunotherapy for the specific treatment of paediatric cancers that express the proteins PAX3 and MYCN. These proteins are specifically over-expressed in a number of aggressive conditions including neuroblastoma and rhabdomyosarcoma. The continued expression of the proteins is essential for tumour survival but they are not expressed on normal tissues. The project will optimise methodology allowing receptors that recognise MYCN or PAX33 to be inserted into the killer cells (T cells) of the patient’s own blood. This transfer of the receptors is achieved by gene therapy of blood cells in the laboratory but the genetically altered T cells can then be transferred back into the patient and should detect and specifically kill tumour.
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INSTITUTE OF CHILD HEALTH £169,832 over 3 years START DATE: March 2007 – February 2010