Gene therapy for paediatric leukaemia

Improvements in the chemotherapeutic and transplant regimens have had a sig nificant impact in improving survival rates for paediatric leukaemia. Howev er, there are still important problems to address including what options ar e available for patients with chemoresistant disease and what strategies ar e available to avoid the concerns regarding the toxicity associated with hi ghly cytotoxic treatment regimens. Gene therapy and immunotherapy protocols hold great promise. Using gene transfer of a marker gene, a number of biol ogical issues in the therapy of leukaemia have been addressed. For example, by gene marking autologous bone marrow grafts it has been possible to demo nstrate that infused marrow contributes to relapse in acute and chronic mye loid leukaemias. In the allogeneic transplant setting, genetically modified T-cells have proven valuable for the prophylaxis and treatment of viral di seases and may have an important role in preventing or treating disease rel apse. Gene transfer is also being used to modify tumour function, enhance i mmunogenicity, and confer drug-resistance to normal haematopoietic stem cel ls. With the continued scientific advancements in this field, gene therapy will almost certainly have a major impact on the treatment of paediatric le ukaemia in the future.