In London’s Great Ormond Street Children’s Hospital, the doors to certain rooms are sealed because the children on that ward have serious genetic defects in their immune systems called Primary Immunodeficiencies or (PID) which means they have to live in completely sterile surroundings. Children with PID don’t survive without a bone marrow transplant and fifty such transplants are carried out each year. Until now chemotherapy has been used to kill the patient’s own bone marrow before the transplant.

But doctors here have discovered a new form of treatment. PID patients can now be given antibodies instead of standard chemotherapy before a transplant to make space for the donor cells. This is more effective and has fewer side effects than traditional chemotherapy which can cause organ damage and infertility. Dr Persis Amrolia, a specialist in the area, says that with the antibody protocol, staff see no hair loss, virtually no sickness and very little damage to the liver, lungs and gut. They hope that this will translate later on into less late effects, so that the children will go on to grow normally, go through puberty normally and be fertile, which was not always the case after chemotherapy. In this new technique antibodies target a molecule specific to bone marrow cells, meaning that the antibody effectively kills the bone marrow but leaves other tissues unaffected. So far 13 out of 16 patients in the trial have survived and have been cured. Now doctors will use this technique to develop similar approaches to children with other genetic diseases like leukaemia. For more information about the hospital: tagURLhttp://www.ich.ucl.ac.uk For information about the charity which supports the hospital: tagURLhttp://www.gosh.orgMore about: Medical research, Research

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