Published in Touching Lives June 2005

Hurler syndrome is a serious disorder caused by a single faulty gene. Sufferers experience progressive mental and physical problems and rarely live beyond ten years of age. The only current treatment is a bone marrow transplant, but this is limited by the availability of donors and frequent rejection of the transplanted cells.

Mesenchymal stem cells can develop into other body tissues including bone (above left), fat (above) and cartilage (left)

Thanks to a grant of more than £105,000 from Action Medical Research, Research Training Fellow Dr John Grainger has been investigating the treatment of Hurler syndrome in a transplant model. The project, based at Manchester Children’s Hospital, explored the use of mesenchymal stem cells (MSC) — rare cells that can be derived from the patient’s bone marrow and have the potential to develop into other tissues of the body. MSC can dampen down the immune reaction to transplanted bone marrow cells, reducing the possibility of rejection.

Dr Grainger has developed a successful method for isolating and purifying MSC, and has shown that they can develop — in the laboratory — into different body tissues including bone, fat and cartilage cells. In addition, by using a transplantation model, Dr Grainger found that when MSC were administered alongside a conventional bone marrow transplant, a dramatically higher uptake of the bone marrow was observed.This demonstrates the potential of MSC to improve the success of bone marrow transplants.

Dr Grainger commented, “The funding from Action Medical Research enabled us to establish a working transplant model to investigate the potential benefits of MSC in a transplant setting. Clearly the work done here is only the first step of a long journey to reach the clinic. Before studies can be undertaken in humans, further work is needed so that any benefits can be maximised and, crucially, made as safe as possible.”