Italian and Israeli researchers have reported that they have successfully used gene therapy in conjunction with stem cell therapy to treat two children with an inherited immune system disorder. The research appears in the journal Science.
The children in the study had a form of severe combined immunodeficiency disorder in which they had inherited a faulty gene for an enzyme called ADA (ADA-SCID). Patients with ADA-SCID must be kept in a sterile environment as they are highly susceptible to infections. The condition can be treated by replacing the enzyme, but this is a short-term, invasive and expensive procedure.
The new treatment involved extracting stem cells from the bone marrow of the children and introducing a virus carrying a correctly functioning gene for ADA. The modified stem cells were then reintroduced to the patients. The bone marrow was seen to begin to start making new blood cells, and the two children were released from hospital with functioning immune systems.
The scientists believe that the effectiveness of the treatment was increased by the use of chemotherapy in conjunction with the gene therapy. This destroys some of the existing bone marrow, 'giving space' for the reintroduced bone marrow to create correctly functioning cells.
Sources and References
Gene therapy comes of age with the saving of a little girl's life