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The potential for stem cell therapy in cystic fibrosis.

Gene therapy by topical delivery to the airway of patients with cystic fibrosis (CF) has proven disappointing to date. Vectors are either inefficient1 or induce an inflammatory response.2 In addition barriers such as mucus prevent transfection of airway cells, even in relatively well patients.3 It could be argued that even if these hurdles were overcome, this approach can at best offer a treatment rather than a ‘cure’ for CF. Another approach in which gene therapy may work in CF is by the utilization of stem cells, which has been successful in children with single-gene blood disorders such as adenosine deaminase (ADA) deficiency and X-linked severe combined immune deficiency disease (SCID).4,5 This method offers a potential cure to patients with CF. We envisage that the ultimate therapy will be gene therapy to CF babies by utilizing their own stored umbilical cord blood stem cells. In recent years there has been a great deal of interest and research into stem cells, with the hope of using such cells in tissue regeneration. Stem cells are a heterogeneous group with differing potentials for self-renewal and differentiation. Much interest in their potential for treating lung disease has come from several recent studies which have demonstrated that adult stem cells may show considerable plasticity with regard to their development potential. This may overcome some of the ethical concerns of using embryonic stem cells for research and therapy. This review will discuss basic stem cell biology and consider the evidence for plasticity in the lung and potential for future novel therapies for CF.