Dr. Giulio Cossu from the Milan University and colleagues have developed a new cellular treatment that provides hope for people suffering from advanced forms of muscular dystrophy. Sclerosis and reduced microvessel density characterize advanced stages of muscular dystrophy and hamper cell or gene delivery, precluding treatment of most individuals with Duchenne muscular dystrophy. In this study, modified tendon fibroblasts expressing an angiogenic factor and a metalloproteinase have been successfully tested in aged dystrophic mice, allowing to restore a vascular network and reduce collagen deposition. The modified fibroblasts were injected into muscles where they destroy the fibrous sclerotic tissue by reducing excess collagen deposits produced by scarring and restore a vascular network in the tissue. Once the fibrous tissue was destroyed, the researchers then treated the underlying problem using mesoangioblasts. Although mesoangioblasts treatments have been very successful in animals at an early stage of the disease, advanced sufferers have previously been unable to benefit due to the fibrous tissue created by scarring. This dual cellular treatment technique using modified fibroblasts and mesangioblasts has been successfully achieved in mice at an advanced stage of this disease as well as in young mice. Although technical problems remain to be resolved these data open the possibility of extending new therapies to currently untreatable individuals.

Nat Med. 2008 Jul 27. [Epub ahead of print]