A team of Italian investigators develops a strategy to use cell therapy also in advanced stage muscle degeneration

The study, conducted by Parco Biomedico San Raffaele of Castel Romano team, coordinated by Giulio Cossu, shows that muscle tissue revascularization can be induced by mesoangioblastic grafts, including in the most advanced stages of the disease.

 

Rome, 27 July  2008 – Nature Medicine, the renowned American Journal, published the findings of a research entitled Cells expressing PlGF-MMP9 restore microcirculation and promote an effective cell therapy in “old” dystrophic muscles, based on the use of mesoangioblasts; these are stems cells usually associated with blood vessels, capable of repairing injured muscle tissue and restoring its function. One of the most interesting aspects of such cells, identified by Prof. Cossu’s team in 2002, is that once they are introduced into the bloodstream, they can migrate outside blood vessels and colonize surrounding tissues. 

 

The study – funded by Parent Project Onlus, BMW, AFM (Association Francaise contre les Myopathies), Ministry of Health and Research and the European Union – is significant because in severe muscle dystrophy, like in Duchenne’s dystrophy, muscle degeneration is accompanied by an inflammatory process followed by a special scarring causing a reduced blood and oxygen supply to muscles, with further damage. The few muscle fibers left, then, receive little or no blood and are surrounded by connective tissue: this is why it is no longer possible to employ a cell therapy. The new study will allow to bypass this obstacle and use systemic cell therapy, including when muscle tissue is severely damaged.

 

 “The result achieved by Prof. Cossu’s team, mostly due to Cesare Gargioli, a young investigator whose work has been funded by Parent Project, along with Marcello Coletta and the Università di Tor Vergata’s group including Fabrizio De Grandis and Stefano Cannata, may eventually give hope to older boys, other than improving many current therapeutic options” – declared Filippo Buccella, president of Parent Project Onlus.

 

These studies open up a new outlook -  stated Giulio Cossu, Professor of Histology at the University of Milan – ; however, in mice, more than in patients, the muscle repair process continues also in the advanced stages of dystrophy, making this approach more difficult in patients, given the size of human muscles; this strategy, then, needs to be further improved.

 

Duchenne’s Muscular Dystrophy is a rare genetic disease, caused by a mutation of a gene found on chromosome X, encoding for dystrophine, an essential protein making up the muscle cell scaffolding. The disease is characterized by a progressive weakening of the entire skeletal muscle tissue, including cardiac and respiratory muscles, leading to total immobility and death. Early symptoms occur around age three and life expectancy, although doubled in recent years, rarely exceeds 25-30 years of age. Being linked to chromosome X, Dmd is transmitted by mothers, affects boys only and has an incidence of 1 in 3500. It is estimated that 5000 people are affected from Dmd in Italy.

 

The study was largely conducted at the Parco Scientifico San Raffaele di Roma laboratories, a new biomedical research unit in Rome, established thanks to the Chamber of Commerce in collaboration with Unicredit Group and Fondazione San Raffaele di Milano. Located approx. 15 km south of Rome, within the Castel Romano natural reserve, the Parco is a modern center at the forefront of scientific research in regenerative medicine, with nine research groups focusing on the cell therapy of myopathies and other genetic conditions, connective tissue engineering, cancer differential therapy, neurodegenerative disease and biomedical quality control. Thanks to its top quality facilities and services and outstanding human resources, the Parco is a major national and international center of reference for the development of basic research and biomedical, regenerative products.