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Claire de Thoisy-Méchin

Press Relations

Tel. +33 (0)1 44 27 23 34

Email: claire.de_thoisy-mechin@upmc.fr


In English:

Katherine Tyrka

International Press Relations

tel. +33 (0)1 44 27 51 05

Email: katherine.tyrka@upmc.fr

New therapies for degenerative diseases

Several research groups have allied with industry partners to launch EndoStem, a large European collaborative project to advance the treatment of muscle diseases that affect a large number of patients. The project will promote the development of strategies involving skeletal muscle, blood vessels, immune system and stem cells to repair damaged muscle tissue directly.


This new collaborative project of the 7th Framework Programme, coordinated by Inserm (David Sassoon, research director at INSERM UMR S 787-Myology Group Inserm / UPMC / Association Institut Myology) aims to develop therapies that will benefit patients with muscle diseases. This new project targets all stages of the development of the therapies: from fundamental research to clinical trials. With this strategy, the project is given every opportunity to achieve concrete results that will benefit patients with muscle diseases, such as aging muscles. Dr. Anne Rutkowski, president of the American Association Cure CMD which helps patients with congenital muscular dystrophy, said: "The launch of the project EndoStem brings hope to patients with rare diseases like congenital muscular dystrophy, for which there is currently no treatment (...) EndoStem will allow us to use stem cells as a treatment for muscular dystrophy and other rare diseases.”


The most advanced candidate drugs are already involved in clinical trials. Two new trials are planned in 18 months with new pharmacological compounds. The 14 partners (1) are working to develop a new generation of therapeutics. The initial goal is to repair the muscle and blood tissues but the impact of research may serve as a basis for the treatment of any degenerative disease or tissue damage: age-related degeneration, diseases or injuries related to sport practices. Dr. Jas Seehra, Vice President and Chief Scientific Officer of Acceleron Medicines, one of the companies involved in developing innovative therapies, explains: "In recent years we have discovered the existence of stem cells and their ability to proliferate. However, many technical and regulatory barriers have prevented the rapid development of new therapies. Understanding how to stimulate stem cells, present in all of us, offers a tremendous opportunity to develop new therapies against diseases for which there is currently no treatment.”


 (1) Novartis, CNR (Italy), Inserm (France), EPFL (Swiss), EMBL (Europe), IRCCS, IFOM, Fondazione Telethon, Fondazione Monte Tabor (Italy), Université Pompeu Fabra (Spain), Université de Francfort (Germany), HMGBiotech S.r.l. (Italy), Acceleron Medicines Ltd (England) and Coretherapix S.U. (Spain)