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Injured Tissue repair targeting patients own stem cells while still in the body now one step closer

Stresa (Italy), March 29th 2010: A collection of top-class scientists and companies today launched a large scale collaboration designed to develop new approaches aimed at stimulating stem cells for treating degenerative diseases.

Stresa (Italy), March 29th 2010: A collection of top-class scientists and companies today launched a large scale collaboration designed to develop new approaches aimed at stimulating stem cells for treating degenerative diseases. Globally, degenerative diseases impact an enormous number of patients and their families and are typically treated via symptomatic pharmacological approaches. In contrast, this newly formed consortium are developing therapies that target the stem cells found within damaged tissues so that they can be encouraged to rapidly repair the tissue themselves. This consortium of European researchers have undertaken to develop new pharmacological approaches and bring them to the clinic as well as back to the laboratory for further improvements. The strategy of developing pharmacological agents to target stem cells for regenerative medicine complement existing strategies used in commercial pharma development. This strategy reduces existing barriers to development and translation to the clinic and affords a far greater chance to be of eventual benefit to citizens. This new project is coordinated by Dr. David Sassoon, originally of Mount Sinai Medical School in New York who is now with Inserm and the University Pierre and Marie Curie (Paris, France). The project is co-funded by the European Commission via the 7th Framework programme for research, and therefore the taxpayers themselves. The project, colloquially and conveniently abbreviated to ‘Endostem’, will develop therapeutics which benefit those same tax payers within the next few years. Dr Anne Rutkowski, Chairman and co-founder of Cure CMD – a US-based charity helping patients with congenital muscle dystrophy – declared: “the upcoming joint Endostem/Optistem meeting provides hope to families of rare diseases, including the Congenital Muscular Dystrophies (CMDs), for which there is currently neither treatment nor cure […] Discussions to identify current knowledge gaps and bridges from basic stem cell science to translational research will highlight stem cells as a future treatment modality for these and other rare diseases”. Indeed, the most advanced therapeutics are already in mid-stage clinical trial, with two additional trials using other therapeutics planned for the forthcoming 18 months; more preliminary development work is also being performed for the next generation of therapeutics. While initial focus and proof-of-principle is on muscle and blood vessel repair, two of the building blocks necessary for virtually all effective tissue regeneration, the impact is far more reaching as the ground work will be laid for all degenerative or traumatic damage to be targeted. The obvious long-term benefits will be for age-related problems that eventually impact all of us and potential sport and activity related injuries, which massively impact the economy through lost days of work. In line with the newly emerging open source approach to research by large pharma companies, as part of this effort, Novartis is collaborating with leading academic specialists from Inserm (France), EPFL (Switzerland), EMBL (Europe), IRCCS E. Medea IFOM, Fondazione Telethon, Fondazione Monte Tabor, CNR (all Italy), the Pompeu Fabra University (Spain) and the University of Frankfurt (Germany) and small companies who are involved in the therapeutic development which include HMG Biotech S.r.l. Acceleron Medicines Ltd and Coretherapix S. U. Dr Jas Seehra, senior vice-president and chief scientific officer of Acceleron Medicines explained : “In recent years we have learned about the existence of stem cells and their enormous ability to proliferate but many technical and regulatory challenges have prevented the rapid development of therapies. Understanding how to stimulate endogenous stem cells present in each one of us provides an enormous opportunity to rapidly develop therapies for diseases where none exist today”. For more information please contact: David Sassoon Director Myology Group UMR S 787 Inserm Université Paris 6 Pierre et Marie Curie 105 bd de l'Hôpital 75634 - Paris Cedex 13 FRANCE david.sassoon@upmc.fr

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