Posted on 05/10/2007 6:22:34 PM PDT by Coleus
CureDuchenne will sponsor a one-year research program that will validate Dr. Giulio Cossu’s stem cell research under GMP (Good Manufacturing Procedures) conditions. Dr. Cossu’s long-term objective is to conduct phase I/II clinical trials in Duchenne muscular dystrophy (“DMD”) pediatric patients with this novel cell therapy protocol based on intra-arterial delivery of donor mesoangioblasts. The immediate objective of Dr. Cossu is to develop a clinical grade protocol that will allow transplantation of patients with mesoangioblasts in a successive clinical trial. During this study, Dr. Cossu will prepare an IMPD, the European equivalent of a US IND (Investigational New Drug Application), and submit to the Regulatory Authorities for the formal authorization of the clinical trial.
Dr. Cossu, director of the Stem Cell Research Institute at the San Raffaele Scientific Institute of Milan in Italy, announced in November 2006 promising results of stem cell transplantation into dogs affected by Duchenne muscular dystrophy. These adult stem cells, called mesoangioblasts, can be harvested relatively easily from small blood vessels and can be grown in tissue culture and still maintain the ability to make muscle cells. The donor cells were then introduced into the dystrophic dog via arteries and in the case of one dog, injected directly into the aorta. The dog receiving the mesoangioblasts in the aorta was dramatically improved, walking well 5 months after the final treatment. The dogs receiving the donor cells via the arteries were improved but not to the same extent. These dogs had almost normal levels of the dystrophin protein and their muscle function improved. This study should provide, within one year of work, definitive information on the safety and efficacy of donor stem cells before a clinical trial is started.
About Duchenne Muscular Dystrophy
Every year, an estimated 20,000 babies (almost all of them boys) are born with Duchenne muscular dystrophy. Their muscle cells will die and not regenerate. They will be in wheel chairs by 12 and, historically, most have not survived their teenage years. An estimated 1 in every 3,500 boys worldwide is afflicted with Duchenne. It knows no boundaries and crosses all cultures and races. The gene mutation that causes Duchenne is usually passed from mother to son, but fully one-third of cases are spontaneous. It can happen in any family. There currently is no cure, but for the first time, there are promising therapies on the horizon.
About Dr. Giulio Cossu
Dr. Cossu’s group has been working for a number of years on the signals and mechanism that regulate the formation of skeletal muscle during embryonic development and, after birth, when the muscle tissue is damaged as a result of an injury or a primary myopathy. For more information, contact Dr. Cossu at the Stem Cell Research Institute, Dibit, H. San Raffaele; Tel: +3902 2643 4954, courriel: cossu.giulio@hsr.it.
About CureDuchenne
CureDuchenne is a non-profit organization founded in 2003 by Debra and Paul Miller. CureDuchenne’s vision is its name … to cure Duchenne muscular dystrophy. CureDuchenne aggressively seeks the most promising, leading-edge research breakthroughs and expedites them to the clinical trial process. The ultimate goal … give the boys that have Duchenne now a chance to live a normal life by expediting the availability of treatments/therapies and/or a cure that can give quality of life to Duchenne boys, much like insulin does for diabetics. For more information, contact: CureDuchenne, 3334 E. Coast Hwy. #157, Corona del Mar, CA 92625. Visit our website at www.cureduchenne.org. Call 949-721-4063 or email debra@cureduchenne.org.
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