Cell Transplantation (Jul 2007)
Autologous Transplantation of Muscle-Derived CD133+ Stem Cells in Duchenne Muscle Patients
- Y. Torrente,
- M. Belicchi,
- C. Marchesi,
- G. D'antona,
- F. Cogiamanian,
- F. Pisati,
- M. Gavina,
- R. Giordano,
- R. Tonlorenzi,
- G. Fagiolari,
- C. Lamperti,
- L. Porretti,
- R. Lopa,
- M. Sampaolesi,
- L. Vicentini,
- N. Grimoldi,
- F. Tiberio,
- V. Songa,
- P. Baratta,
- A. Prelle,
- L. Forzenigo,
- M. Guglieri,
- O. Pansarasa,
- C. Rinaldi,
- V. Mouly,
- G. S. Butler-Browne,
- G. P. Comi,
- P. Biondetti,
- M. Moggio,
- S. M. Gaini,
- N. Stocchetti,
- A. Priori,
- M. G. D'angelo,
- A. Turconi,
- R. Bottinelli,
- G. Cossu,
- P. Rebulla,
- N. Bresolin
Affiliations
- Y. Torrente
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- M. Belicchi
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- C. Marchesi
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- G. D'antona
- Department of Experimental Medicine, Human Physiology Unit, University of Pavia, Pavia, Italy
- F. Cogiamanian
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- F. Pisati
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- M. Gavina
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- R. Giordano
- Centro Trasfusionale e di Immunologia dei Trapianti, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
- R. Tonlorenzi
- Stem Cell Research Institute, San Raffaele Hospital, Italy
- G. Fagiolari
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- C. Lamperti
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- L. Porretti
- Centro Trasfusionale e di Immunologia dei Trapianti, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
- R. Lopa
- Centro Trasfusionale e di Immunologia dei Trapianti, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
- M. Sampaolesi
- Stem Cell Research Institute, San Raffaele Hospital, Italy
- L. Vicentini
- Department of Surgery, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- N. Grimoldi
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- F. Tiberio
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- V. Songa
- Department of Anesthesia and Critical Care Medicine, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- P. Baratta
- Department of Anesthesia and Critical Care Medicine, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- A. Prelle
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- L. Forzenigo
- Radiology Unit, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- M. Guglieri
- IRCCS Eugenio Medea, Bosisio Parini, Italy
- O. Pansarasa
- Department of Experimental Medicine, Human Physiology Unit, University of Pavia, Pavia, Italy
- C. Rinaldi
- Department of Experimental Medicine, Human Physiology Unit, University of Pavia, Pavia, Italy
- V. Mouly
- CNRS, Cytoskeleton and Development, Paris, France
- G. S. Butler-Browne
- CNRS, Cytoskeleton and Development, Paris, France
- G. P. Comi
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- P. Biondetti
- Radiology Unit, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- M. Moggio
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- S. M. Gaini
- Department of Surgery, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- N. Stocchetti
- Department of Anesthesia and Critical Care Medicine, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy
- A. Priori
- Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy
- M. G. D'angelo
- IRCCS Eugenio Medea, Bosisio Parini, Italy
- A. Turconi
- IRCCS Eugenio Medea, Bosisio Parini, Italy
- R. Bottinelli
- Department of Experimental Medicine, Human Physiology Unit, University of Pavia, Pavia, Italy
- G. Cossu
- Stem Cell Research Institute, San Raffaele Hospital, Italy
- P. Rebulla
- Centro Trasfusionale e di Immunologia dei Trapianti, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
- N. Bresolin
- IRCCS Eugenio Medea, Bosisio Parini, Italy
- DOI
- https://doi.org/10.3727/000000007783465064
- Journal volume & issue
-
Vol. 16
Abstract
Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive muscle disease due to defect on the gene encoding dystrophin. The lack of a functional dystrophin in muscles results in the fragility of the muscle fiber membrane with progressive muscle weakness and premature death. There is no cure for DMD and current treatment options focus primarily on respiratory assistance, comfort care, and delaying the loss of ambulation. Recent works support the idea that stem cells can contribute to muscle repair as well as to replenishment of the satellite cell pool. Here we tested the safety of autologous transplantation of muscle-derived CD133+ cells in eight boys with Duchenne muscular dystrophy in a 7-month, double-blind phase I clinical trial. Stem cell safety was tested by measuring muscle strength and evaluating muscle structures with MRI and histological analysis. Timed cardiac and pulmonary function tests were secondary outcome measures. No local or systemic side effects were observed in all treated DMD patients. Treated patients had an increased ratio of capillary per muscle fibers with a switch from slow to fast myosin-positive myofibers.
