Autologous skeletal myoblast transplantation for severe postinfarction left ventricular dysfunction

Menasché, Philippe; Hagège, Albert; Vilquin, Jean‐Thomas; Desnos, Michel; Abergel, Éric; Pouzet, Bruno; Bel, Alain; Sarateanu, Sorin; Scorsin, Márcio; Schwartz, Ketty; Bruneval, Patrick; Benbunan, M; Marolleau, Jean‐Pierre; Duboc, Denis

doi:10.1016/s0735-1097(03)00092-5

Cited by 1,032 publications

(742 citation statements)

References 34 publications

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“…We evaluated in vitro proliferation and differentiation and in vivo regenerating ability of myoblasts obtained from phenotypically unaffected muscles of five FSHD patients and of 10 control donors prepared in a previous clinical trial using the same procedures. 34 We could not observe any difference between FSHD and control myoblasts.…”

Section: Introductionmentioning

confidence: 56%

“…31,34 These myoblasts also preserve, at different passages, their ability to in vitro differentiate and to in vivo participate to muscle regeneration. The combination of our results and studies on intramuscular injection procedures raise the possibility of future clinical trials of autologous cell therapy for FSHD patients.…”

Section: Discussionmentioning

confidence: 99%

“…The 10 control donors (C 1-10) had been recruited in the context of a previous clinical study dedicated to the autologous MT in postinfarction left ventricular dysfunction. 34 The mean ages of the FSHD patients and control donors were, respectively, 5375 and 61711 years. All FSHD patients and controls were males.…”

Section: Patient Selectionmentioning

confidence: 99%

“…The threshold number of cells required for potential clinical trials was arbitrarily established at 8 Â 10 8 on the basis of previous experience. 34 To reach this number, FSHD cells were grown up to P5, for three supplementary divisions, and overall performed 9.8 divisions. We produced 8.5 Â 10 8 to 1.6 Â 10 9 myoblasts (mean 1.06 Â 10 9 ) within 22.7 days.…”

Section: Cell Productionmentioning

confidence: 99%

“…22,29 This issue is completely avoided in the context of autologous MT, which has been successfully developed as an experimental treatment for postischemic heart failure or dilated cardiomyopathy, showing its feasibility, safety and its great clinical interest. [32][33][34][35][36] In some myopathic condition such as FSHD, characterized by a relatively localized muscle involvement, and the absence of therapeutic options, autologous MT could be considered as an alternative strategy. In this case, unaffected muscles could be the source of myoblasts to be transferred into affected muscles in order to improve their regeneration ability.…”

Section: Introductionmentioning

confidence: 99%

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Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Vilquin

Sacconi

et al. 2005

Gene Ther

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Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant disease characterized by a typical regional distribution, featuring composed patterns of clinically affected and unaffected muscles. No treatment is available for this condition, in which the pathophysiological mechanism is still unknown. Autologous transfer of myoblasts from unaffected to affected territories could be considered as a potential strategy to delay or stop muscle degeneration. To evaluate the feasibility of this concept, we explored and compared the growth and differentiation characteristics of myoblasts prepared from phenotypically unaffected muscles of five FSHD patients and 10 control donors. According to a clinically approved procedure, 10 9 cells of a high degree of purity were obtained within 16-23 days. More than 80% of these cells were myoblasts, as demonstrated by labeling of the muscle markers CD56 and desmin. FSHD myoblasts presented a doubling time equivalent to that of control cells; they kept high proliferation ability and did not show early telomere shortening. In vitro, these cells were able to differentiate and to express muscle-specific antigens. In vivo, they participated to muscle structures when injected into immunodeficient mice. These data suggest that myoblasts expanded from unaffected FSHD muscles may be suitable tools in view of autologous cell transplantation clinical trials.

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Section: Introductionmentioning

confidence: 56%

Section: Discussionmentioning

confidence: 99%

Section: Patient Selectionmentioning

confidence: 99%

Section: Cell Productionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Vilquin

Sacconi

et al. 2005

Gene Ther

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Adult Stem Cell Therapy and Heart Failure, 2000 to 2016

2016

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Importance Stem cell therapy is a promising treatment strategy for patients with heart failure, which accounts for over 10% of deaths in the U.S. annually. Despite over a decade of research, further investigation is still needed to determine whether stem cell regenerative therapy is clinically effective and can be routinely implemented in clinical practice. Objective The purpose of this review is to describe the current progress in cardiac stem cell regenerative therapy using adult stem cells and highlight the merits and limitations of clinical trials performed to date. Evidence Review Information for this review was obtained through a search of PubMed and the Cochrane database for English language studies published between January 1, 2000 and April 20, 2016. Twenty-nine randomized clinical trials and 7 systematic reviews and meta-analyses were included in this review. Findings Although adult stem cells were once believed to have the ability to create new heart tissue or grow blood vessels, preclinical studies suggest instead that these cells release cardio-protective paracrine factors that activate endogenous pathways, leading to myocardial repair. Subsequent randomized controlled clinical trials, the majority of which used autologous bone marrow mononuclear cells, have found only a modest benefit in patients receiving stem cell therapy. The lack of a significant benefit may result from variations in trial methodology, discrepancies in reporting, and an over-reliance on surrogate endpoints. Conclusions and Relevance Although stem cell therapy for cardiovascular disease is not yet ready for routine clinical application, significant progress continues to be made. Physicians should be aware of the current status of this treatment so that they can better inform their patients who may be in search of alternative therapies.

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Post‐Myocardial Infarction

2006

Wiley Handbook of Current and Emerging Drug Therapies

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Autologous skeletal myoblast transplantation for severe postinfarction left ventricular dysfunction

Cited by 1,032 publications

References 34 publications

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Normal growth and regenerating ability of myoblasts from unaffected muscles of facioscapulohumeral muscular dystrophy patients

Adult Stem Cell Therapy and Heart Failure, 2000 to 2016

Post‐Myocardial Infarction

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