185th ENMC International Workshop: Stem/precursor cells as a therapeutic strategy for muscular dystrophies 3–5 June 2011, Naarden, The Netherlands

Abstract

The 185th ENMC Workshop on stem/precursor cells as a therapeutic strategy for muscular dystrophies was held at Naarden, The Netherlands, on 3–5 June 2011. Sixteen participants from seven countries (United Kingdom, The Netherlands, France, Germany, Italy, Spain and Singapore) with expertise in satellite cells, muscle stem cells, muscle development, muscular dystrophies, clinical trials and patients’ perspectives, attended. Although skeletal muscle is largely a post-mitotic tissue, it regenerates efficiently following injury. Satellite cells, quiescent cells under the basal lamina of muscle fibres, become activated, proliferate to give a pool of myoblasts that can repair and regenerate muscle fibres [1]. In muscular dystrophies such as Duchenne muscular dystrophy (DMD), satellite cells contribute to muscle fibre regeneration, but soon become exhausted and muscle fibres are lost and replaced by fibrous connective tissue and fat (reviewed [2]). Stem/precursor cells have long been considered as a possible therapy of muscular dystrophies, since work in the late 1980s on myoblast transplantation in the dystrophindeficient mdx mouse model of DMD [3,4]. However, early clinical trials on intra-muscular myoblast transplantation were disappointing, the main problems being low efficiency of donor-derived muscle regeneration and lack of migration of donor cells within transplanted muscle [5]. Nevertheless, lessons learnt from these trials may be used as a basis of future trials. In planning a stem cell clinical trial, decisions have to be made about ethical implications, age of participants, scaling up of cell preparations, testing of cells and criteria used to assess safety and efficacy.