Challenging Therapies for Muscular Dystrophies

4th October 2018.  
Giulio Cossu, University of Manchester

On the 4th of October 2018 Giulio Cossu, of the University of Manchestterl, will give a seminar on Challenging therapies for muscular dysttrophies at 5.00 pm in the College lecture theatre. Muscular dysttrophies constitute an important group of diseases that have posed exceptional challenges to scientists and physicians. G Cossu will review progress and will highlight the major remaining challenges that have to be overcome in order to ensure successful therapy. All College students are invited to attend, especially thse reading Medicine, Biology, Biotechnology and Pharmaceutical Sciences. The poster of the lecture can be downloaded here.

In the last few years there has been significant advance in pre-clinical and clinical work of gene and cell therapy for muscular dystrophy. Currently there are several trials ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remain and it is unclear now whether they will be solved by current strategies or further improvements will be necessary. After reviewing current work in the field I will describe our current effort for an implemented protocol of cell therapy for muscular dystrophy. My laboratory has been working for many years on skeletal myogenesis and on the development of cell therapy protocols with stem cells for muscular dystrophy. We showed that neural tube (Vivarelli and Cossu, Dev. Biol. 1986) and dorsal ectoderm (Cossu et al., Development 1996) activate, through different Wnts, distinct myogenic programs in epaxial and hypaxial somitic progenitors. We also described myogenic cell heterogeneity (Cossu and Molinaro, Curr. Topics Dev. Biol. 1987) whose molecular basis has been recently elucidated (Messina et al. Cell 2010). We developed the first pre-clinical model for analysis of human myogenic progenitors in vivo (Salvatori et al., Hum Gene Ther. 1993), and discovered the myogenic potential of a bone-marrow derived, circulating progenitor cell (Ferrari et al., Science 1998). Our studies on unorthodox derivation of myogenic cells by non-somitic tissues (Tajbakhsh et al., Neuron 1994; Salvatori et al., J. Cell Sci. 1995) set the basis for the understanding of recruitment to myogenesis of non-myogenic cells, and the origin of multipotent mesoderm stem cells. We identified a mesoderm progenitor in the dorsal aorta (De Angelis et al. J Cell Biol. 1999), i.e. the mesoangioblast, able to contribute to mesoderm tissues upon transplantation (Minasi et al. Development 2002). Mesoangioblasts were used for the first successful cell therapy protocols of muscular dystrophy in mice and dogs (Sampaolesi et al. Science 2003; Nature, 2006). We characterized human mesoangioblasts as a subset of muscle pericytes (Dellavalle et al. Nature Cell Biol. 2007). Together, this data set the basis for a first clinical trial in Duchenne patients with stem cells (Cossu et al. EMBO Mol Med. 2015). We are currently optimising the protocol aiming at reaching clinical efficacy.

I am currently the Constance Thornley Professor of Regenerative Medicine at the University of Manchester. I received my MD degree from the University of Rome, then moved as Fogarty fellow at the University of Pennsylvania and in 1983 as Research Associate in Rome. In 1990 I spent an extended sabbatical at the Pasteur Institute in Paris. In 1992 I became Professor of Histology and Embryology in Rome. In 2000 I moved to Milan as Director of the Division of Regenerative Medicine at San Raffaele; in 2012 I moved as Professor of Human Stem Cell Biology to University College London and in 2013 to the University of Manchester. I began working as CAT member at EMA (2012-13). I am a member of EMBO, of the European Academy of Science, a fellow of the Academy of Medical Sciences and of Accademia dei Lincei. I am recognized for my work on muscle development and on cell therapy for muscular dystrophies. I have published more than 250 peer-reviewed papers and secured grants for more than15 M£

Section of skeletal muscle under the light microscope.

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