Stéphane Roques, General Secretary of the Institute of Myology tells us about a new research unit dedicated to the development of innovative therapies.
The year 2009 starts with changes in the research units of the Institute, what are they?
Since January 1st 2009, a mixed research unit, UPMC- INSERM-CNRS-AIM has been created which is headed by Thomas Voit, Scientific and Medical Director of the Institute. The primary objective of this new unit is to develop innovative approaches to cell, gene or pharmacological therapies, based on understanding the molecular pathophysiology of a disease, and therefore adapted to it. Four of the five teams in the unit are from a reorganisation of the two existing mixed research units UMR S-582 and UMR-S 787 Myology Group and teams of the Myology Institute Association (AIM). In parallel, the research unit UMR-S 787 Myology Group headed by David Sassoon, slightly modified, was renewed for 4 years and continues its more fundamental work on molecular and cellular mechanisms involved in the development of muscle and its pathologies.
What is the idea behind this reorganisation?
We wanted to create a unit dedicated to the development of therapies, notably cellular and gene biotherapies for neuromuscular diseases. In the past few years, the members who compose this unit have guided their research in this direction; they are also involved in four of the six biotherapy trials taking place in Europe. And if the methodologies implemented by these teams can be adapted and transferred to other diseases, each one focuses on a specific project that follows the paradigm of development of pharmacological, cell and gene therapies, based on the understanding of molecular mechanisms of the disease. To summarize, after identifying a gene responsible for a disease, the detection of several phenotypes related to specific mutations follows, which encourages the creation of a database based on the mutations. In addition, the creation of cellular or animal models of this disease with a specific aspect or phenotype facilitates the exploration of pathophysiological mechanisms and a therapeutic approach at the scale of the cell and the animal.
Can you clarify the research fields of the teams from this unit?
The unit therefore has five teams whose objectives and research themes are complementary. In addition, interactions are reinforced by four servicing platforms that are common to the entire unit. Team 1, led by Gisèle Bonne, will focus on genetics and the pathophysiology of neuromuscular diseases. This team has under its responsibility the histopathology platform. Team 2, led by Gillian Butler-Browne will focus on remodeling, regeneration and cell therapy of striated muscle. This team oversees the cell culture platform. Team 3, led by Luis Garcia, will develop biotherapies, particularly gene therapy approaches for striated muscle. This team is responsible for the vectorology platform. Team 4, led by Yves Fromes, will focus on the physiological assessment of muscle and development of biomarkers. This team is in charge of the physiology platform. Finally, team 5, led by Lucie Carrier will focus on functional genomics of familial hypertrophic cardiomyopathy and heart failure, and develop therapeutic approaches for the mechanisms of autosomal dominant pathologies of striated muscle. Of course, the teams will work in synergy with each other but they will also rely on the other poles of expertise available at the Institute (NMR Laboratory CEA-AIM, reference centre) and the regional and national networks already established (Stéphane Blot’s laboratory at ENVA). Finally, of note is that each team is scientifically independent, and that the purpose of this new unit is to promote and synchronize exchanges between the teams and their platforms as well as to promote new strategies and the recruitment of new researchers.
Interview by Anne Berthomier, translation by Racquel N. Cooper