I-Stem results from the combination of two different and independent entities: a joined Research Unit of Inserm and the University Evry-Val d’Essonne (UMR 861) and the Centre d’Etude des Cellules Souches ( CECS) supported by the French Muscular Disease Association (AFM-Téléthon). I-Stem is one of the three laboratories that are directly associated to the patients’ Association. I-Stem has also benefitted until recently of a strong support from Genopole, which has registered its development as one of the major projects of the site in its strategic plans between 2005 and 2015.
- The CECS
CECS is a non profit association governed by the Law of 1901, which was created by AFM with the help of Genethon and Institute of Myology, in order to ensure the development of the I-Stem institute. CECS emplys permanent engineers and administrative staffs in close collaboration with the I-Stem academic teams.
The board of directors of CECS is as follows:
President: JF. Malaterre, Vice President of Families and AFM Administrator
- MC. Loys: AFM Administrator and Treasurer CECS
- A. Bouchard: CECS Administrator
- KS. Robert: CECS Administrator
- S. Braun: Scientific Director of the AFM
- B. Fontaine: APHP Neurologist,
- F. Lamy: Vice President of Research and AFM Administrator
The general director of the CECS is Raymond Zakhia, the scientific director is Marc Peschanski.
In 1958, patients and relatives gathered within the AFM with one goal: to cure neuromuscular diseases. Their strategy: to carry on a combat on all fronts against diseases, scientific research with patient assistance and to implement a strategy of general interest against genetic diseases. The AFM initially sought to understand the origin of the diseases, before innovating on assistance and therapy. The annual Telethon provides the means to fund research and to create a care system, assumption of responsibility and adapted diagnosis. This innovating strategy is bearing its fruits since some of these diseases considered incurable are under therapeutic test. The AFM has become one main actor of the scientific and medical research and on economy of health.
The national institute of health and medical research has the role of developing biological, medical research and in pubic health, of ensuring the diffusion of knowledge and developing the results of its research. It comprises more than 10,000 researchers, engineers, technicians and research directors, academics hospitals experts, and students, 7,000 publications each year, 300 laboratories and 60 federative research institutes to improve health.
The University of Evry-Val d’Essonne is one of the four new universities created in 1991 within the framework of the development of higher education in France and of the reorganisation of the Parisian universities. Located in an expanding district, the University was constituted as a multi-field school and turned to professionalism lessons to mix the needs for its economic and social environment. The UEVE is now attached to Paris-Saclay University (UPSay), which opens up considerable opportunities for mutualisation and new development. With more than 150 formations suggested – of which more than half in professional matter -, it gives training in scientific and technological disciplines, law and economics, management and social sciences. Acknowledged as a dynamic structure, supporting a fast integration in the active life, it counts today more than 11,000 students and meets the expectations of a wide variety of audiences, enrolled in both initial and continuing education.
The University also represents an important pole of research, with 22 laboratories and 2 doctoral schools, in particular in Life Sciences in connection with Genopole. Created in 1998 on the initiative of the French government, the local authorities and the AFM, Genopole® is carrying on an original concept: to mix in the same place academic and private research laboratories, biotechs and high level teaching. The objective of Genopole® is to support the development of large scale biology and the creation of biotechs companies in order to position France at a good level in the international scientific and industrial competition. Genopole® has quickly become a site of reference. The campus currently federates about twenty research laboratories and about sixty biotechnology companies around the University of Evry-Val d’Essonne which provides education in close liaison with the research structures of the site.
The I-Stem Institute has three different activities: basic biological research, technological and application research, and a center for biological and technological resources. For each of these major types of activities correspond different organizational frameworks, financing methods and partnerships that are gradually implemented and coordinated in order to ensure complementarity.
I-Stem first includes a biological research laboratory, whose field is defined by practical purposes but which explores fundamental areas. The study of the therapeutic application potentials of stem cells involves the characterization of their biological properties, by deciphering the mechanisms that underlie their pluripotency and their differentiation, by understanding the signals that direct their destiny towards cellular phenotypes, etc. Their use for therapeutic purposes requires in particular, in the case of substitutive cell therapies, the analysis of their capacity for integration into injured adult tissues and their tumorigenicity, the development of original delivery routes. The application to monogenic diseases also covers multiple topics of basic research: definition of pathological mechanisms, identification of biomarkers, search for compounds capable of counteracting phenotypic alterations related to the presence of causal mutations of monogenic pathologies, definition of modes of action molecular of these compounds.The basic research laboratory relies on seven teams dedicated to sets of pathologies allowing coordinated approaches: pathologies of the neuromuscular axis associated with type 1 myotonic dystrophy and spinal muscular atrophy (under the direction of Cécile Martinat), neurological disorders related to myopathies of Steinert and Duchenne (under the direction of Alexandra Benchoua), muscular involvement in Duchenne muscular dystrophy (under the direction of Christian Pinset), limb girdle myopathies (under the direction of Xavier Nissan ), Wolfram syndrome (under the direction of Laetitia Aubry), genodermatoses (under the direction of Christine Baldeschi) and retinitis pigmentosa (under the direction of Christelle Monville).
The ultimate goal of I-Stem is to provide doctors and patients with therapeutic products applied to monogenic diseases. It must therefore install the circuits that go from discovery to application, that is to say, coherent technological packages structured in a framework of industrial type that effectively, under the conditions required for human application, to develop therapeutic products. All these activities are carried by 12 engineers grouped in the team “technological platforms” (under the direction of Marc Peschanski). Their objectives are, respectively: automated mass production of stem cells and their derivatives, cell culture and quality control methodologies, high throughput sequencing, screening of high and medium rate compounds, cell imaging microscopic and automated.
The similarity that can be found between these activities and those conducted by many biotechnology companies is not fortuitous. In fact, I-Stem can be considered part of a “non-profit biotech”, which shares the business of many private companies.
I-Stem develops biological and technological resources as well as valuable know-how for its own purposes. We are able to offer these resources in collaboration, on site, to external research teams, academic and industrial, whose objectives are different from ours. I-Stem can provide for them a service activity, which can go as far as hosting teams for short term periods on the site. In accordance with its AFM-Telethon supervision, I-Stem is able to devote up to 20% of its capacity to this service activity apart from its own objectives restricted to monogenic diseases.