Stem cells potential
New leads are emerging in cell therapy, particularly with stem cells. As they have a capacity the transform themselves into different types of cells, they offer promising therapeutic prospects in the fields of organ reconstruction and tissue repair.
The AFM has therefore considerably reinforced its action in the field of stem cells :
>>> Programme aimed at the characterisation of stem cells, the study of their proliferation, plasticity and capacity to regenerate certain organsAs well as the transplants of myoblasts (muscle stem cells) which are applied in neuromuscular diseases (Duchenne myopathy, oculo-pharyngeal muscular dystrophy, facioscapulohumeral dystrophy), other stem cells can be used in degenerative diseases of the liver, nervous system and immune deficiencies etc. This work is carried out with the support of the AFM in its Généthon laboratory, at the
Cochin Institute in Paris directed by Prof Axel Kahn (adult stem cells) (agreement signed in September 2004. AFM financing 500 000 euros set aside in 2002 and 2003) and the
Biotherapy Research Institute in Montpellier directed by Bernard Klein (multipotent and adult embryo stem cells). From 2007, this Institute will receive teams of fundamental researchers, clinicians and biotechnical experts. AFM financing 2004 for the construction of a P3 laboratory: 185 000 euros.
These projects complement the research carried out by the stem cell group at Généthon.
In fact, in collaboration with Italian and US researchers, an active research effort is under way at Généthon for the development of bone marrow stem cell transplants. To begin with, the objective is to colonise affected muscles in the animal from stem cells in the blood circulation, then to identify the embryonic cell types in the bone marrow likely to provide muscle cells and identify the factors which stimulate these stem cells to become muscle fibres. In the future we can envisage stem cell transplants taken from compatible donors in order to limit as far as possible immune reactions on the part of the receiver. Another approach would be to take bone marrow from the patients themselves, select the precursor muscle cells, modify them by gene therapy and re-inject them back into the patient, thus avoiding certain immune reactions.
>>> Support for the I-Stem research programme (Institute of stem cells for the study and treatment of monogenic diseases)
Inaugurated in September 2007 by the Minister for Research and Higher Education, I-Stem is the stem cell institute for the study and treatment of monogenic diseases, of which the AFM is one of the founders. Headed by Marc Peschanski, research director at Inserm, I-Stem explores the therapeutic potential of both embryonic and adult human stem cells in rare genetic diseases. It is situated in the biopark at Evry and is the result of a partnership between the AFM, Inserm and the University of Evry-Val-d’Essonne.
At the beginning of 2005 the Agency of Biomedicine granted I-Stem the first authorisation in France to work on human embryonic stem cells from imported cell lines. In June 2006 it obtained the Agency’s authorisation to set up a bank of cell lines carrying a genetic mutation to serve as models for the study of monogenic diseases.
The first results were announced as early as June 2006: I-Stem researchers demonstrated that embryonic stem cells could differentiate themselves into cardiac cells and regenerate the failing hearts of rats. This represents a great hope for the treatment of heart failure, whether due to a common coronary attack or disease.
Thanks to Téléthon donations, the AFM provided 3.4 million Euros of the laboratory’s financing between 2004 and 2006. And in 2007 the AFM will provide another
3.4 million Euros.
- 70 researchers (85 by the beginning of 2008)
- Annual budget (2007) : 6.8 million Euros
- 1 800 square metres of laboratory space
- 5 poles of research with a particular target for each field: Human embryonic stem cell biotechnology (mass production of cells, genetic engineering and high-throughput screening), Cardiomyopathies (cell therapy, pathological modelling), Neurodegenerative diseases (cell therapy, pathological modelling of Huntington disease), Genodermatoses (pathological modelling of Clouston disease), Neuromuscular diseases (pathological modelling of Steinert myotony).
- High-throughput screening robot capable of managing the simultaneous culture of cells placed in 40 000 wells (in order to study on a large scale the efficacy of molecules on model cell lines carrying a characterised genetic mutation).
To know more
Updating of the computer graphic (October 2004)
Ethics and law
Embryonic stem cells In France, all embryo research is forbidden. Exceptionally and by special dispensation, the new law relating to bioethics promulgated on 6 August 2004 authorises embryo stem cell research for a five-year period. This research can be carried out only on frozen embryos conceived in vitro within the framework of an assisted reproduction programme, which are no longer the subject of any parental project and on condition that both members of the couple have given their written consent. The study and research protocols on these cells will be examined by the newly-created Agency of Biomedicine.Embryonic stem cells obtained by cloning This practice is not authorised by the law relating to bioethics, which describes therapeutic cloning as an offence.This technique continues to be the subject of debate, though it has been adopted in certain countries (notably Great Britain). In France, the National Ethics Committee found in favour of this technique (Recommendation 67), contrary to the European Ethics group, which declared its utilisation to be premature (14 November 2000).Foetal cellsThe law relating to bioethics requires that a precise research protocol be established.It institutes a right of opposition by the mother to the use of these tissues. This right of opposition can be exercised only after appropriate prior information about the purposes of the sample has been given.
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