Key Benefits:
Director General of the Biomedicine Agency,
Vu la Act No. 2013-715 of 6 August 2013 to amend the Act No. 2011-814 of 7 July 2011 relating to bioethics by authorizing under certain conditions research on embryo and embryonic stem cells;
Vu le Public Health Codearticles L. 2151-5 and R. 2151-1 to R. 2151-12;
Considering the Order of Ministers for Health and Research of August 19, 2005 and the decision of the Director General of the Biomedicine Agency of June 25, 2010 authorizing the National Institute of Health and Medical Research (UMR861) to implement a research protocol on human embryonic stem cells;
In view of the decision of 17 September 2013 setting the application file model for the authorizations referred to in theArticle R. 2151-6 of the Public Health Code ;
Considering the application submitted on March 31, 2015 by the National Institute of Health and Medical Research (Unit UMR 861) for the purpose of obtaining the renewal of its research protocol authorization on human embryonic stem cells;
Considering the report of the inspection mission of the Biomedicine Agency dated 22 May 2015;
Considering the expert reports of 19 and 25 May 2015;
Having regard to the advice issued by the Guidance Board of the Biomedicine Agency on 17 September 2015;
Considering that the dystrophic myotomy of type 1 (DM1, known as Steinert's disease) is the most common of neuromuscular diseases transmitted on a dominant autosomal mode; that each child whose one of the parents is attained has a probability of 50% being carrier of abnormality; that apart from the muscular impairments (multi-sytemic impairment often in the hands and arms, dystrophys
Considering that the team's project has been, since a first authorization granted in 2005 for a period of five years having been renewed in 2010 for another five years, to develop a cellular model from human embryonic stem cells (CSEh) derived from embryos carrying the mutation or normal cells in which the transgen is introduced in order to better understand the physiopathology of the new DM1
Considering that these cellular models have led the team to major results published in very high-level international journals: identification of biomarkers, identification of microARN abnormalities (small non-coding DNA that interfere with other RNAs and block their translation into proteins) and therapeutic approach by screening of therapeutic compounds in the search for modulatory genes that can restore a normal function
Considering that the application is part of the continuity of the previous protocol and that it is therefore a research protocol that is part of a medical purpose;
Considering that in the state of scientific knowledge, it cannot be carried out without using human embryonic stem cells; that the expected result cannot be obtained by other means, in particular by the exclusive use of other stem cells; that the process of reprogramming of induced pluripotent stem cells (iPS) results, by itself, of the genetic changes that distinguish CSEh and iPS
Considering that the scientific and medical community agrees that for the next few years, CSEh appears in the current state of safer and healthy knowledge in this type of application, which justifies the choice of CSEh; that there is no alternative cellular product with comparable efficiency, and that it is therefore justified that this team pursues its program with CSEhs, in the interest of patients;
Considering, therefore, that the applicant provides sufficient elements concerning the scientific relevance of the research project, on the one hand, and its conditions of implementation under ethical principles, on the other hand; that it justifies in particular that the project will be carried out in accordance with the ethical principles relating to research on embryo and human embryonic stem cells and that these cells have been obtained in accordance with the fundamental principles provided to human embryos Articles 16 to 16-8 of the Civil Code and with the prior consent of the generator couple and without any payment, in any form, being allocated to them;
Considering that the titles, diplomas, experience and scientific work provided in support of the application allow to ensure the skills of the research manager and the members of the team in this matter; that the INSERM UMR 861 unit is part of the I-Stem laboratory, institute dedicated to stem cells with therapeutic aim directed by Marc Peschanski; that the human and financial means of the team are in keeping with the project;
Considering that the premises, materials, equipment, processes and techniques are adapted to the proposed research activity; that this research will be carried out in conditions that guarantee the safety of persons engaged in a professional activity on the site, the respect of the applicable provisions for the protection of the environment, the respect of the sanitary safety rules as well as the safety, quality and traceability of embryos and embryonic cells; that the material conditions of security, conservation,
Decides:
The National Institute of Health and Medical Research (UMR Unit 861) is authorized to implement, under the conditions described in the application for authorization, the research protocol on human embryonic stem cells with the purpose of the study of the modelling of the myotonic dystrophy of type 1 ( Steinert disease) using human embryonic stem cells. This research is under the responsibility of Ms. Cécile Martinat and Ms. Sandrine Baghdoyan.
This authorization is granted for a period of five years. It may be suspended at any time for a maximum of three months, in the event of a violation of the legislative or regulatory provisions, by the Director General of the Biomedicine Agency. Authorization may also be withdrawn, in accordance with the terms and conditions set out in the provisions of Public Health Code.
Any changes to the elements in the application for authorization must be made available to the Director General of the Biomedicine Agency.
The Assistant Director General for Resources of the Biomedicine Agency is responsible for the execution of this decision, which will be published in the Official Journal of the French Republic.
Done on 13 October 2015.
A. Courts
