Ethics committee
Following the symposium organized by ELA International in September 2022 on “Therapeutic Innovation: what rules and access?”, ELA International decided to set up a research ethics committee.
The scientific community mobilized
Most leukodystrophies begin in childhood and it is the parents who notice the first symptoms. When the diagnosis falls, although devastating, it opens the door to possible treatment. But how do you manage the disease? For ELA it has been a daily struggle for more than 30 years. What can we do? How do we access treatments when they exist or therapeutic trials? Should we screen from birth? So many questions that raise ethical questions.
Its members
This committee brings together 11 experts in ethics, law, human sciences, representatives of ELA families, and scientists, to promote a collegial and multidisciplinary work.
The committee has families representing ELA, who are experts in their disease. Their testimonies and their positions on ethical issues are valuable to feed the debate.
Guy Alba
Founder member and President of ELA International – Luxembourg
Father of a young adult with adrenomyeloneuropathy, he founded ELA in 1992 with some families. He was behind the creation of this committee.
Crystelle Cottart
President of ELA France
Mother of a child with Pelizaeus-Merzbacher disease, is herself a carrier of the mutation. Her position as a mother but also as a patient is invaluable to better understand the ethical issues of research and the management of the disease.
Gaël de Miomandre
President of ELA Belgium
Is very involved in ethical issues regarding his family situation. Himself a father of 3 children, he had to deal with the disease and the ethical issues raised by screening and therapeutic trials.
Carmen Sever
President of ELA Spain
Mother of 2 girls affected by metachromatic leukodystrophy, one sick, the other carrier. She already chairs the Ethics Committee of the Community of Madrid as a parent of sick children and, as such, takes a great interest in these issues.
It also includes international experts and clinicians, specialists in leukodystrophies, on which the scientific endorsement of this committee is based.
Jean-Hugues Dalle
Pediatrician hematologist Robert Debré Hospital – France
He works on leukodystrophies and, for more than 10 years, on hematopoietic stem cell transplantation and gene therapy. He is confronted every day with the ethical aspect of transplants in children.
Florian Eichler
Pediatrician neurologist Massachusetts Hospital – United States of America
Director of the Department of Leukodystrophies at the Massachusetts General Hospital in Boston and Chairman of the Scientific Council of ELA International.
Nicole Wolf
Pediatrician neurologist UMC Amsterdam – Netherlands
She works on metachromatic leukodystrophy and hypomyelinating diseases. She is familiar with these diseases and their medical management.
Finally, this committee benefits from the experience of experts from different backgrounds.
Grégoire Moutel
Director of the Regional Ethical Reflection Area – France
Professor Grégoire Moutel, a specialist in ethical issues and alongside the association for several years, is supporting ELA in this project. Responsible for a regional space for bioethics reflection at the University of Caen, he is a member of the bioethics law commissions. He leads the meetings of the ethics committee of ELA International and works closely with the association to move forward on these topics.
Bruno Flesselles
Industrial Property Consultant in Paris – France
He is an intellectual property consultant. He is very interested in ethical issues for having worked on issues of exclusive exploitation rights for companies, and on the valuation of treatments.
Nicolas Koebel
CEO Gene therapy company targeting CNS diseases – United Kingdom
He led the commercialization of Libmeldy (lentiviral vector used in stem cell transplantation) for three years, following its approval by the European Medicines Agency in 2020. Since September 2022, he has been Chairman and CEO of a gene therapy company targeting “refractory epilepsy”. Within the committee, he represents the perspective of industry as a stakeholder in health systems in Europe.
Séverine Mathieu
Director of Studies “Sociology of Religions and Ethics” – France
This expertise of the humanities without direct link with leukodystrophies brings an additional dimension to the committee.
Its role
The Ethics Committee supports ELA International on ethical issues in its research activities. It helps ELA to feed its reflection and positioning. Topics such as newborn screening, how to better involve the patient in clinical research, how to facilitate access to innovative treatments and frame ELA’s partnerships in research are at the heart of the discussions.
Committee opinion and ethical charter
For each theme, the committee’s opinion is compiled in a document which will serve as a support for ELA International’s actions. The committee’s long-term objective is to draw up an ELA ethics charter, a fundamental document that will define ELA’s principles, values and commitments.
Its mission
On the basis of the opinions issued and the ethical charter, the ethics committee may examine proposals, projects or actions to determine whether they are in line with ELA’s ethical values. They will be able to provide informed advice and recommendations. It will also ensure that there are no conflicts of interest that could compromise the integrity of the association’s decisions and actions. The committee can also play a role in informing and raising families’ awareness of ethical issues.
Its functioning
Members meet regularly in virtual session. A simultaneous translation into English and Spanish is proposed to allow all committee members to follow the sessions and participate actively. Depending on the theme, speakers from outside the committee, both families and professionals, may be invited to testify and provide elements of reflection. The sessions are punctuated by times of exchange and discussion.