logo leukotreat

Under the coordination of Prof. Odile Boespflug-Tanguy (University of Auvergne / Université Paris Diderot, France), the European scientific community of leukodystrophies rallied around multidisciplinary project entitled “LeukoTreat – Therapeutic challenge in Leukodystrophies: Translational and ethical research Towards clinical trials “funded by the 7th framework Programme for Research and Development of the European Commission since March 2010.

The project, with 6 million over three years and includes 23 partner institutions in seven European countries, aims to promote the development of innovative therapeutic strategies for the treatment of leukodystrophies known causes (adrenoleukodystrophy, metachromatic leukodystrophy , Krabbe disease, Pelizaeus-Merzbacher disease, Canavan disease, CACH syndrome, Alexander disease).

You are a patient or family member with a patient with leukodystrophy?

You can contribute to the work of the research project by completing a questionnaire LeukoTreat whose goal is to understand your expectations in terms of information and interaction between you and the medical world in order to promote research in listening patients and their families.

Download the questionnaire

The LeukoTreat project gathers experts  :
  • teams of basic and clinical research of European renown,
  •   SMEs specialized in the development of information systems in the European project management and in the development of innovative therapeutic molecules for neurodegenerative
  •   Experts in medical ethics
  •   associations of patients with leukodystrophies

photo de groupe


Seven working groups were defined:

Working Group No. 1 leukodystrophies characterization for the development of therapies

Responsible for: E. BERTINI, Ospedale Pediatrico Bambino Gesu, Rome, Italie

Objective: To collect clinical information and biologiquessur leukodystrophies and sharing at European level with the medical community through a bioinformatics platform managing clinical databases, biological samples (biobank) and genetic mutations. Data will be analyzed to better understand the European level (i) the number of patients likely to benefit from these therapies (epidemiology), (ii) the natural history of the disease in order to assess more effectively a potential therapeutic effect and (iii) the effect of genetic defects on the response to treatment.


Working Group No. 2 biomarkers for the treatment of leukodystrophies

Manager: A. PUJOL, Fundació Privada Institut d'Investigacio Biomedica de Bellvitge, Barcelona, Spain
Objective: To determine, in biological samples collected from patients, markers (biomarkers) of the disease or its severity in order to improve the detection of patients to have a quantifiable marker for monitoring the disease or find new therapeutic targets. This work focuses on the one hand to the validation of markers already identified as potentially interesting for some leukodystrophies and secondly to identify new markers through the development of innovative technologies (lipidomics).

Working Group No. 3 pharmacological strategies to treat leukodystrophies

Responsibles for : H. WERNER, Max Planck Gesellschaft zur Foerderung der Wissenschaften e.v., Göttingen, Allemagne
Objective : evaluation :

(i) the potential of some molecules with neuroprotective properties, antioxidant, anti-stress proteins capable of eliminating abnormally formed using cellular and animal models of leukodystrophies,
(ii) of the modulation of the expression of a specific gene in order to minimize the toxic effect of the transfer to the cell,
(iii) the targeted enzyme replacement therapy in the brain.

Working Group No. 4 gene and cell therapies innovative leukodystrophies

Responsibles for : P. AUBOURG, Inserm, Paris, France
Objective : develop innovative strategies such as gene therapy and cell therapy for the greatest number of leukodystrophies known cause.

Working Group No. 5 ethical impact of therapeutic challenges in leukodystrophies

Responsibles for : G. MOUTEL, université Paris Descartes, Paris, France
Objective : determine expectations and impacts for patients with leukodystrophies known cause and their families:

(i) the development of new therapies to optimize information and communicationnécessaire the implementation of clinical trials.
(ii) their involvement as agents of clinical research
The work carried out by the laboratory of Medical Ethics at the University Paris Descartes and ELA, invite the participation of patients with leukodystrophies known cause and their families throughout Europe, these clinical trials are designed to s to send to all European patients.

Working Group No. 6 disseminating information

Responsibles for :  ELA association, France
Objectives: dissemination of information and discoveries made during the project with patients, doctors, researchers and the general public through a dedicated website, newsletters, scientific publications, events ...

Working Group No. 7 project management

Responsibles for : Auvergne University, France
Objective: the administrative management of the project and ethical issues associated with specific project information and permits patients to participate in research, clinical trials in terms of the animal.

En savoir plus:

LeukoTreat website

The Ethics Committee met for the first time on 19 March 2010 under the chairmanship of Dr Ingrid Callies. Its role is to ensure the ethical aspects during the project LeukoTreat and be a place of reflection in relation to the ethical aspects associated with leukodystrophies.

Sit in this instance:

  • Independent members

Marc Dupont, lawyer
Dr. Marie-France Mamzer nephrologist
Dr Marie-Laure Explorer, neurologist
Sylvie Seguret psychologist
Roger Picard, representing patients' association - Association Huntington Hope
Anne-Sophie Lapointe, a representative of patients' association - Association VML
Jean-Christophe Coffin, a social scientist
Béatrice Godard, sociologist
Benaroyo Lazarus, an expert in medical ethics


  • Members invited

Prof. Odile Boespflug Tanguy, Project Coordinator
Professor Patrick Aubourg, a member of the steering committee of the project
Dr Aurora Pujol, a member of the steering committee of the project
Marie-Louise Vendeville coordinator, paramedic reference center for rare diseases "leukodystrophies"

Under the Working Group No. 5
Dr. Gregory Moutel, deputy director of the Laboratory of Medical Ethics, University of Paris Descartes, France
Dr Nathalie Duchange expert in ethics, medical ethics, laboratory
Thomas Leclercq, PhD
Boris Loeve, PhD
Marie-Claude Blondeau, psychologist, coordinator patients / families for the project
Tracy Coutrix, representative association of patients - ELA France
Ingrid Callies, Chair of the Ethics Committee

Under the Working Group No. 6
Mikel Renteria, representing patients' association - ELA Spain
Gael de Miomandre, representing patients' association - ELA Belgium
Ella Haven, representing patients' association - association X-ALD, Netherlands
Silvano Vona, representing patients' association - ELA Italy
Catherine Schorderet, representative association of patients - ELA France