Therapeutic strategies for leukodystrophy affected patients - LEUKOTREAT project

Management Overview

 The LeukoTreat project was coordinated by Prof. Odile Boespflug-Tanguy from Université Paris Diderot - Paris 7 (UPD-P7, France), with the assistance of Estelle Tournier, from Université d'Auvergne (UDA, France), Olivier Degrand and Delphine Chatard, from France Europe Innovation (FEI, France).

During the project, Prof. Boespflug-Tanguy also benefited from the support of two specific Committees:

• The LeukoDB Committee (LeukoDataBase & Biobank Committee), coordinated by Enrico Bertini (OPBG, Italy), particularly responsible for the validation, use and management of clinical data, biological samples and mutations collected in LeukoTreat,
• The LeukoReC Committee, coordinated by Odile Boespflug-Tanguy and responsible for the coordination of the LeukoReC centers (France, Germany, Italy), which task was to collect clinical data, biological samples and mutations in their defined geographical area.

Finally, the Ethics Committee, chaired by LEM (France), was composed of persons independent of the scientific programme of LeukoTreat, including European experts in medical ethics, Human Sciences and Law professionals, and representatives of patients’ associations named by ELA.

Programme Overview

The LeukoTreat project aimed at promoting the development of therapeutic strategies for the largest number of leukodystrophy (LD) affected patients and further applications to more common white matter (WM) disorders and neurodegenerative diseases. To reach these objectives, LeukoTreat pursued the following actions:

• Collection of information on the epidemiology, the natural history, the genotype / phenotype correlation of LDs for at least 500 patients,
• Validation / Identification of biomarkers for therapeutic decisions / follow-up to isolate new therapeutic targets,
• Development of pharmacological strategies with the objective to launch at least 4 pharmacological clinical trials during the 5 years following the project,
• Development of innovative gene and cell therapies with the objective to launch at least 3 clinical trials during the next 5 years following the project,
• Tackling of ethical impacts of the proposed therapeutic challenges.

For a presentation of each of these research axes, see the section Videos.