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Therapeutic challenge in Leukodystrophies: Translational and ethical research towards clinical trials

Objective

Leukodystrophies (LDs) are inherited rare neurodegenerative diseases of the white matter and its main component, the myelin, that are affecting predominantly children. Severity of the disease is related to the axonal dysfunction due to myelin deficiency or destruction. Despite the achievement of remarkable advances made in the past decade, there is no current curative therapy. The development of therapeutic approaches for myelin repair and neuroprotection constitutes the main objective of the LeukoTreat project. Indeed LDs constitute prototypic pathologies to tackle myelin formation/destruction issues as well as glial cells dysfunctions in neurodegeneration. The global aim is to promote the development of therapeutic strategies for the largest number of LD affected patients and further applications to more common white matter disorders and finally neurodegenerative diseases. For this purpose, the project will combine the expertise of (i) recognized European research teams in the field of White Matter diseases (COST Myelinet), (ii) high-technology SMEs, (iii) experts in medical ethics and (iv) LD patients and families associations. To develop efficient therapies, the LeukoTreat project is based on 5 complementary approaches consisting in: (i) collecting information on the epidemiology, the natural history, the genotype/phenotype correlation of LDs for at least 500 patients; (ii) validating/identifying biomarkers for therapeutic decisions/follow up to isolate new therapeutic targets; (iii) developing pharmacological strategies with the ultimate objective to launch at least 4 pharmacological clinical trials during 5 years following the project; (iv) developing innovative gene and cell therapies with the ultimate objective to launch at least 3 clinical trials during the next 5 years; (v) tackling ethical impacts of the proposed therapeutic challenges by integrating the participation of patients driven by a well-experienced research team strongly skilled in ethics
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Coordinator

UNIVERSITE D'AUVERGNE CLERMONT-FERRAND 1

Address

Boulevard Francois Mitterand 49
63001 Clermont Ferrand

France

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 796 258,60

Administrative Contact

Isabelle Rhit

Participants (24)

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OSPEDALE PEDIATRICO BAMBINO GESU

Italy

EU Contribution

€ 84 612

ISTITUTO GIANNINA GASLINI

Italy

EU Contribution

€ 100 300

UNIVERSITATSKLINIKUM BONN

Germany

EU Contribution

€ 352 480

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 671 624

Soluscience SA

France

EU Contribution

€ 300 800

THE UNIVERSITY OF MANCHESTER

United Kingdom

EU Contribution

€ 359 380

FUNDACIO INSTITUT D'INVESTIGACIO BIOMEDICA DE BELLVITGE

Spain

EU Contribution

€ 376 282,01

MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV

Germany

EU Contribution

€ 401 758

MEDIZINISCHE UNIVERSITAET WIEN

Austria

EU Contribution

€ 337 888

TROPHOS SA

France

EU Contribution

€ 84 714,80

UNIVERSITY COLLEGE LONDON

United Kingdom

EU Contribution

€ 93 825,76

Fondazione Centro San Raffaele

Italy

EU Contribution

€ 388 600

ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM

Netherlands

EU Contribution

€ 254 800

UNIVERSITE PARIS DESCARTES

France

EU Contribution

€ 237 840

FRANCE EUROPE INNOVATION

France

EU Contribution

€ 277 119

ASSOCIATION EUROPEENNE CONTRE LES LEUCODYSTROPHIES

France

EU Contribution

€ 51 164,80

THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE

United Kingdom

EU Contribution

€ 89 886

UNIVERSITE PARIS DIDEROT - PARIS 7

France

EU Contribution

€ 107 304

FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA

Italy

EU Contribution

€ 52 361,91

UNIVERSITAETSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAET MAINZ

Germany

UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF

Germany

EU Contribution

€ 112 920,34

EBERHARD KARLS UNIVERSITAET TUEBINGEN

Germany

EU Contribution

€ 215 519,78

UNIVERSITY OF NEW SOUTH WALES

Australia

EU Contribution

€ 207 287

FONDAZIONE CENTRO SAN RAFFAELE

Italy

EU Contribution

€ 23 400

Project information

Grant agreement ID: 241622

Status

Closed project

  • Start date

    1 March 2010

  • End date

    31 August 2013

Funded under:

FP7-HEALTH

  • Overall budget:

    € 9 249 281,30

  • EU contribution

    € 5 978 126

Coordinated by:

UNIVERSITE D'AUVERGNE CLERMONT-FERRAND 1

France

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