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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

Field of science

  • /medical and health sciences/health sciences/medical ethics
  • /medical and health sciences/basic medicine/neurology

Call for proposal

FP7-HEALTH-2009-single-stage
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

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)

OSPEDALE PEDIATRICO BAMBINO GESU
Italy
EU contribution
€ 84 612
Address
Piazza Sant Onofrio 4
00165 Roma
Activity type
Other
Administrative Contact
Enrico Bertini (Prof.)
ISTITUTO GIANNINA GASLINI
Italy
EU contribution
€ 100 300
Address
Via Gerolamo Gaslini 5
16147 Genova
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Maria Gabriella Marinari
UNIVERSITATSKLINIKUM BONN
Germany
EU contribution
€ 352 480
Address
Venusberg-campus 1
53127 Bonn
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Beate Gieselmann (Ms.)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 671 624
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
Administrative Contact
Patrick Aubourg (Mr.)
Soluscience SA
France
EU contribution
€ 300 800
Address
Biopole Clermont-limagne
63360 Saint-beauzire
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Yann Dantal (Mr.)
THE UNIVERSITY OF MANCHESTER
United Kingdom
EU contribution
€ 359 380
Address
Oxford Road
M13 9PL Manchester
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Liz Fay (Ms.)
FUNDACIO INSTITUT D'INVESTIGACIO BIOMEDICA DE BELLVITGE
Spain
EU contribution
€ 376 282,01
Address
Avenida Gran Via Hospitalet 199-203
08908 L'hospitalet De Llobregat
Activity type
Research Organisations
Administrative Contact
Victoria Cochrane (Ms.)
MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV
Germany
EU contribution
€ 401 758
Address
Hofgartenstrasse 8
80539 Munich
Activity type
Other
Administrative Contact
Klaus-Armin Nave (Prof.)
MEDIZINISCHE UNIVERSITAET WIEN
Austria
EU contribution
€ 337 888
Address
Spitalgasse 23
1090 Wien
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Johannes Berger (Prof.)
TROPHOS SA
France
EU contribution
€ 84 714,80
Address
Parc Scientifique Luminy Case 931
13288 Marseille Cedex 9
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Julien Veys (Mr.)
UCL Elizabeth Garrett Anderson Institute for Women’s Health
United Kingdom
EU contribution
€ 93 825,76
Address
Gower Street
WC1E 6BT London
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Greta Borg-Carbott (Ms.)
FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR
Italy
EU contribution
€ 388 600
Address
Via Olgettina 60
20132 Milano
Activity type
Research Organisations
Administrative Contact
Mario Valsecchi (Mr.)
ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM
Netherlands
EU contribution
€ 254 800
Address
Meibergdreef 15
1105AZ Amsterdam
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ronald Wanders (Prof.)
UNIVERSITE PARIS DESCARTES
France
EU contribution
€ 237 840
Address
Rue De L Ecole De Medecine 12
75270 Paris Cedex 06
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Grégoire Moutel (Dr.)
FRANCE EUROPE INNOVATION
France
EU contribution
€ 277 119
Address
Rue Des Renaudes 32
75017 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Olivier Degrand (Mr.)
ASSOCIATION EUROPEENNE CONTRE LES LEUCODYSTROPHIES
France
EU contribution
€ 51 164,80
Address
Rue Mi-les-vignes 2
54520 Laxou
Activity type
Other
Administrative Contact
Guy Alba (Mr.)
THE CHANCELLOR MASTERS AND SCHOLARSOF THE UNIVERSITY OF CAMBRIDGE
United Kingdom
EU contribution
€ 89 886
Address
Trinity Lane The Old Schools
CB2 1TN Cambridge
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Keith Cann (Mr.)
UNIVERSITE PARIS DIDEROT - PARIS 7
France
EU contribution
€ 107 304
Address
Rue Thomas Mann 5
75205 Paris
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Muriel Maurice (Ms.)
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
Italy
EU contribution
€ 52 361,91
Address
Via Giovanni Celoria 11
20133 Milano
Activity type
Research Organisations
Administrative Contact
Francesca Grechi (Dr.)
UNIVERSITAETSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAET MAINZ

Participation ended

Germany
Address
Langenbeckstrasse 1
55131 Mainz
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Matthias Klugmann (Dr.)
UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF
Germany
EU contribution
€ 112 920,34
Address
Martinistrasse 52
20251 Hamburg
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Alfried Kohlschütter (Prof.)
EBERHARD KARLS UNIVERSITAET TUEBINGEN
Germany
EU contribution
€ 215 519,78
Address
Geschwister-scholl-platz
72074 Tuebingen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ingeborg Krägeloh-Mann (Prof.)
UNIVERSITY OF NEW SOUTH WALES
Australia
EU contribution
€ 207 287
Address
Anzac Parade
2052 Sydney
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Deirdre Mckay (Ms.)
FONDAZIONE CENTRO SAN RAFFAELE
Italy
EU contribution
€ 23 400
Address
Via Olgettina 60
20132 Milano
Activity type
Research Organisations
Administrative Contact
Maria Rosa Pedrazzi (Dr.)