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Treat Iron-Related Childhood-Onset Neurodegeneration

Objective

Neurodegeneration with brain iron accumulation (NBIA) is a heterogeneous group of rare hereditary neurodegenerative disorders characterized by high levels of brain iron. The most common form is pantothenate kinase-associated neurodegeneration (PKAN). Classic PKAN and most other NBIA cases are characterised by early childhood onset and rapid progression. Currently, there is no proven therapy to halt or reverse PKAN or any other NBIA. This is especially unfortunate as both the iron accumulation in NBIA and the biochemical defect in PKAN are predicted to be amenable to drug-based treatment. Thus, the current absence of clinical trials is not due to lack of therapeutic options but to rarity of the disease, lack of patient registries and fragmentation of therapeutic research worldwide. For example, the iron-chelating drug deferiprone has been administered to PKAN patients on an individual basis or in pilot trials, both precluding firm conclusions about its efficacy. With TIRCON, we will address this urgent and unmet need for NBIA/PKAN therapy with an ambitious and highly collaborative plan that leverages worldwide expertise. We propose a large investigator-driven randomized clinical trial of deferiprone in PKAN, bringing together leading centres and patient advocacy groups from Europe and the US to reach the required patient cohort size. In addition, together with a European SME, we propose to pursue preclinical development of pantethine and its derivatives which have shown promising efficacy in a Drosophila PKAN model. To facilitate future research, we will develop a harmonized patient registry and biomaterial bank to allow for natural history studies and biomarker development, two critical needs in NBIA research. TIRCON partners, apart from their unique clinical and basic science expertise in NBIA, have longstanding experience in investigator-driven and industry-driven randomized clinical trials. Importantly, they have been closely collaborating in recent years.

Field of science

  • /natural sciences/chemical sciences/inorganic chemistry/inorganic compounds

Call for proposal

FP7-HEALTH-2011-two-stage
See other projects for this call

Funding Scheme

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

Coordinator

LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Address
Geschwister Scholl Platz 1
80539 Muenchen
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 346 708,42
Administrative Contact
Thomas Klopstock (Prof.)

Participants (12)

KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITAT MUNCHEN
Germany
EU contribution
€ 844 340
Address
Ismaninger Strasse 22
81675 Muenchen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Renate Raedlein (Ms.)
INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA
Poland
EU contribution
€ 77 600,40
Address
Aleja Dzieci Polskich 20
04730 Warszawa
Activity type
Research Organisations
Administrative Contact
Tomasz Kmiec (Dr.)
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
Italy
EU contribution
€ 403 432,95
Address
Via Giovanni Celoria 11
20133 Milano
Activity type
Research Organisations
Administrative Contact
Francesca Grechi (Dr.)
ACADEMISCH ZIEKENHUIS GRONINGEN
Netherlands
EU contribution
€ 387 000
Address
Hanzeplein 1
9713 GZ Groningen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Johanna M Van Apeldoorn (Ms.)
OREGON HEALTH AND SCIENCE UNIVERSITY
United States
EU contribution
€ 281 700
Address
3181 Sw Sam Jackson Park Road
97239-3098 Portland, Oregon
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Susan Hayflick (Prof.)
CHILDREN'S HOSPITAL & RESEARCH CENTER AT OAKLAND
United States
EU contribution
€ 248 388,85
Address
52Nd Street 747
94609 Oakland Ca
Activity type
Research Organisations
Administrative Contact
Rebecca Rosales (Ms.)
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
EU contribution
€ 244 094,92
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Amanda Gregory (Dr.)
ACIES BIO BIOTEHNOLOSKE RAZISKAVE IN RAZVOJ DOO
Slovenia
EU contribution
€ 394 000
Address
Tehnoloski Park 21
1000 Ljubljana
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Enej Kuscer (Dr.)
NBIA DISORDERS ASSOCIATION
United States
EU contribution
€ 114 720
Address
Monaco Court 2082
92019-4235 El Cajon Ca
Activity type
Other
Administrative Contact
Patricia Wood (Ms.)
HOFFNUNGSBAUM EV VEREIN ZUR FORDERUNG DER ERFORSCHUNG UND BEHANDLUNG VON NBIA (VORMALS: HALLERVORDEN-SPATZ-SYNDROM)*TREE OF HOPE EV
Germany
EU contribution
€ 153 600
Address
Hardenberger Strasse 73
42549 Velbert
Activity type
Other
Administrative Contact
Angelika Klucken (Mrs.)
BAYERISCHE FORSCHUNGSALLIANZ BAVARIAN RESEARCH ALLIANCE GMBH
Germany
EU contribution
€ 246 251,60
Address
Prinzregentenstrasse 52
80538 Munchen
Activity type
Other
Administrative Contact
Florence Gauzy (Dr.)
APO-PHARMA INC CORP
Canada
EU contribution
€ 473 382,66
Address
Signet Drive 150
M9L 1T9 Toronto
Activity type
Other
Administrative Contact
Michael Spino (Dr.)