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Clinical development of Enzyme Replacement Therapy in alpha-Mannosidosis patients using recombinant human enzyme

Objective

Alpha-Mannosidosis is a rare Lysosomal Storage Disorder with a worldwide incidence of about 1:500 000. This orphan and devastating disease is caused by the deficiency of the lysosomal alpha-mannosidase (LAMAN) which is responsible for the intralysosomal degradation of mannosyl linked oligosaccharides. To date no causative treatment for alpha-Mannosidosis is available and since most of the children are born healthy, an early initiated therapy could contribute to a normal development. To develop an efficient therapy for this disease the collaborative research project EURAMAN and HUE-MAN were initiated within FP5 and FP6, respectively. Within these collaborative networks, European scientists, clinicians and the industry successfully i) developed an efficient pre-clinical Enzyme Replacement Therapy (ERT) protocol using recombinant human (rh) LAMAN in a mouse model for alpha-Mannosidosis, ii) built up a database collecting patient data and iii) defined clinical endpoints for the future clinical trials in alpha-Mannosidosis patients by an European wide natural history study. Furthermore, the HUE-MAN network developed the conditions for a large-scale production of the recombinant enzyme and the way is now paved for the first clinical trials in man, which we aim to realize within FP7. The main objectives of the ALPHA-MAN project are i) the performance of efficient clinical trials (phase I-III) in alpha-Mannosidosis patients, ii) a better understanding of the pathophysiology and the mechanism of how the recombinant enzyme enters the cells of the central nervous system by performing ERT in a newly generated immuntolerant alpha-Mannosidosis mouse model, which allows chronic treatment and iii) the determination of the minimal effective dose with chronic treatment in the immuntolerant mice.

Call for proposal

FP7-HEALTH-2010-single-stage
See other projects for this call

Funding Scheme

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

Coordinator

CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL
Address
Olshausenstrasse 40
24118 Kiel
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 593 128
Administrative Contact
Paul Saftig (Prof.)

Participants (11)

Zymenex AS
Denmark
EU contribution
€ 329 732
Address
Roskildevej 12C
3400 Hillerød
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Jens Fogh (Dr.)
REGION HOVEDSTADEN
Denmark
EU contribution
€ 2 826 623
Address
Kongens Vaenge 2
3400 Hillerod
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Allan Meldgaard Lund (Dr.)
KATHOLIEKE UNIVERSITEIT LEUVEN
Belgium
EU contribution
€ 299 360
Address
Oude Markt 13
3000 Leuven
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Elke Lammertyn (Dr.)
THE UNIVERSITY OF MANCHESTER
United Kingdom
EU contribution
€ 37 282
Address
Oxford Road
M13 9PL Manchester
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Liz Fay (Ms.)
UNIVERSITAETSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAET MAINZ
Germany
EU contribution
€ 589 434
Address
Langenbeckstrasse 1
55131 Mainz
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Silvia Tschauder (Dr.)
HOSPICES CIVILS DE LYON
France
EU contribution
€ 255 561
Address
Quai Des Celestins 3
69002 Lyon
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Cecile Legros (Ms.)
UNIVERSITETET I TROMSOE - NORGES ARKTISKE UNIVERSITET
Norway
EU contribution
€ 97 400
Address
Hansine Hansens Veg 14
9019 Tromso
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Stig Orsje (Mr.)
TEKNOLOGISK INSTITUT
Denmark
EU contribution
€ 187 636
Address
Gregersensvej 1
2630 Taastrup
Activity type
Research Organisations
Administrative Contact
Anne Maria Hansen (Ms.)
INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA
Poland
EU contribution
€ 11 172
Address
Aleja Dzieci Polskich 20
04730 Warszawa
Activity type
Research Organisations
Administrative Contact
Anna Tylki-Szymańska (Prof.)
LARIX APS
Denmark
EU contribution
€ 543 958
Address
Tempovej 44 - 1St Floor
2750 Ballerup
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Klaus Juel Olsen (Dr.)
UNILABAS AS
Denmark
EU contribution
€ 115 864
Address
Nygaardsvej 32
2100 Copenhagen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Persson Poul (Dr.)