EUROFANCOLEN
Phase I/II Gene Therapy Trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action
| Coordinatore | CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT
Organization address
address: Avenida Complutense 22 contact info |
| Nazionalità Coordinatore | Spain [ES] |
| Totale costo | 7˙002˙945 € |
| EC contributo | 5˙380˙169 € |
| Programma | FP7-HEALTH
Specific Programme "Cooperation": Health |
| Code Call | FP7-HEALTH-2012-INNOVATION-1 |
| Funding Scheme | CP-FP |
| Anno di inizio | 2013 |
| Periodo (anno-mese-giorno) | 2013-01-01 - 2017-12-31 |
Partecipanti
| # | ||||
|---|---|---|---|---|
| 1 |
CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT
Organization address
address: Avenida Complutense 22 contact info |
ES (MADRID) | coordinator | 689˙659.00 |
| 2 |
INNOVACION DESARROLLO Y TRANSFERENCIA DE TECNOLOGIA SA
Organization address
address: CALLE HERMOSILLA 48-3 IZQ contact info |
ES (Madrid) | participant | 1˙055˙227.00 |
| 3 |
GATC BIOTECH AG
Organization address
address: JAKOB STADLER PLATZ 7 contact info |
DE (KONSTANZ) | participant | 654˙192.00 |
| 4 |
ASSOCIATION GENETHON
Organization address
address: RUE DE L INTERNATIONALE 1 BIS contact info |
FR (EVRY) | participant | 630˙918.75 |
| 5 |
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS
Organization address
address: 3 Avenue Victoria contact info |
FR (PARIS) | participant | 498˙120.00 |
| 6 |
UNIVERSITY COLLEGE LONDON
Organization address
address: GOWER STREET contact info |
UK (LONDON) | participant | 493˙800.00 |
| 7 |
SERVICIO MADRILENO DE SALUD
Organization address
address: PLAZA CARLOS TRIAS BERTRAN 7 contact info |
ES (MADRID) | participant | 293˙760.00 |
| 8 |
DEUTSCHES KREBSFORSCHUNGSZENTRUM
Organization address
address: Im Neuenheimer Feld 280 contact info |
DE (HEIDELBERG) | participant | 271˙680.00 |
| 9 |
INSTITUT CATALA DE LA SALUT
Organization address
address: GRAN VIA DE LES CORTS CATALANES 587 contact info |
ES (BARCELONA) | participant | 268˙368.00 |
| 10 |
UNIVERSITE PARIS DIDEROT - PARIS 7
Organization address
address: RUE THOMAS MANN 5 contact info |
FR (PARIS) | participant | 264˙600.00 |
| 11 |
UNIVERSITAT AUTONOMA DE BARCELONA
Organization address
address: Campus UAB -BELLATERRA- s/n contact info |
ES (CERDANYOLA DEL VALLES) | participant | 259˙845.00 |
Mappa
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Obiettivo del progetto (Objective)
'Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age. Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased incidence of squamous cell carcinoma. The genetic correction of autologous hematopoietic stem cells (HSC) with lentiviral vectors constitutes a recent and safe alternative for the treatment of different genetic diseases affecting mature cells from different tissues and/or committed progenitors of the hematopoietic system. One of the key features of FA that make it a unique disease for gene therapy approaches rely on the characteristic proliferation defect that is already evident in the very primitive HSCs. Thus, a marked survival advantage would be expected from corrected HSCs, potentially allowing normalization of hematopoiesis in the absence or after mild conditioning. Difficulties in the collection of sufficient numbers of HSC from FA patients and the use of sub-optimal transduction protocols with gammaretroviral vectors limited the success of FA gene therapy trials conducted 10 years ago in the USA. Our innovative approach to develop for the first time an efficient and safe gene therapy of FA is based on two recent innovations: 1) Discovery of potent HSC mobilizers, such as plerixafor, and 2) Development of a new lentiviral vector by members of this Consortium, designed as Orphan Drug by the EC in December 2010. The principal objective of this Project is, therefore, the development of a multicentric Phase I/II gene therapy trial for FA-A patients, based on the genetic correction of plerixaforG-CSF mobilized HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring studies.'