Objetivo
Primary immune deficiencies (PID) are inherited disorders of the adaptive and innate immune system marked by severe infections, autoimmunity and high risk of cancer. Treatment entails hematopoietic stem cell (HSC) transplantation from allogeneic donors, however in the absence of an HLA compatible donor, HSCT outcome is limited by delayed or suboptimal reconstitution and complications. SCID-X1 and ADA-SCID have been successfully treated with autologous gene corrected HSC, however, associated with safety issues inherent to first generation retroviral vectors. This project utilizes genetically modified HSC and their descendants as immunotherapeutic cells to build a healthy immune system in PID patients, and is carried out by clinical centres, scientists and industrial partners pioneering in the field of advanced therapies and aiming at broad clinical application of safe cell-based therapeutic products. Multicentre phase I/II clinical trials for SCID-X1 and WAS are ready to start. Disease targeted technology to cure ADA-SCID, V(D)J recombination defects and CGD by gene corrected HSC and novel approaches in IPEX and HLH to gene modify already committed cells will be investigated. Based on rigorous preclinical efficacy and toxicology evaluation, flanked by basic studies aimed at improving HSC homing capacity and thymic epithelium regeneration, new clinical trials will be implemented. The consortium will establish a technology platform to implement, harmonize and run controlled, standardized multicentre preclinical studies using state-of-the-art advanced therapy. Strict observance of good practice quality guidelines and regulation of medicinal product development will be ensured. The successful completion of the project will be instrumental to accomplish and broaden clinical application of medicinal products able to rebuild and modulate the immune system with an anticipated impact that extends beyond PID to acquired immune disorders, allogeneic HSCT and cancer treatment.
Ámbito científico
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- medical and health sciencesbasic medicineimmunology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- medical and health sciencesclinical medicineoncology
- medical and health sciencesbasic medicinetoxicology
Tema(s)
Convocatoria de propuestas
FP7-HEALTH-2010-single-stage
Consulte otros proyectos de esta convocatoria
Régimen de financiación
CP-IP - Large-scale integrating projectCoordinador
20132 Milano
Italia
Ver en el mapa
Participantes (19)
75654 Paris
Ver en el mapa
WC1E 6BT London
Ver en el mapa
60596 Frankfurt
Ver en el mapa
7610001 Rehovot
Ver en el mapa
01069 Dresden
Ver en el mapa
La participación finalizó
3015 GD Rotterdam
Ver en el mapa
00165 Roma
Ver en el mapa
30625 Hannover
Ver en el mapa
20091 BRESSO MI
Ver en el mapa
51429 Bergish Gladbach
Ver en el mapa
4051 Basel
Ver en el mapa
78467 Konstanz
Ver en el mapa
69003 Lyon
Ver en el mapa
55743 IDAR OBERSTEIN
Ver en el mapa
06800 Cankaya Ankara
Ver en el mapa
8006 Zurich
Ver en el mapa
2333 AA Leiden
Ver en el mapa
3360 BIERBEEK
Ver en el mapa
20132 Milano
Ver en el mapa