Objectif
Type 1 diabetes is a serious chronic disease with major health risks and heavy burden on patients and society. It is caused by massive immune-mediated loss of insulin-producing beta cells in the pancreas that can so far not be locally corrected. A cellular allotransplant in the liver can install a new beta cell mass but the size is insufficient and the procedure faces limitations of donor shortage, inaccessibility of the implants, risks of associated immunosuppression. Our consortium of research, clinical and bioindustry teams is focused on overcoming these obstacles and implementing a roadmap for translation to preclinical models and clinical trials. We will pursue three interacting tracks. First, our ability to induce beta cell progenitors and stimulate beta cell proliferation in vivo should lead us to cells and compounds that activate this process in a diabetic pancreas, thus activating endogenous beta cell regeneration. Second, we will produce human beta (progenitor) cells in vitro by derivation from stem cells as well as from reprogrammed autologous cells; their therapeutic potential will be compared to that of primary human beta cells following implantation in rodents using a site that is accessible to modulation and monitoring. Third, we will design an antibody-based therapy for inducing immune tolerance to regenerated beta cells and to a beta cell implant. Efficacy, safety and regulatory criteria will be determined for clinical implementation. Clinical protocols will be prepared by adjusting associated therapy and by adopting an accessible and controlled implant site. Clinical trials will benefit from state-of-the art biologic markers for comparative analysis of the developed forms of beta cell therapy. This program should provide proof of principle for strategies that make beta cell transplantation and beta cell regeneration realistic for large numbers of type 1 diabetic patients, and probably also for some categories of type 2 diabetes.
Champ scientifique
Appel à propositions
FP7-HEALTH-2009-single-stage
Voir d’autres projets de cet appel
Régime de financement
CP-IP - Large-scale integrating projectCoordinateur
1050 Bruxelles / Brussel
Belgique
Voir sur la carte
Participants (17)
CB2 1TN Cambridge
Voir sur la carte
OX1 2JD Oxford
Voir sur la carte
2333 ZA Leiden
Voir sur la carte
75654 Paris
Voir sur la carte
59000 LILLE
Voir sur la carte
91904 Jerusalem
Voir sur la carte
1015 Lausanne
Voir sur la carte
3000 Leuven
Voir sur la carte
20132 MILANO
Voir sur la carte
3400 Hillerod
Voir sur la carte
75794 Paris
Voir sur la carte
1731 ZELLIK
Voir sur la carte
75007 Paris
Voir sur la carte
1090 Bruxelles / Brussel
Voir sur la carte
7491 Trondheim
Voir sur la carte
92121 San Diego Ca
Voir sur la carte
20132 Milano
Voir sur la carte