Projektbeschreibung
Akute Transplantat-gegen-Wirt-Reaktion mit mesenchymalen Stromazellen behandeln
Regenerative Therapien auf Stammzellenbasis bergen viel Potenzial für die Behandlung einer Vielzahl von Krankheiten. Die Regeneration beruht auf gewebespezifischen Stammzellen oder Vorläuferzellen. Mesenchymale Stromazellen verfügen über die Fähigkeit, direkt Knochen- oder Knorpelgewebe zu bilden. Zudem ist die indirekte endogene Reparatur mithilfe der entzündungshemmenden Eigenschaften mesenchymaler Stromazellen wichtig für die Behandlung der akuten Transplantat-gegen-Wirt-Reaktion. Das Ziel des EU-finanzierten Projekts RETHRIM besteht darin, die erste placebokontrollierte, randomisierte Phase-III-Studie für eine Therapie auf der Grundlage von mesenchymalen Stromazellen zur Behandlung von steroidresistenter viszeraler Transplantat-gegen-Wirt-Reaktion durchzuführen. Das Konsortium schlägt vor, 150 Patientinnen und Patienten für die Studie zu rekrutieren, um mesenchymale Stromazellen anhand von molekularen und funktionellen Markern zu analysieren, um das Ansprechen auf die Therapie vorherzusagen.
Ziel
Stem cell regenerative therapies hold great promise for patients suffering from a variety of disorders that are associated with tissue or organ injury. Regeneration relies on tissue or organ-specific stem and progenitor cells, but can also aim at promoting the endogenous repair capacity of the body.
Mesenchymal stromal cells (MSC) are undergoing clinical testing in a variety of clinical conditions aiming at repair through direct or indirect mechanisms. Their ability to form bone or cartilage is used to directly repair these tissues. In other conditions their regenerative effects are based on endogenous repair through their anti-inflammatory properties. The latter mechanism is important in the treatment of acute Graft-versus-Host Disease (GvHD). We have been involved in the clinical development from the beginning and we have shown the therapeutic potential. However, no results of controlled randomized phase 3 studies have been published to date, thereby hampering safety and efficacy assessment.
Within our consortium we have developed an academic infrastructure for the harmonized production of MSC. In the RETHRIM proposal this will be combined with our clinical expertise to conduct the first Europe-wide placebo controlled randomized phase III trial using MSC regenerative therapy for the treatment of steroid-resistant visceral GvHD. Central to the RETHRIM project is the clinical trial for which 150 patients will be recruited. All MSC products will be extensively analysed using molecular and functional markers, in order to develop a potency signature for the product and for the prediction of response. We also intend to collect data from additional quality of life, health technology assessment and ethical studies. We will apply for an Orphan Drug Designation in Europe and this may serve as a stepping-stone for the further commercialization of the MSC product, once a positive outcome has been obtained.
Wissenschaftliches Gebiet
- social sciencessociologydemographymortality
- natural sciencesbiological sciencesbiochemistrybiomoleculesproteins
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- medical and health sciencesclinical medicineemergency medicinegraft versus host disease
- medical and health sciencesclinical medicinetransplantation
Programm/Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenUnterauftrag
H2020-PHC-2014-single-stage
Finanzierungsplan
RIA - Research and Innovation actionKoordinator
2333 ZA Leiden
Niederlande