Descrizione del progetto
Trattamento della malattia del trapianto contro l’ospite acuta mediante l’uso di cellule stromali mesenchimali
Le terapie rigenerative basate sulle cellule staminali hanno un grande potenziale per il trattamento di molte patologie. La rigenerazione si basa su cellule staminali specifiche del tessuto o progenitrici. Le cellule stromali mesenchimali (MSC) hanno la capacità di formare direttamente tessuti ossei o cartilaginei. Inoltre, la riparazione endogena indiretta attraverso le proprietà antinfiammatorie delle MSC è importante nel trattamento della malattia del trapianto contro l’ospite acuta. Il progetto RETHRIM, finanziato dall’UE, intende condurre il primo studio di fase III randomizzato e controllato con placebo per la terapia a base di MSC nel trattamento della malattia del trapianto contro l’ospite viscerale resistente agli steroidi. Il consorzio propone l’arruolamento di 150 pazienti per lo studio, al fine di analizzare le MSC utilizzando marcatori molecolari e funzionali per la previsione della risposta terapeutica.
Obiettivo
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.
Campo scientifico
- 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
Programma(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-PHC-2014-single-stage
Meccanismo di finanziamento
RIA - Research and Innovation actionCoordinatore
2333 ZA Leiden
Paesi Bassi