Objetivo
CARAMBA is proposing to use the revolutionary CAR-T cell technology to tackle multiple myeloma, a rare and incurable hematologic malignancy. Myeloma is associated with a substantial socioeconomic burden and there is a strong demand by patients, their families and caregivers for a curative myeloma treatment. The CARAMBA investigators will conduct a phase I/II clinical trial of immunotherapy with patient-derived T cells that are engineered to express a synthetic chimeric antigen receptor (CAR) specific for the myeloma antigen SLAMF7. SLAMF7 is uniformly expressed on all myeloma cells in every patient, and SLAMF7 CAR-T cells will be equally effective in women and men. The efficacy and safety of SLAMF7 CAR-T cells has been validated by the coordinator in comprehensive pre-clinical testing which highlighted their unprecedented curative potential. SLAMF7 CAR-T cells have received an orphan designation for the indication multiple myeloma by the EC. The successful conduct, rapid and complete recruitment into the clinical trial is ensured by participation of 4 clinical myeloma centres of excellence in 4 EU member states, and the largest myeloma patient advocacy group in Europe, MPE. The SME partners in CARAMBA are prepared to develop the SLAMF7 CAR product all the way to market authorization. CAR-T cell therapy has been recognized as a ‘Breakthrough Therapy’ and clinical proof-of-concept obtained with CD19 CAR-T cells in leukemia and lymphoma. CARAMBA is building on novel and proprietary, cutting-edge CAR technologies including virus-free Sleeping Beauty CAR gene-transfer from DNA minicircles which substantially reduces the cost of CAR-T cell manufacturing, making it an economically viable and sustainable medicinal product. We are confident, that if funded, CARAMBA will accomplish its objective to establish the role of CAR-T cell therapy as a therapeutically effective, commercially attractive and socioeconomically desirable treatment in myeloma and other rare diseases.
Ámbito científico
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- medical and health sciencesbasic medicineimmunologyimmunotherapy
- medical and health sciencesclinical medicineoncologyleukemia
- medical and health sciencesmedical biotechnologycells technologies
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2017-Two-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
97080 Wurzburg
Alemania