Descripción del proyecto
Un novedoso enfoque de terapia génica para el linfedema
El linfedema es una afección discapacitante consistente en la acumulación localizada de líquido en un tejido a causa de problemas en el sistema linfático. Se trata de una enfermedad incurable que afecta a millones de personas en todo el mundo y suele ser consecuencia del tratamiento contra el cáncer. El principal objetivo del proyecto TheraLymph, financiado con fondos europeos, es desarrollar un tratamiento para aliviar esta afección mediante un enfoque de terapia génica no integrador. El programa de investigación traslacional del consorcio del proyecto reúne a científicos y médicos de cinco países europeos y se centrará en pacientes afectados por linfedema tras un cáncer de mama. La investigación determinará los factores de riesgo del linfedema y los principales factores moleculares que influyen en la disfunción linfática. El objetivo del proyecto es validar la mejor combinación molecular para la terapia génica y finalizar ensayos clínicos de fase I/II en el hospital afiliado.
Objetivo
Lymphedema is a disabling condition induced by the accumulation of fluid and fat in the arm or in the leg. It is an untreatable disease that affects 4 millions people in Europe and more than 120 millions people worldwide. It is handicapping, painful and impacts substantially the quality of life. In western countries, lymphedema is generally a consequence of cancer treatments i.e. ten to fifteen percent of women will develop lymphedema after breast cancer. The main objective of Theralymph will be to establish a non-integrative gene therapy for this unmet medical need.
The theralymph translational research program brings together bench scientists from 5 European countries and physicians from the hosted Rangueil hospital in which the PI institute is located to perform a Phase I/II trial focusing on women who developed lymphedema after breast cancer.
Based on decades of disappointing results of monotherapy-gene delivery in cardiovascular diseases, our approach will be based on multiple gene therapy targeting both superficial lymphatic endothelium and deeper lymphatic collectors. We will identify molecules that possess a synergistic effect with the established lymphangiogenic factor VEGFC.
Theralymph project will determine risk factors for lymphedema and cartography the lymphatic network in the pathology. It will decipher whether lymphatic intrinsic molecules or microenvironmental peptides or lipids are modified in the lymphedematous arm to promote the lymphatic dysfunction. Theralymph will validate the best molecule combination that restores the lymphatic drainage in in preclinical lymphedema models before human study.
The Phase I/II gene therapy clinical trial will be performed in the vascular medicine department of Toulouse’s hospital, where the PI laboratory is located. This trial will use an innovative technology based on recently developed non-integrative lentiflash lentiviral vectors that allow a transient and highly efficient in vivo gene delivery.
Ámbito científico
- medical and health sciencesclinical medicineangiologyvascular diseases
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesclinical medicineoncologybreast cancer
- medical and health sciencesclinical medicinecardiologycardiovascular diseases
- medical and health sciencesbasic medicinepathology
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2019-Single-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
75654 Paris
Francia