Descripción del proyecto
Un simple tratamiento oral para la enfermedad olvidada de la leishmaniasis cutánea
Muchas enfermedades potencialmente no mortales de países desarrollados no reciben los recursos ni la atención necesarios para investigar su prevención, tratamiento o cura. La leishmaniasis cutánea (LC), causada por un parásito que se transmite a través de moscas de la arena infectadas, es una de ellas. Las infecciones son comunes en poblaciones con bajos ingresos de regiones en desarrollo. La LC, que provoca úlceras, a veces hasta doscientas, en partes expuestas del cuerpo, puede dejar cicatrices permanentes que a menudo son fuente de prejuicios sociales extremos. TT4CL está trabajando duro para llevar un tratamiento oral prometedor para la LC a la fase de ensayos clínicos. Su registro ante los organismos reguladores que exigen pruebas de conformidad con requisitos exigentes de seguridad, eficacia y tolerabilidad podría cambiar las vidas de millones de personas en todo el mundo.
Objetivo
Cutaneous leishmaniasis (CL) is a poverty related, neglected tropical disease, which is without an effective and cheap systemic treatment. The aim of TT4CL is to develop a new orally available drug for treatment of CL aimed towards registration with stringent regulatory authorities. Oleylphosphocholine (OlPC), regarded as a new drug by the FDA, is structurally related to the anti-leishmanial miltefosine. It is being developed as an immediate-release tablet for the treatment of CL and, currently, is the only systemically delivered drug specifically being developed for this indication. OlPC is active in vitro and in vivo against different CL-causing Leishmania parasite species and shows curative advantage over miltefosine in rodent models of leishmaniasis. The primary objective of this study is to complete the pre-clinical package that is essential for the subsequent clinical development of OlPC. The project will aim to optimize the synthesis and formulation of OlPC, including stability testing that is appropriate for tropical climates. It will include in vitro drug sensitivity analyses in parasites causing CL (Leishmania tropica and L. major) in the Islamic Republic of Iran, with our endemic-country partner. Comparative studies in animal models with existing anti-leishmanial compounds will establish efficacy advantages and determine pharmacokinetic-pharmacodynamic relationships for OlPC. Phase 1 studies will confirm tolerability and pharmacokinetics of single doses and multiple dosing regimens. Results will be used to guide decisions by future partners on the clinical development of OlPC. This proposal directly addresses the priorities highlighted in this H2020 call. To our knowledge, we are the only consortium that is implementing this type of approach, and there is no other interest in the pharmaceutical sector to carry out a development programme for the oral treatment of CL.
Ámbito científico
Not validated
Not validated
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2018-Two-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
SW17 0RE London
Reino Unido