Description du projet
Un traitement oral simple contre une maladie négligée, la leishmaniose cutanée
Dans les pays développés, de nombreuses maladies non mortelles ne reçoivent pas les ressources et l’attention nécessaires pour déclencher des recherches sur leur prévention, leur traitement ou leur guérison. La leishmaniose cutanée (LC), causée par un parasite transmis par des phlébotomes infectés, en fait partie. Les infections sont courantes dans les populations à faible revenu des régions en développement. Produisant des ulcères, parfois jusqu’à 200, sur les parties exposées du corps, la LC peut laisser des cicatrices permanentes qui sont souvent la source de préjugés sociaux extrêmes. Le projet TT4CL s’efforce d’apporter un essai prometteur de traitement par voie orale contre la LC. L’enregistrement auprès d’organismes de réglementation exigeant une preuve de conformité aux exigences strictes en matière d’innocuité, d’efficacité et de tolérabilité pourrait changer la vie de millions de personnes dans le monde.
Objectif
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.
Champ scientifique
- medical and health scienceshealth sciencestropical medicine
- natural sciencesearth and related environmental sciencesatmospheric sciencesclimatologyclimatic zones
- medical and health sciencesclinical medicineallergologydrug allergy
- medical and health sciencesbasic medicinepharmacology and pharmacypharmacokinetics
- social sciencespolitical sciencesgovernment systems
Mots‑clés
Programme(s)
Régime de financement
RIA - Research and Innovation actionCoordinateur
SW17 0RE London
Royaume-Uni