Descrizione del progetto
Un semplice trattamento orale per una malattia trascurata: la leishmaniosi cutanea
Molte malattie non potenzialmente letali nei paesi sviluppati, non ricevono le risorse e l’attenzione necessarie per studiarne la prevenzione, il trattamento o la cura. La leishmaniosi cutanea (LC), causata da un parassita trasmesso attraverso mosche di sabbia infette, è una di queste. Le infezioni sono comuni nelle popolazioni a basso reddito di regioni in via di sviluppo. Producendo ulcere, a volte fino a 200, sulle parti esposte del corpo, la LC può lasciare cicatrici permanenti che sono spesso fonte di estremi pregiudizi sociali. Il progetto TT4CL sta lavorando duramente per portare un promettente trattamento orale per la LC nelle sperimentazioni cliniche. La registrazione presso le agenzie di regolamentazione che richiedono una prova di conformità ai severi requisiti di sicurezza, efficacia e tollerabilità potrebbe cambiare la vita di milioni di persone in tutto il mondo.
Obiettivo
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.
Campo scientifico
- 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
Parole chiave
Programma(i)
Argomento(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-SC1-2018-Two-Stage-RTD
Meccanismo di finanziamento
RIA - Research and Innovation actionCoordinatore
SW17 0RE London
Regno Unito