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Clinical development of oral oleylphosphocholine as a new drug for the treatment of Old World Cutaneous Leishmaniasis

Project description

A simple oral treatment for the neglected disease cutaneous leishmaniasis

Many diseases that are not life-threatening in developed countries, do not receive the resources and attention required to investigate prevention, treatment or cure. Cutaneous leishmaniasis (CL), caused by a parasite transmitted via infected sand flies, is one among them. Infections are common in low-income populations of developing regions. Producing ulcers, sometimes up to 200, on exposed parts of the body, CL can leave permanent scars that are often the source of extreme social prejudice. TT4CL is working hard to bring a promising oral treatment for CL into clinical trials. Registration with regulatory agencies requiring proof of conformance to stringent safety, efficacy and tolerability requirements could change the lives of millions of people globally.

Objective

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.

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RIA - Research and Innovation action

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Call for proposal

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(opens in new window) H2020-SC1-BHC-2018-2020

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Coordinator

ST GEORGE'S HOSPITAL MEDICAL SCHOOL
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 1 178 863,88
Address
CRANMER TERRACE
SW17 0RE London
United Kingdom

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Region
London Inner London — West Wandsworth
Activity type
Higher or Secondary Education Establishments
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Total cost

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Participants (7)

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