Precision medicine tools for the neglected tropical disease visceral leishmaniasis

Visceral leishmaniasis is a fatal neglected tropical parasitic disease, mainly affecting the poorest of the poor, particularly in Eastern Africa. No good, safe, affordable and easy-to-use drugs are currently available, due to a consistent lack of drug development.

This project aims at optimizing and individualizing current and future oral (combination) treatments for visceral leishmaniasis, adapted to the largely paediatric patient population.

To this end, dosing of the only currently available oral drug miltefosine will be adapted and optimized for children and loading dose strategies will be developed to enable shorter regimens.

Evidence-based optimal fixed-dose combination strategies of future novel all-oral combination regimens will be established.

Innovative sets of biomarkers to monitor treatment response will be identified to enable more effective personalized therapy for leishmaniasis.The project will utilize state-of-the-art pharmacometric modelling and simulation techniques in combination with systems pharmacology, machine learning, and pharmacokinetic and pharmacodynamic data collected in Eastern African clinical trials.The precision medicine tools developed in this project will lead to shorter and more effective oral dosing regimens for leishmaniasis and provide new ways to identify patients at risk for relapse of disease.

This will improve accessibility, cure rate and patient-friendliness of the treatment for neglected leishmaniasis patients, including children.