Recipient organisationDrugs for Neglected Diseases Initiative
Funding£10.0M
PeriodJan 2019 — Jan 2022
In plain English
AI plain-English summary
For millions of people in some of the world’s poorest regions, treatment for the parasitic disease leishmaniasis still means weeks of painful, toxic injections. This programme aims to replace those injections with a simple pill—an oral combination of two drugs that patients could take at home. The problem is stark: current antimonial therapies are so harsh that many patients abandon treatment, and drug resistance is rising. The research brings together a consortium including DNDi, GlaxoSmithKline, and Pfizer to evaluate ten candidate compounds. The goal is to select two orally-active new chemical entities and test them together in Phase II human trials, with a backup option of pairing one new drug with the existing oral drug miltefosine. If successful, this would transform leishmaniasis from a disease requiring hospital-based care into one treatable with a short, well-tolerated course of tablets. That shift would not only save lives but also ease the burden on fragile health systems in rural Africa, Asia, and Latin America, where the disease quietly disfigures and kills.
View original technical description
The programme goal is to transform leishmaniasis treatment from the use of toxic, painful injectable antimonial therapies to a simple, orally acting and well tolerated treatment easy to use in resource poor settings. The strategy will rely on developing an oral combination of two co-administered drugs to maximise efficacy, reduce dose and duration of treatment, minimise side effects, and prevent or delay the emergence of drug resistance. DNDi, GlaxoSmithKline, the University of Dundee, Anacor, Pfizer, The Global Alliance for TB Drug Development, Takeda Pharmaceuticals and Celgene have built an unprecedented portfolio of candidates against Leishmania parasites. This pipeline is a strong basis for advancing towards oral leishmaniasis therapy(ies), to overcome attrition in development and identify one or more combination treatments for visceral leishmaniasis as the priority disease and also potentially for cutaneous leishmaniasis. This programme will evaluate 10 candidates aiming to select two orally-active New Chemical Entities (NCEs) ready for testing as a combination in Phase II studies in patients, maintaining a backup option of a combination of a single NCE with the existing oral drug, miltefosine. In the longer term, the programme aims to deliver new oral, high efficacy and affordable drug combinations for leishmaniasis.”
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