Other organisations recorded by Europe PMCAberystwyth University · The University of Texas Southwestern Medical CenterThese are researcher affiliations; the award is counted once under the primary recipient above.
Funding£10.0M
PeriodSept 2025 — Sept 2030
In plain English
AI plain-English summary
A single drug, praziquantel, is the only treatment available for the 140 million people in 78 countries infected with schistosomiasis, and it fails to kill juvenile worms. This leaves patients vulnerable to reinfection and creates a dangerous reliance on one agent, as resistance could emerge at any time. The research team has built a toolkit of methods to find new drug candidates that kill the parasite at all stages of its life cycle in the human body. They will combine two strategies: testing compounds for their effect on whole worms (phenotypic screening) and designing molecules to hit specific parasite proteins (target-based approaches). A key step is “target deconvolution”—identifying exactly which protein a successful compound attacks, which bridges the two approaches and guides further development. If successful within five years, the project will deliver a pre-clinical drug candidate active against adult worms, juvenile worms, and schistosomula. That candidate could then enter human trials, potentially breaking the cycle of mass drug administration that currently keeps the disease at bay but cannot eliminate it.
View original technical description
Schistosomiasis is a neglected tropical disease (NTD) affecting more than 140 million people in 78 countries. Mass drug administration with a single agent, praziquantel (PZQ), is the primary strategy for disease control, with an estimated 260 million people receiving periodic treatment. However, the sustainability of this strategy is at risk due to PZQ’s inactivity against juvenile worms and the potential for PZQ resistance to emerge in endemic communities. To achieve morbidity control and elimination, new effective treatment options are required. Over the last 3 years, our team has assembled a unique toolkit of approaches to support schistosomiasis drug discovery. We are now in an excellent position to leverage this toolkit to deliver lead compounds with activity against all human-residing life-cycle stages for the treatment of schistosomiasis. We will combine phenotypic and target-based approaches to accelerate the drug discovery process. These two strategies will be bridged through comprehensive drug target deconvolution studies to identify the molecular target(s) of phenotypically active compounds. We will be guided by Compound Progression Criteria based on the Target Product Profile for new anti-schistosomal treatments. Our overarching aim is to deliver a pre-clinical drug candidate with activity against adult and juvenile worms and schistosomula within 5 years.
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