A clinical trial is underway in Liverpool, bringing together NHS University Hospitals of Liverpool Group (UHLG), the University of Liverpool, and Drugs for Neglected Diseases initiative (DNDi), a medical research non-profit organisation, in the effort to address visceral leishmaniasis - one of the world's deadliest parasitic diseases.
Visceral leishmaniasis is a neglected tropical disease found in parts of Asia, East Africa, and Brazil. It is caused by leishmania parasites and transmitted through the bites of infected sandflies. Despite its impact, the disease has received limited attention in many regions.
The disease presents with flu-like symptoms, including fever, fatigue and weight loss, and is almost always fatal if left untreated.
Current treatments require daily injections for up to 17 days which can cause immediate pain during the treatment, nausea, vomiting, as well as more serious complications.
This study is testing an oral drug (DNDi-6899) that could overcome these barriers by being easier to store, transport, and administered-potentially saving thousands of lives.
The trial is being conducted at the NIHR Liverpool Clinical Research Facility (CRF) at the Royal Liverpool University Hospital and is funded by Wellcome. It is also part of the work done at the University's Centre for Experimental Therapeutics (TherEx). The centre aims to improve health by optimising existing drugs and developing new treatments.
Professor Richard Fitzgerald, Director of the Liverpool CRF and Principal Investigator of the study, and Professor Saye Khoo from the Centre for Experimental Therapeutics at the University are leading the trial to evaluate the safety and effectiveness of the new oral treatment.
Professor Fitzgerald said: "Visceral leishmaniasis claims thousands of lives every year, and current treatments are often inaccessible to those who need them most, particularly in low-income regions where healthcare systems are limited.
"This trial offers hope for a more practical and effective solution, one that could reach those most at risk and save countless lives.
"It also reflects the skills and expertise here in Liverpool, showing that we are at the forefront of developing new medicines for a disease that affects people on the other side of the world."
Professor Saye Khoo, Director of TherEX, University of Liverpool added: "By collaborating with DNDi, we aim to develop a treatment that can be stored in varying conditions, delivered directly to vulnerable communities, and administered easily-without the need for hospitalisation."
The trial is currently recruiting healthy volunteers from Liverpool and the surrounding areas, with several participants already having completed the screening process. The trial will take place over two weeks at the Liverpool CRF, where volunteers will be closely monitored by clinical specialists.
The CRF offers a 26-bed unit, consisting of two four-bed bays and 18 en-suite single rooms. Additional amenities include a TV lounge, accessible en-suite bathroom facilities, and Wi-Fi for remote work and internet browsing. Catering services are also provided throughout the duration of the stay.
Dr Fabiana Alves, Director of leishmaniasis programme at DNDi said: "Visceral leishmaniasis affects the poorest of the poor and is linked to climate change, which can lead to its expansion to new areas.
"One billion people are at risk globally. An estimated 50,000 to 90,000 new cases occur every year - most of them in Eastern Africa, where half of those infected are children under 15. We urgently need better, effective, safe and patient-friendly medicines, and this Phase I trial in Liverpool is playing a critical role toward that goal."
Sabrina Lamour-Julien, Research Lead in Therapeutics at Wellcome, said: "Neglected tropical diseases, including visceral leishmaniasis, are debilitating diseases that disproportionately affect the poorest communities. Their absence from the global health agenda means there are limited treatment options.
"This clinical trial is a great example of the progress already being made for visceral leishmaniasis, particularly with the focus on areas with the highest incidence of disease - providing hope for a treatment that is tailored specifically to benefit those most at risk. It is a step in the right direction; we need to continue advancing research and innovation if we are to eliminate these diseases."
Photo: Lab technician at the NIHR Liverpool Clinical Research Facility.
