Liverpool Researchers Trial Oral Treatment for Deadly Leishmaniasis Disease
Key Insights
A groundbreaking clinical trial for visceral leishmaniasis (search) is underway in Liverpool, testing an oral drug (DNDi-6899) that could replace current painful injection treatments requiring hospitalization.
The collaborative effort involves NHS University Hospitals of Liverpool Group (search), University of Liverpool, and Drugs for Neglected Diseases initiative (DNDi) to combat a disease that affects 50,000-90,000 people annually.
Researchers aim to develop a treatment that can be easily stored, transported, and administered in low-income regions, potentially saving thousands of lives affected by this neglected tropical disease.
A clinical trial testing a new oral treatment for visceral leishmaniasis (search), one of the world's deadliest parasitic diseases, is currently underway at the NIHR Liverpool Clinical Research Facility. The collaborative research effort brings together the NHS University Hospitals of Liverpool Group (search) (UHLG), the University of Liverpool, and the Drugs for Neglected Diseases initiative (DNDi).
Visceral leishmaniasis (search), transmitted through the bites of infected sand flies, affects between 50,000 and 90,000 people annually, with half of those infected in Eastern Africa being children under 15. The disease presents with flu-like symptoms including fever, fatigue, and weight loss, and is almost always fatal if left untreated.
"Visceral leishmaniasis (search) 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," said Professor Richard Fitzgerald, Director of the Liverpool CRF and Principal Investigator of the study.
Current Treatment Challenges
Existing treatments for visceral leishmaniasis (search) require daily injections for up to 17 days, causing immediate pain during administration, nausea, vomiting, and potentially more serious complications. These treatments also present logistical challenges in remote areas with limited healthcare infrastructure.
The new oral drug being tested, DNDi-6899, could overcome these barriers by being easier to store, transport, and administer—potentially eliminating the need for hospitalization and making treatment accessible to vulnerable communities in parts of Asia, East Africa, and Brazil where the disease is prevalent.
The Liverpool Trial
The trial is being conducted at the NIHR Liverpool Clinical Research Facility at the Royal Liverpool University Hospital and is funded by Wellcome (search). It forms part of the work done at the University's Centre for Experimental Therapeutics (TherEx), which aims to improve health by optimizing existing drugs and developing new treatments.
Professor Saye Khoo, Director of TherEX at the University of Liverpool, emphasized the practical benefits of the potential new treatment: "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 hospitalization."
The trial is currently recruiting healthy volunteers from Liverpool and surrounding areas, with several participants already having completed the screening process. The study will take place over two weeks, with volunteers closely monitored by clinical specialists.
Global Health Impact
Dr. Fabiana Alves, Director of the leishmaniasis programme at DNDi, highlighted the global significance of the research: "Visceral leishmaniasis (search) 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."
The disease has received limited research attention despite its devastating impact. This Phase I trial in Liverpool represents a critical step toward developing more effective, safe, and patient-friendly medicines for those most at risk.
Sabrina Lamour-Julien, Research Lead in Therapeutics at Wellcome (search), noted: "This clinical trial is a great example of the progress already being made for visceral leishmaniasis (search), 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."
Local Expertise with Global Reach
The trial also showcases Liverpool's position at the forefront of global health research. "This trial offers hope for a more practical and effective solution, one that could reach those most at risk and save countless lives," Professor Fitzgerald said. "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."
The 26-bed research facility where the trial is taking place provides comprehensive amenities for participants, including en-suite rooms, catering services, and facilities for remote work during their stay.
As climate change potentially expands the geographical reach of neglected tropical diseases like leishmaniasis, the development of accessible treatments becomes increasingly urgent. This Liverpool-based research represents a significant step toward addressing a critical global health challenge that disproportionately affects the world's most vulnerable populations.