A new oral medicine for treating visceral leishmaniasis is in the offing in a Phase II clinical trial conducted in Ethiopia by Drugs for Neglected Diseases initiative (DNDi) and its partners.

The medication, called LXE408, is a safer alternative to the current treatment, which involves painful daily injections administered in hospitals over 17 days, and can have severe and occasionally life-threatening side effects affecting the heart, liver, and pancreas.

LXE408 is being tested alongside the standard treatment, involving daily injections of sodium stibogluconate and paromomycin over 17 days. A similar Phase II trial is under way in India to assess the response to LXE408 in different regions.

The fight against visceral leishmaniasis in Eastern African countries necessitates sustainable elimination strategies, with better treatments playing a crucial role. The potential effectiveness of LXE408 could significantly contribute to these elimination efforts.

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This disease, also known as kala-azar, ranks as the world’s second deadliest parasitic killer following malaria. An estimated 50,000 to 90,000 new cases occur worldwide annually, and half are children under 15.

Manifesting with symptoms like fever, weight loss, enlargement of the spleen and liver, and if left untreated, death, the disease is spread through the bite of infected sandflies and is prevalent in 80 countries, particularly in Eastern Africa, South Asia, and Latin America.

It affects the poor, and is associated with malnutrition, population displacement, poor housing, and a weak immune system.

The changing climate due to global warming is altering the epidemiology of leishmaniasis, potentially expanding it to new regions.

“Our hope is that this new oral treatment will be efficacious and less toxic and can be given to patients at the primary healthcare level, close to their homes. This would help them access treatment earlier, which could significantly decrease morbidity and transmission. If the trial is successful, it could go a long way toward enabling the sustainable elimination of this terrible disease,” explained Dr Eleni Ayele, co-Principal Investigator at the Leishmaniasis Research and Treatment Centre in Ethiopia.

Efforts to eliminate visceral leishmaniasis align with the World Health Organisation’s goal to address neglected tropical diseases and eliminate visceral leishmaniasis as a public health concern by 2030.

LXE408 was initially discovered by Novartis. DNDi and Novartis initiated a collaboration and licence agreement in early 2020 to jointly develop LXE408.

Various organisations and countries, including the European and Developing Countries Clinical Trials Partnership, Norway, Germany, Médecins Sans Frontières, Switzerland, the UK, and other private foundations, are providing financial and technical support for this study, underscoring the collaborative global effort to combat this severe and potentially fatal disease.