A sponsored satellite session heard how teams at the Drugs for Neglected Diseases Initiative (DNDi) are making exceptional progress in the battle against neglected tropical diseases.
Neglected tropical diseases (NTDs) are extremely common and are responsible for a huge burden of disease, especially in sub-Saharan Africa. However, the lack of markets in high-income countries leads to very low levels of investment in intervention development. One of the organisations addressing this gap is DNDi, and at a sponsored satellite session, DNDi staff and academic collaborators described recent progress in three key disease areas.
Welcoming delegates, Dr Luis Pizarro, DNDi Executive Director, Switzerland, noted that, as well as EDCTP, it was also the 20th anniversary of DNDi. Like EDCTP, DNDi can point to considerable impact, including in the area of NTDs. These achievements, Dr Pizarro suggested, were based on multiple international partnerships: “It’s very important to have all of you in the room because every achievement we have done, it’s a collective achievement.”
Innovative R&D and built capacity for the elimination of sleeping sickness
Dr Florent Mbo, HAT Platform/DNDi, Democratic Republic of the Congo (DRC), summarised DNDi’s work on human African trypanosomiasis (HAT) and the activities of the HAT Platform, a cross-sectoral collaboration, which was launched in the DRC in 2005.
DNDi adopted a strategic approach, identifying development needs for treatments and diagnostics, developing target product profiles (TPPs) for each. For treatment, it first developed nifurtimox-eflornithine combination therapy (NECT), which was introduced in 2009, as a shorter and simpler treatment for stage 2 (advanced) disease. In 2018, an all-oral treatment, fexinidazole, was approved for both stage 1 and stage 2 disease. In late-stage development is an additional drug, acoziborole, a single-dose oral drug that could form part of “test and treat” approaches and make a major contribution to disease elimination.
The HAT platform has also had a focus on capacity building. Priorities have included building technical skills, strengthening ethics committees, and promoting exchange between endemic countries.
Visceral leishmaniasis: On the brink of game-changing treatment options
Dr Fabiana Alves, DNDi, Switzerland, described recent progress in therapy development for Leishmania infections, including the potentially lethal visceral leishmaniasis, a particular public health challenge in East Africa.
Key to elimination will be early diagnosis and treatment. DNDi is working with partners to refine existing treatments and develop new ones, particularly short-course oral treatments. Great progress has been made by the EDCTP-funded AfriKADIA project on optimised treatments for visceral leishmaniasis, results from which led to an updating of WHO treatment guidelines recommending use of miltefosine and paromycin.
Parallel to these studies has been longer-term work on all-oral regimens. A pipeline of potential new treatments has been developed with partners, including a Novartis compound being evaluated in a phase II proof-of-concept trial in the EDCTP-funded VL-INNO project. Studies are also addressing the need for improved diagnostic tools, while developing formulations specifically for children is also a long-term priority.
Helminths: Partnering for the pipeline
Professor Coralie Martin, National Museum of Natural History, France, discussed progress in combating helminth infections, a diverse range of parasite infections affecting many millions of people worldwide.
The mainstay of disease control is mass drug administration. However, although several effective drugs exist, they do have some drawbacks and some treatment gaps remain. New tools will be needed to achieve elimination goals.
Key partnerships include the HELP (Helminth Elimination Platform) Consortium funded through the Eu Horizon 2020 programme, a public–private partnership focused on development of new treatments, for example through drug repurposing. In addition, the eWHORM collaboration is undertaking a range of activities to advance progress in line with the WHO elimination roadmap.
Nothing less than health for all – what is at stake when we talk about partnership and innovation for NTDs
Spring Gombe, Market Access Africa, Switzerland, concluded by noting the continuing need to maintain the spotlight on NTDs, notwithstanding key investments from bodies such as EDCTP and increasing country interest. Furthermore, progress will be dependent on groups working together: “We have heard from our presenters that no one stakeholder will do it all. Deep and broad partnerships are required to achieve progress and to sustain success,” she said.
Key activities include an analysis by Policy Cures Research of health and economic impacts over the past 20 years, development of an indicator framework that will help to enhance understanding of these infections and the benefits of control, to support country decision-making, and an updated infectious disease pipeline tracker which includes all the investigational candidates for neglected diseases and emerging infectious diseases and the approved products registered since 1989. The new analyses will be launched at the World Health Assembly meeting in 2024.