Neglected tropical diseases occur almost exclusively in impoverished populations in low- and middle-income countries. Hardly known in industrialized nations, they attract little public attention or research funding. One example is schistosomiasis. Our aim is to eliminate this neglected disease as well as other, more familiar infectious diseases such as malaria.
Strategy for preventing and treating infectious diseases
Our strategy focuses on the elimination of schistosomiasis and malaria. To implement our strategy, we develop and provide medicines, improve diagnostics, counter disease transmission, increase disease control, expand access to healthcare, conduct awareness programs, and strengthen local health systems.
Our fight against schistosomiasis
Schistosomiasis, also known as bilharzia, is a tropical disease caused by parasitic worms and one of the most prevalent parasitic infections in Africa, placing a significant burden on public health and the local economies. The disease affects almost 240 million people worldwide, with more than 90% of cases occurring in Africa. An estimated 200,000 people die every year from long-term effects of schistosomiasis, such as liver and kidney infections, bladder cancer, genital schistosomiasis, and anemia. School-aged children are particularly vulnerable to the disease and often suffer from the long-term consequences and complications, including anemia, stunted growth, and learning difficulties.
Our ultimate aim in all our schistosomiasis-related work is to eliminate the disease as a public health problem. To achieve this goal, we adopted an integrated schistosomiasis strategy that we are implementing in close collaboration with multiple partners worldwide. The approach focuses on five pillars to ensure progress:
- Treatment: We donate up to 250 million tablets of praziquantel per year to endemic countries in partnership with WHO. Nearly 50 years after its development, praziquantel still remains the standard of care for the effective treatment of schistosomiasis around the world.
- Research and Development (R&D): We advance R&D to support the global fight against schistosomiasis. In particular, we drive collaborative R&D programs for innovative treatments, including a new, pediatric formulation of praziquantel for children under the age of six, more sensitive diagnostics, and vector control methods. We also strengthen research expertise and capacity through collaborations with local institutions in endemic countries.
- WASH (Water, sanitation and hygiene): Since schistosomiasis is transmitted through contaminated water sources, we also support WASH projects that aim to prevent transmission of the disease through the provision of sanitary infrastructure and new access-to-water technologies.
- Health education: We invest in education and behavior change initiatives that raise awareness of the causes and dangers of schistosomiasis and teach people how to prevent it.
- Advocacy and Partnerships: Collaborating with partner organizations for our programs and initiatives, as well as with the wider stakeholder community through the Global Schistosomiasis Alliance (GSA), we are accelerating the progress towards schistosomiasis elimination.
Our fight against malaria
According to World Health Organization (WHO) estimates, nearly half of the world’s population is at risk of contracting malaria. More than 200 million cases of malaria and over 400,000 related deaths are recorded every year, with almost 70% occurring in children under the age of five. Over 90% of cases and of deaths occur in Africa. Every two minutes, a child dies from the disease.
There is a need for new products to overcome the problem of increasing drug resistance and to achieve our goal of elimination. Through our “As One against Malaria” program, we are helping to deliver integrated and sustainable health solutions entailing treatments, diagnostics and prevention methods to fight malaria in endemic countries.
Schistosomiasis: Over one billion tablets donated
As part of our long-standing partnership with WHO, we are committed to donating praziquantel tablets annually. To date, our tablets have been distributed in 47 endemic African countries to treat school-aged children. In 2020, we donated around 226 million tablets for distribution in 30 countries, 27 of which are in sub-Saharan Africa. Moreover, we maintain our commitment by ensuring that we have sufficient production capacity to manufacture up to 250 million tablets a year.
Schistosomiasis health education project
In 2020, we extended our partnership with the NALA Foundation for another three years. This joint health education project focuses on southwestern Ethiopia and is aimed at promoting long-term behavioral change in the drive to eliminate schistosomiasis and other neglected tropical diseases. WASH measures play a crucial role in these efforts. Our local partners conduct training sessions in schools and among local communities. The majority of participating schools have established hand-washing stations: Safe water and latrines are now available in these stations throughout the school year, and teachers also report major improvements in students’ personal hygiene and general levels of cleanliness in the schools.
The GSA: A central platform in the battle against schistosomiasis
The Global Schistosomiasis Alliance (GSA) is a coordinated, multi-sectoral effort to combat the complex disease schistosomiasis. The GSA continues to help eliminate the disease through its working groups and to raise awareness through coordinated campaigns. In November 2020, the World Health Assembly endorsed a new WHO road map that “sets global targets and milestones to prevent, control, eliminate and eradicate 20 neglected tropical diseases and disease groups” for the time period from 2021-2030. The GSA contributed to WHO consultations during the roadmap conception phase.
Partners in schistosomiasis research
Over time, we have developed a portfolio of R&D projects on schistosomiasis. These include making a new child-friendly pediatric formulation of praziquantel available to treat children under the age of six, identifying new drugs to prevent and treat schistosomiasis, developing innovative and highly sensitive schistosomiasis diagnostic methods, and defining new technologies for safe water access and approaches for vector control.
If left untreated in children of preschool age, schistosomiasis can have long-term effects such as anemia, stunted growth and impaired learning. It can seriously affect their lives and potentially cause chronic diseases, including bladder cancer or female genital schistosomiasis. Together with the Pediatric Praziquantel Consortium, we develop, register and provide access to the pediatric formulation of praziquantel. Consortium partners include both public and private sector representatives from developed and endemic countries to ensure that the strategy and implementation meets local requirements and needs.
The program is currently in Phase III clinical development. The pivotal trial is designed to evaluate the efficacy and safety of the new pediatric praziquantel ODT (orodispersible tablet) formulation in children three months to six years of age who are infected with schistosomes. The trial is taking place in Côte d’Ivoire and Kenya, and is co-funded by the Consortium, the European & Developing Countries Clinical Trials Partnership (EDCTP), and the Global Health Innovative Technology (GHIT) Fund. The study represents the last step of the clinical development program and is designed to support registration and market authorization for this important new treatment for very young children. Due to the Covid-19 pandemic, the study needed to be paused in line with national restrictions.
Meanwhile, together with international key stakeholders, we are defining an innovative procurement access path, including local manufacturing, to ensure the future affordability, availability and adoption of the new medicine.
Praziquantel is an effective and well-tolerated drug, but it is not effective in all developmental stages of the parasite. We continue to collaborate on research activities with many partners. This work aims to discover new, long-lasting compounds to treat juvenile forms of the parasite, thereby improving efficacy and preventing reinfections. In 2019, we obtained promising assets from Salvensis and the London School of Hygiene and Tropical Medicine. We have since worked to identify potential new candidates for preventing infection and curing patients affected by schistosomiasis. In 2020, we identified a lead molecule for further development to potentially serve as an alternative to praziquantel.
The need for more sensitive diagnostics is crucial in the fight against schistosomiasis. Since early 2019, we have been collaborating with the Foundation for Innovative New Diagnostics (FIND) to develop a sensitive rapid diagnostic test (RDT) to improve mapping and case detection for schistosomiasis. Building on the prototype, a consortium of partners was formed in 2020 to accelerate the development.
In 2020, we entered into a strategic alliance with Janssen Pharmaceuticals Inc. to develop an artificial intelligence-based diagnostic tool to improve the diagnosis of neglected tropical diseases, schistosomiasis and soil-transmitted helminthiasis (STH).
Beyond these efforts, we continue to explore technologies that control transmission factors through basic research activities, for example the elimination of the infectivity of snails through gene editing, or through an access-to-water program in Senegal. In partnership with the Access to Water Foundation, we intend to provide innovative safe-water platforms to local communities and health centers, in order to improve sanitation and reduce exposure to parasites. Details can be found under Community Engagement.
We also continue to implement research and advocacy initiatives to address female genital schistosomiasis (FGS), a major challenge to women’s health in Africa, and its impact on HIV/AIDS. In particular, we supported a clinical trial to optimize therapeutic treatment for women suffering from FGS in Madagascar, with results expected in 2021.
Malaria: Developing new therapeutic solutions
As part of our “As One against Malaria” program, we are developing a new drug (M5717) for the prevention and treatment of malaria. In 2020, we initiated a Phase Ib clinical trial (for prevention) to test efficacy of the compound in the liver/first stage of infection in healthy volunteers.
The World Health Organization (WHO) recommends that new drug therapies against malaria should include a combination of two active principles with different mechanisms of action, in order to prevent the emergence of resistance. We have therefore initiated the discussions with other partners to evaluate drug candidates that can be combined to initiate the next clinical development phase for treatment and/or prevention.
Our strategic collaboration with the University of Cape Town in South Africa and the Medicines for Malaria Venture continues its drug discovery activities with the aim of identifying new therapeutic solutions for malaria and building research capacity in and for Africa. Together with our partners, we have identified promising drug candidates that are progressing into preclinical stage.
Preventing and controlling malaria transmission
Preventive methods, such as the use of insect repellents, form part of our strategic toolkit to combat malaria. We are testing our insect repellent IR3535®, which is already used for protection against the bites of insects and ticks that can transmit diseases such as Lyme, Zika, Dengue, and Chikungunya.
In a three-phase program defined in 2020 and implemented in Ghana, we are evaluating a new formulation technology for long-lasting efficacy of IR3535® – through laboratory tests and in a community-based study. Positive results would enable IR3535® to serve not only as a preventive method for personal use, but also, on a larger scale, as a vector control method to support population-based National Malaria Control programs.
In partnership with local institutions in Africa, we are improving health worker capacity in Ghana in using microscopy to detect cases of malaria and other diseases that can be diagnosed via blood samples even faster. In addition, we have established PAVON (Pan-African Vivax and Ovale Network), an African network of centers of excellence for the epidemiological surveillance and scientific research on malaria. We involved over ten African countries in the PAVON project in 2020.