2023 Highlights
Confronting the double neglect of women and children
An R&D agenda to upend a harmful status quo
Chagas disease in Colombia. Dengue fever in Sri Lanka. Mycetoma in Sudan. Some of these so-called neglected tropical diseases are viral, some protozoan, some fungal, but all bring the same anguish to affected families across the globe, particularly when the treatments are painful, toxic, barely effective, or require lengthy hospitalization far from home. The anguish is even greater when these diseases affect young children or pregnant women.
Women and children are doubly neglected when it comes to the availability of safe, appropriate, and effective treatments for neglected diseases.
Innovation for children’s treatment needs
Children have unique treatment needs that change as they grow. Very young children cannot swallow pills. Crushed tablets can be bitter-tasting and challenging to administer and dose accurately. Simple oral treatments adapted for different ages would be best. However, developing medicines for children can be expensive and difficult, requiring specialized studies for drugs that will ultimately be ordered only in small quantities. So, child-friendly medicines tend to come late or not at all.
DNDi is tackling this treatment gap by accelerating the development of new treatments and child-friendly formulations that meet the unique needs of kids with neglected diseases. Since 2003, we have developed four affordable treatments for malaria, Chagas disease, and HIV for children, and two treatments for sleeping sickness and leishmaniasis that are suitable for both children and adults. We are now working to develop new child-appropriate treatments for at least six neglected diseases.
In 2023, DNDi and partners published a study evaluating the effectiveness of a new combined visceral leishmaniasis (VL) treatment with fewer injections and a shorter hospital stay: miltefosine, the only oral drug available for leishmaniasis, and paromomycin, an injectable antibiotic. We studied the results in children at the same time as adults to avoid any delay in the availability of a better treatment for young patients. The positive results of this and other studies looking at how well these drugs functioned and were absorbed in children and adults in a 14-day regimen in Eastern Africa have been reviewed by the World Health Organization with the goal of an updated treatment recommendation in 2024.
The year also saw other positive advancements for children. DNDi has partnered with Novartis to develop an oral drug candidate, LXE408, currently in clinical trials for VL in India and Ethiopia, with plans for a child-friendly formulation and future studies on its effectiveness for Chagas disease, another disease that affects children. DNDi is working with Sanofi to develop acoziborole for sleeping sickness, a single-dose oral treatment that is being evaluated for children. For river blindness, DNDi is working with Insud Pharma to develop an ivermectin formulation for small children, collaborating with Bayer AG to assess emodepside, and working with the eWHORM consortium to develop oxfendazole.
Cooperation is essential. In 2023, as a Global Accelerator for Paediatric Formulations (GAP-f) partner, DNDi worked with experts from health ministries, research institutions, and health facilities in affected countries to produce the first-ever list of priority drug formulations for children affected by five neglected tropical diseases.
A watch list of the most promising drugs in the development pipeline now includes LXE408 for VL, acoziborole for sleeping sickness, and paediatric formulations of ivermectin, emodepside, and oxfendazole for treating river blindness – all drugs in DNDi’s portfolio.
DNDi also consulted experts and caregivers when defining the desired characteristics of a new treatment for kids living with Chagas disease and is now testing shorter benznidazole regimens in children to reduce the side effects of the current treatment.
Mary, from West Pokot County, Kenya, lost 35 kg (77 lbs) and was too weak to walk by the time she was diagnosed with visceral leishmaniasis, but she couldn’t be treated with the usual drugs because she was pregnant. She was cured after receiving liposomal amphotericin B, an expensive treatment for special populations such as pregnant women, the elderly, and children under two.
Developing safe and effective treatments for women at all stages of life
Women, too, have unique treatment needs that can change at different ages, but they are often excluded from the clinical trials that would provide essential data on sex-specific physiological differences and on medicine safety and efficacy during pregnancy or breastfeeding. As a result, safe treatment options for women affected by neglected diseases can be limited. In some cases, women may have to delay treatment until they are no longer pregnant or breastfeeding.
DNDi is committed to implementing best practices in gender-responsive drug development and access by promoting the inclusion of women in clinical trials and, when safe, including pregnant and breastfeeding women.
Some medicines, and therefore participation in clinical trials by women during their child-bearing years, may require the use of contraceptives to prevent pregnancy, which may not be acceptable to some women or their communities. In 2023, DNDi worked with the University of Geneva to conduct a literature review on the acceptability of contraceptive use in Eastern Africa in different contexts. As a next step, partners in Kenya, Brazil, and Colombia are planning a project to assess the acceptability of contraceptive use when medically indicated for the treatment of Chagas disease and leishmaniasis in endemic regions of Africa and Latin America.
DNDi also continued evaluations of its drug development programmes to prioritize drug candidates that are safe for everyone at all stages of life, including by ensuring that gender-specific considerations are part of all disease strategies.
DNDi now ensures that clinical study protocols explicitly include women, or justify any exclusion, and our teams are working towards reporting fully disaggregated results to uncover differences between the sexes at every stage of a study. DNDi is also ensuring that partners’ Phase I trial facilities can accommodate both women and men.
In 2024, DNDi will investigate the possibility of conducting laboratory studies to evaluate reproductive toxicity earlier in the development process to facilitate earlier and better-informed decisions about the safety of priority drug candidates during pregnancy.
DNDi is committed to developing new treatments that are safe, effective, and affordable for everyone at all stages of life.
Photo credits: Ana Ferreira-DNDi, Paul Kamau-DNDi
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