Breakthroughs Blog: Medicines

The Democratic Republic of the Congo has approved the use of a new single-dose oral treatment for sleeping sickness, a potentially fatal disease which is endemic in several countries in Central and West Africa. The approval of Acoziborole Winthrop, codeveloped by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, follows successful Phase 2 and 3 clinical trials conducted in the DRC and Guinea by DNDi in partnership with national authorities, which demonstrated that the drug is safe and effective. Existing treatments require a ten-day oral treatment regimen or a combination of injection and oral therapy that requires several days of hospitalization for patients in the most advanced stages. The DRC approval, which will make the drug available free of cost to patients in the country, is expected to be followed by endorsements in other endemic countries soon.A research team from the University of Maryland School of Medicine’s Center for Vaccine Development and Global Health has shared promising early results from a first-in-human clinical trial comparing a new investigational vaccine against Lassa fever and rabies to a licensed rabies vaccine, finding that the vaccine candidate was safe and induced rapid and robust immune responses against both viruses. There are currently no available vaccines for Lassa fever, a dangerous disease considered a priority threat with epidemic and pandemic potential that is common in parts of Western and sub-Saharan Africa, which also have a high burden of another severe disease: rabies. A dual vaccine could help streamline health care delivery in settings with limited health care infrastructure; the vaccine candidate can also be freeze-dried for storage, making it well suited to low-resource settings where it can be difficult to maintain the cold chain. Last week, DNDi, the Medicines for Malaria Venture (MMV), and the Global Antibiotic Research and Development Partnership (GARDP) announced a new cooperative agreement to explore how the three organizations can work together to advance global health research and development (R&D) amid global aid cuts and calls to reshape the global health architecture. The organizations plan to pool their expertise and resources to advance the R&D of effective and affordable medicines, while centering patient needs and equity. In addition, the partners will collaborate on policy advocacy and communications activities to raise awareness of the impact and promise of the nonprofit product development partnership model in advancing innovative, lifesaving medicines for critical, but often overlooked, diseases.  

Preeclampsia affects millions of pregnancies around the world, claiming the lives of 42,000 women each year and contributing significantly to fetal and newborn mortality and complications. Early detection and management are crucial to prevent progression to eclampsia, which is characterized by seizures and can be life-threatening.  The burden of preeclampsia is greatest in low- and middle-income countries (LMICs), where workforce shortages, limited laboratory capacity, and procurement challenges undermine the implementation of recommended tools and protocols.  Despite its global impact, preeclampsia remains under-researched and under-represented in the health innovation pipeline compared to conditions with similar prevalence that are not specific to women. These challenges have created critical gaps in screening, diagnosis, and treatment, but new tools in the pipeline could help transform care, particularly in low-resource settings worldwide. Rethinking preeclampsia screening and diagnosis Preeclampsia screening aims to identify high-risk pregnant women before symptoms appear and before they can be formally diagnosed, enabling closer monitoring and preventive care. Screening has traditionally relied on a checklist to evaluate patient history and risk factors. While simple, this approach has shown limited effectiveness and depends heavily on the accuracy of assessment.Newer recommended approaches that combine risk-factor screening with biomarker testing offer improved predictive performance. However, they require medical and laboratory imaging infrastructure that is not widely available in LMICs, limiting their uptake.  Preventive therapies, including low-dose aspirin, calcium supplements, and antihypertensive medications, are recommended for women identified as high risk, but they are not consistently offered to women in LMICs. As a result, many women are diagnosed later in pregnancy, when risks are greater.  The Accelerating Innovation for Mothers project identified 89 preeclampsia risk screening tools in the R&D pipeline but concluded current approaches are unlikely to adequately address the needs of women in LMICs, underscoring the need for greater investment in tools explicitly designed to address their realities.  Artificial intelligence (AI) is emerging as a promising tool to bridge screening and diagnosis gaps. With support from the Gates Foundation, Siemens Healthineers is developing an AI-driven tool that leverages data from blood tests already routinely performed as part of prenatal care to screen for both preeclampsia and anemia. By using existing samples and integrating screening for multiple conditions, this tool could enable more efficient and scalable screening in resource-limited settings. Confirmatory preeclampsia diagnosis relies on detecting elevated blood pressure and the presence of protein in urine, alongside the potential presence of severe symptoms. This requires trained health care workers and access to laboratory equipment, which can hinder accurate, timely diagnosis in resource-challenged environments. Monod Bio, with Gates Foundation support, is developing a rapid, point-of-care diagnostic test that can deliver results in less than 15 minutes. Using an AI-derived algorithm to analyze results, this tool could significantly reduce workforce and equipment needs. Together, these innovations reflect a promising shift toward designing tools specifically to address the needs of low-resource environments, rather than adapting tools used in high-income contexts, a shift essential to closing global gaps in care. Expanding the treatment pipeline There is currently no specific cure for preeclampsia other than early delivery, which can be dangerous for both mother and baby, especially in low-resource settings. Existing treatments focus on managing symptoms, including preventing seizures with magnesium sulfate and controlling blood pressure with antihypertensive medications. A new generation of therapies aims to go further by addressing underlying disease causal pathways, rather than managing symptoms alone.  A research team at the University of Nebraska Medical Center, supported by a Gates Foundation grant, is advancing an innovative treatment based on a naturally occurring compound that reduces the levels of a protein that plays a key role in the development of preeclampsia. In preclinical studies, the compound has improved vascular function, reduced inflammation, and lowered blood pressure. Importantly, it is also stable at room temperature and inexpensive to produce, making it well-suited for settings with limited resources. Another Gates-supported team at the Medical College of Wisconsin has launched a clinical trial evaluating a promising supplement for preeclampsia. The compound, which occurs naturally in foods, has been shown to improve vascular functions in animal models and early human studies. It is also shelf-stable and inexpensive. These novel therapeutics could help transform preeclampsia care by slowing or halting the progression of the condition, rather than simply responding to the presence of symptoms. Their affordability and suitability for austere settings could also help expand access for women who currently lack effective treatment options. Looking ahead Preeclampsia mortality rates remain far too high, reflecting long-standing gaps in research advancing women’s health and a lack of tools suited to low-resource settings. Innovations that leverage emerging technologies like AI and respond to the needs of pregnant women everywhere could help save more mothers and babies worldwide. Solutions designed for LMIC health systems could also strengthen care in underserved or overstretched settings in high-income countries like the United States, where rising health care costs, workforce shortages, and even conflict-related supply chain disruptions are straining health care service delivery.  Turning this potential into impact will require sustained investment to ensure innovations are not only developed but designed for and delivered to the women who need them most. 

A new US National Institutes of Health-funded study conducted in seven high tuberculosis (TB)-burden countries found that a new and improved near point-of-care TB test platform was highly accurate, effective, and scaleable. Pluslife Biotech’s MiniDock MTB test platform is battery operated, low-cost, provides results in 30 minutes, and can be used with tongue swabs in addition to sputum samples, which certain groups, including babies and people living with HIV, cannot produce. The new test is well-suited for low-resource, smaller, or more rural health clinics, which may not have access to current resource-intensive, expensive molecular testing options. The University of Oxford and the Serum Institute of India have signed a license agreement to support the development, large-scale manufacturing, and potential future commercialization of the malaria vaccine candidate R78C for use in clinical settings. R78C combines two Plasmodium falciparum blood-stage antigens, designed to target the malaria parasite at multiple points in its lifecycle, offering longer and more effective protection. This new license, which builds on a long-standing collaboration between the partners on malaria vaccine work, will initially help accelerate the candidate into clinical evaluation. The Serum Institute of India’s global manufacturing capabilities will also help ensure that resulting successful malaria vaccines can be manufactured at scale and deployed affordably to malaria-endemic countries. The Medicines for Malaria Venture (MMV) and Novartis recently announced that the World Health Organization (WHO) has prequalified Coartem® Baby, the first and only antimalarial drug developed specifically for newborns and young infants weighing between 2 and 5 kilograms. This treatment will help address a long-standing gap in malaria care for this group, who are often treated with antimalarial formulations intended for use in older children, which can increase the risk of toxicity or underdosing. The prequalification designation enables countries and global procurers to start purchasing and deploying the treatment in malaria-endemic countries; Novartis is making the treatment available on a largely nonprofit basis in malaria-endemic regions. 

Last week, the Medicines for Malaria Venture (MMV) and GSK announced the world’s first deployment of a novel cure for Plasmodium vivax malaria, the predominant malaria species in Latin America. Pediatric tafenoquine, codeveloped by MMV and GSK, replaces a multiday regimen with a single-dose, child-friendly formulation, improving treatment completion and preventing relapse. P. vivax can remain dormant in the liver, so if patients don’t receive combination treatment for both the blood and liver stages of the disease, they could have multiple malaria relapses. Up until now, the lack of child-friendly tafenoquine formulation left younger kids at risk of the disease, which is associated with long-lasting cognitive impairment. The rollout is taking place among the Yanomami people, an Indigenous population living in the Yanomami Indigenous Territory in northern Brazil. Last Tuesday, Moderna announced the start of a late-stage study of its experimental mRNA vaccine candidate for bird flu, marking the first time a mRNA pandemic bird flu vaccine has reached late-stage trials. The trial is being supported by a $54 million investment by the Coalition for Epidemic Preparedness Innovations (CEPI) following last year’s cancellation by the US government of more than $700 million in contracts with Moderna supporting the late-stage development of the vaccine candidate. Moderna’s trial will evaluate the candidate, mRNA-1018, in 4,000 healthy adults in the United States and the United Kingdom. Under the agreement, Moderna also committed to, if the vaccine is approved and a flu pandemic is declared, setting aside 20 percent of its manufacturing capacity for supplying the vaccine to low- and middle-income countries at affordable prices. The US Food and Drug Administration recently approved Merck’s once-daily, oral combination treatment for HIV, offering patients another treatment option. The combination of doravirine and islatravir is being branded as Idvynso. In two large late-stage studies, the treatment combination demonstrated its ability to significantly suppress replication of HIV-1 in adults receiving another HIV treatment. While another late-stage study in adults living with HIV who had previously received treatment found that Idvynso was not inferior to Gilead’s HIV drug Biktarvy, the approval still provides another treatment option for patients. It is also currently being tested in multiple trials in combination with other antiretrovirals for potential daily and once-weekly HIV treatments.