News

Recently published results from a Phase 2b trial testing a new regimen for people with chronic Trypanosoma cruzi infection could offer a safer, more practical treatment option for the infection, which affects more than seven million people globally, mainly in Latin America, and can develop into Chagas disease. Compared to the current standard regimen that involves twice-a-day treatment for 60 days and is frequently accompanied by uncomfortable side effects, leading to treatment failure, the new regimen involves a 30-day course taken once daily. Trial participants treated with the shorter regimen had significantly reduced side effects, found it easier to follow, and saw lower treatment interruption. If it continues to demonstrate success in large Phase 3 trials, the regimen could transform treatment for patients globally, as T. cruzi infection becomes a growing challenge in the United States, Europe, and Japan.The first participants have been vaccinated in a trial in the Democratic Republic of the Congo (DRC), where the country’s ongoing outbreak of the Bundibugyo strain of Ebola has now become the largest Ebola outbreak in the country’s history and the second largest in history. Health workers in Bunia, a DRC city at the center of the outbreak, were the first to be vaccinated in the real-world efficacy trial evaluating whether the Ervebo vaccine, which is designed to target the more common Zaire strain, offers some cross-protection against Bundibugyo. There are no approved vaccines for Bundibugyo Ebola, and there were no candidates in late-stage development before the start of the outbreak of the rarer strain of the virus, which has only caused three outbreaks since it was first discovered around 20 years ago. Since the outbreak began, global partners have fast-tracked the development of earlier-stage candidates, but researchers hope the Ervebo vaccine could provide some protection before those candidates are ready for evaluation in DRC. As artificial intelligence (AI) continues to rapidly transform many aspects of technology and science, including health research, a new report from the World Health Organization (WHO) speaks to how stakeholders can help ensure AI-enabled health research is conducted responsibly and benefits everyone. According to the report, while AI has the potential to accelerate health innovation and improve health outcomes, it also presents risks related to equity, privacy, safety, transparency, and more. WHO suggests that researchers, ethics committees, regulators, funders, and policymakers can act to strengthen ethical oversight of AI throughout the health research continuum. WHO also notes that most AI development remains concentrated in high-income settings and contends that strengthening leadership, capacity, and participation in AI in low- and middle-income countries will be important to ensuring the technology does not exacerbate existing inequities in health and health research. 

Last week, Gilead Sciences announced that it has expanded the scope of its voluntary licensing agreements with six manufacturing partners, who are already manufacturing the recently approved twice-yearly lenacapavir for HIV prevention, to include investigational once-yearly lenacapavir. Following the unprecedented Phase 3 trials for twice-yearly lenacapavir, an ongoing Phase 3 trial is evaluating a once-yearly option that could provide a full year of prevention with just one annual dose, further expanding prevention options and addressing persistent barriers to uptake and adherence. Establishing these agreements at this early stage helps manufacturers, regulators, health systems, and communities prepare for the broad, rapid, and equitable scale-up of the once-yearly option, if it is eventually approved. The World Health Organization (WHO) has updated its malaria guidelines to include Coartem® Baby, the first antimalarial developed specifically for newborns and young infants weighing between two and five kilograms. This patient group previously had no medicine specifically designed for their unique needs, leaving them to be treated with modified doses of formulations intended for older children, raising the risk of treatment failure or toxicity. The drug was developed by Novartis and partner Medicines for Malaria Venture (MMV), with financial support from the European & Developing Countries Clinical Trials Partnership and the Swedish International Development Cooperation Agency. WHO’s inclusion of Coartem® Baby in the guidelines informs and enables malaria-endemic countries to introduce the treatment in national malaria programs, supporting broader access. After receiving prequalification by WHO and approval from Swiss and Ghanian regulatory authorities, Coartem® Baby has so far been rolled out in 11 countries. The Coalition for Epidemic Preparedness Innovations (CEPI) recently announced that it is awarding $3.9 million to support research projects aimed at improving understanding of the Bundibugyo ebolavirus behind the ongoing outbreak in the Democratic Republic of the Congo. Specifically, the funding will support modelling and analytic teams led by the Institut National de Recherche Biomédicale, Imperial College London, and Makerere University School of Public Health. These teams will collect, integrate, and analyze outbreak data to guide the outbreak response; inform the design of fast, efficient vaccine clinical trials; and support longer-term preparedness against similar outbreaks in the region. To ensure long-term applicability, the partners have placed a strong emphasis on capacity strengthening and open sharing of data and methods, so that the tools and knowledge these projects generate can be utilized in future outbreaks in the region. 

GHTC submitted the below response to a public hearing on a proposed reorganization of the National Institute of Allergy and Infectious Diseases (NIAID), warning of potential implications and urging the need for transparency throughout any future restructuring efforts.The National Institute of Allergy and Infectious Diseases (NIAID) is one of the world’s leading funders of global health research and development (R&D) and plays a unique role within the National Institutes of Health (NIH). The Institute’s mandate requires it to anticipate and respond to emerging and persistent infectious disease threats, advance understanding of disease mechanisms, and support the development of critical countermeasures. As the Global Health Technologies Coalition (GHTC), a coalition of more than 50 nonprofit organizations, academic institutions, and aligned businesses working to accelerate the development and delivery of new drugs, vaccines, diagnostics, and other health technologies, we are committed to ensuring that NIAID’s structure and mandate sustain this work.The Division of Clinical Research (DCR) is a key component of the research ecosystem, providing the infrastructure, oversight, expertise, and standards needed to translate scientific discoveries into safe and effective interventions for patients. DCR’s centralized structure within NIAID has supported patient safety, scientific integrity, clinical trial operations, and the advancement of medical innovation. These functions are essential for moving promising discoveries from the laboratory into products that can improve and save lives.This is particularly important for global health R&D. Clinical research increasingly spans borders, disease areas, and institutions, with universities, academic medical centers, nonprofit product development partnerships, and other partners contributing essential expertise and infrastructure. International clinical research is necessary to ensure that new tools are effective across diverse populations and can ultimately be developed into affordable, fit-for-purpose products for communities facing the greatest disease burdens. DCR provides multi-disciplinary services across NIAID to help facilitate high-quality clinical trials across the globe. Maintaining strong coordination between domestic and international research efforts is therefore essential to US leadership in biomedical innovation and global health.The proposed reorganization of NIAID, including the dissolution of DCR and redistribution of its core functions, risks undermining this coordination by creating new silos, fragmenting clinical research expertise and oversight, and weakening integration across domestic and international research activities. It could also introduce unnecessary disruption at a time when the United States must strengthen, not weaken, the infrastructure needed to respond rapidly to infectious disease threats and translate scientific advances into effective countermeasures.Additionally, it is critical to ensure that with any restructuring, NIAID maintains transparency, consistency, and strong oversight of clinical operations. Disruptions to established systems and expertise should not contribute to funding and administrative delays, increase burdens on clinical research partners, or jeopardize the timely, safe, and effective conduct of clinical trials. Ultimately, we ask NIAID to ensure these changes do not slow the development of new interventions against some of the world’s most pressing health challenges.NIAID’s ability to connect basic science, clinical research, and product development is a critical component of US biomedical leadership. Preserving the infrastructure and coordination that make this possible—including a strong, centralized clinical research function within NIAID—is essential to protecting research participants, maintaining scientific rigor, advancing global health R&D, and ensuring that US investments in discovery translate into health technologies that reach the people who need them most.

Last week, the World Health Organization (WHO) prequalified multi-dose vials of the maternal vaccine against respiratory syncytial virus (RSV), enabling rollout in lower-income countries. RSV poses a severe global threat, especially for children under five and newborns, in particular. The Gates Foundation supported the development of a multi-dose version of Pfizer’s Abrysvo maternal RSV vaccine, which is administered to pregnant people in their third trimester, protecting newborns against infection and greatly reducing babies’ risk of being hospitalized with RSV. Multi-dose vials simplify the logistics of vaccination, allowing health care professionals to vaccinate more people with fewer vials, which need to be stored and transported in ultra-cold conditions. The prequalification now enables international organizations, like Gavi, the Vaccine Alliance, which has already put implementation plans in place, to procure them for rollout in low-income countries. A research team has developed a nanobody-based antivenom that could provide a more flexible and effective approach for treating snakebites, which kill tens of thousands of people globally and disable hundreds of thousands more. Traditional venom is made by processing antibody-filled blood from horses injected with small doses of venom, but the quality of these antivenoms can vary, they can cause severe allergic reactions, they are expensive to produce, and they offer inconsistent protection across snakebites from different species. Nanobodies, tiny versions of antibodies, can move through tissue more quickly and reach venom before it can cause permanent damage. In a new study, all mice treated with the nanobody-based treatment survived. The treatment also prevented the most severe effects of the venom, is not expected to cause unwanted side effects, and remained intact at high temperatures, which is key in the resource-constrained rural settings that face heightened risk. Further studies will be needed to optimize the dose and ensure safety and efficacy in humans, but the study suggests that nanobody-based antivenoms could be a promising approach for further study. Immunoscript Life Science will receive up to $8.9 million from the Coalition for Epidemic Preparedness Innovations (CEPI) to further development of its thermostable next-generation self-amplifying RNA (saRNA) vaccine platform, which could help improve access to RNA vaccines globally. mRNA vaccines have demonstrated significant promise as a tool against emerging disease threats because of the speed, flexibility, and adaptability with which they can be produced, but their requirement for ultra-cold temperatures for storage and transportation has proven a barrier to access in low-resource and remote settings. The saRNA vaccine platform, now owned by Immunoscript, is an alternative technology that could help overcome challenges associated with the cold chain. With CEPI’s funding, the company will conduct an early-stage trial in people in India to assess the safety and immune response of the technology.