Breakthroughs Blog: Child health

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. 

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. 

The Gates Foundation has awarded ProFound Therapeutics up to $35 million to find new drug targets for preeclampsia, a high blood pressure condition during pregnancy that can be severe for mother and child and is a significant contributor to maternal and fetal deaths globally, particularly in low- and middle-income countries. ProFound will utilize their vast repository of proteins and data to improve scientific understanding of how preeclampsia is developed in the body, with the goal of identifying promising approaches for future drug development. Once targets are identified, the company will explore their features using an AI tool and then evaluate them in laboratory experiments.Last week, the Coalition for Epidemic Preparedness Innovations (CEPI) announced that it will invest up to $16.5 million to advance the development of an African-developed vaccine candidate for the Bundibugyo strain of Ebola, which is behind the fast-growing outbreak in the Democratic Republic of the Congo. CEPI’s support will help advance the candidate through preclinical development and into an early-stage clinical trial in Africa. The vaccine candidate uses a technology that is utilized by a licensed vaccine against smallpox and mpox and has elicited long-term immunity in a vaccine targeting the Zaire strain of Ebola. It is the fifth Bundibugyo-specific vaccine candidate supported by CEPI in response to the current outbreak and the only of the five designed to induce two major parts of the immune system, both B- and T-cells, for comprehensive and long-lasting immunity.A novel tuberculosis (TB) vaccine candidate failed to match the current, more-than-100-years-old vaccine in preventing TB infections in newborns, according to the recently shared results of a Phase 3 trial conducted among 6,897 healthy newborns in sub-Saharan Africa. TB remains the world’s leading infectious disease killer and there is an urgent need for a new vaccine—the current TB vaccine, BCG, is effective at preventing some types of TB in infants, but it offers inconsistent protection in adults against pulmonary TB. One of 17TB vaccine candidates in the pipeline, the vaccine in the trial, VPM1002, is a genetically modified recombinant BCG vaccine developed by the Serum Institute of India. While disappointing, the results of the trial do provide significant insights about how best to design robust, feasible efficacy trials for future vaccine candidates.

Last Thursday, the World Health Organization (WHO) announced that, with support from Gavi, the Vaccine Alliance, it will allocate 70,000 doses of an approved Ebola vaccine to the Democratic Republic of the Congo, which is facing the largest Ebola outbreak in the country’s history. The vaccine, Ervebo, targets Ebola Zaire, the most common Ebola strain, not the Bundibugyo strain behind the current outbreak—for which there are no approved vaccines —but early laboratory and animal data suggest it may provide some cross-protection. Twenty thousand doses will be used in a Phase 3 trial to evaluate whether the vaccine offers cross-protection for Bundibugyo, while the remaining doses will be allocated to frontline health care workers, serving the dual goals of providing some protection for at-risk populations against severe illness and death during the current outbreak while gathering critical data to inform use of the vaccine in future Bundibugyo outbreaks.Researchers at the Walter and Eliza Hall Institute of Medical Research have developed a rapid blood test that could improve detection of iron deficiency, a widespread health challenge disproportionately affecting mothers and children in low-resource settings where access to conventional laboratory-based screening methods are limited. Using a finger-prick blood sample, the test provides results within 15 minutes without the need for specialized laboratory infrastructure and highly trained health care providers. It has demonstrated strong correlation with gold-standard laboratory methods used in high-income countries and includes a built-in reference line to make the results easier to interpret. The test is currently undergoing evaluation in sub-Saharan Africa.The Coalition for Epidemic Preparedness Innovations (CEPI) shared last week that it will dedicate $4.17 million to four research projects designed to assess whether additional vaccines currently or previously licensed against Ebola Zaire could offer some cross-protection against severe illness and death during the current rapidly growing outbreak of Bundibugyo Ebola. Specifically, the funding will go to teams at the University of California, Los Angeles in the United States; the Institute of Tropical Medicine in Belgium; the University of Oxford in the United Kingdom; and the MRC-University of Glasgow Centre for Virus Research in the United Kingdom. The research teams will use blood samples from vaccinated individuals to assess immune responses and potential cross-reactivity of antibody responses, helping establish and standardize antibody tests for Bundibugyo, inform interpretation of clinical trial results, and guide future strategies. These research initiatives will support trials during the current outbreak and complement ongoing efforts to develop Bundibugyo-specific vaccines.