Hannah Sachs-Wetstone

This month marks 35 years since the World Summit for Children, the first time in history when a meeting of heads of state and government was held exclusively to address the well-being of children. At this landmark event in New York City, world leaders adopted a Declaration on the Survival, Protection, and Development of Children and a Plan of Action for its implementation, which made specific recommendations in a variety of areas, including child and maternal health and family planning. We’ve made significant progress in children’s health globally since then—the total number of deaths of children under five worldwide has declined from 12.8 million in 1990 to 4.8 million in 2023. The number of neonatal deaths (deaths in the first 28 days) has also dropped, although more slowly, from 5.2 million in 1990 to 2.3 million in 2023. However, there are still major challenges in children’s health, especially in the Africa region, where children continue to have the highest risk of dying before reaching age five. Infectious diseases, including acute respiratory infections, diarrhea, and malaria, as well as birth complications, remain among the leading causes of death for children under five. More than half of child deaths are due to conditions that could be prevented and treated with adequate access to health care or other quality of life improvements, particularly in that crucial first month: neonatal deaths make up 47 percent of under-five child deaths.Another challenge is the lack of medicines designed specifically for children—only 30 percent of essential medicines are available in child-appropriate formulations. Instead, children are often treated with imprecise partial doses of medicines that were designed and tested in adults, which can lead to toxicity and complications. This gap results from a variety of barriers: it can take up to 25 years to bring a pediatric medicine to market and only one in ten clinical trials registered between 2000 and 2023 included children. Innovative technologies rolled out since 1990 have already saved millions of lives, and new tools in the pipeline designed with the needs of children, and particularly the most vulnerable kids, in mind could bring down child mortality even more. Diarrheal disease vaccines While diarrheal-disease related deaths among children under five have decreased drastically since 1990, down an estimated 79 percent from 1990 to 2021, they still cause a severe burden; 4.8 million children under five died in 2023. Rotavirus, Shigella, and enterotoxigenic Escherichia coli (ETEC) are among the common pathogens that can lead to diarrheal disease.  The first rotavirus vaccines of the generation still in use today become available just under 20 years ago, approved in the United States in 2006 and later recommended by the World Health Organization for all children. Today, these vaccines are used in more than 110 countries around the world and have contributed to a significant drop in child hospitalizations and deaths from diarrhea. Rotavirus vaccines have had a marked impact on child mortality, preventing 139,000 deaths in children under five between 2006 and 2019. However, rotaviruses are still the leading cause of severe diarrhea in children under five in low- and middle-income countries, pointing to the need for increased access to existing vaccines, as well as new and more effective ones.Unfortunately, approved vaccines don’t yet exist for ETEC and Shigella, though promising candidates are in development. PATH is working with partners to advance vaccines against these bacteria, which could help save thousands of additional lives. For Shigella, PATH is working with the US Naval Medical Research Command and the Walter Reed Army Institute of Research to advance a vaccine candidate, which demonstrated strong safety and efficacy in a recent Phase 1 trial. PATH is also working with these US partners, alongside Sanofi Pasteur and the Infectious Disease Research Institute, to advance an ETEC vaccine candidate, which displayed strong potential in a Phase 1 trial in adults, paving the way for the development of a vaccine for infants and young children, who face the highest risk. If they continue to prove successful, these vaccines could have a tremendous public health impact, not only preventing deaths but also reducing the long-term health and economic effects of malnutrition and developmental delays associated with experiencing severe diarrhea, as well as limiting the growth of antimicrobial resistance.  Pediatric products for malaria and more Children under five account for nearly 80 percent of global malaria deaths. Despite this significant burden, for a long time, there was a lack of a suitable malaria medicines for children, who were treated with crushed tablets designed for adults. The first child-friendly, quality-approved malaria treatment, launched in 2009 by Medicines for Malaria Venture (MMV) and Novartis, is sweet-tasting and quickly dissolves in water. 500 million courses of Coartem® Dispersible have been delivered worldwide as of this April, saving more than 1.8 million lives. MMV also delivered the first malaria treatment for newborn babies and young infants, which is an adapted version of Coartem® that was approved earlier this year. The two first-ever malaria vaccines, approved in the last five years, have been another major game changer for children with malaria, with 21 countries in Africa having introduced them so far, alongside other complementary prevention methods like chemoprevention and vector controlMMV is also working with partner Eyam Health to develop next-generation monoclonal antibody-based approaches to create affordable, long-acting malaria preventative therapies and treatments. Eyam’s technology has the potential to deliver future durable therapies without the need for cold storage and at an extremely low cost, projecting that a single long-acting therapeutic injection could provide protection for more than 50 million children and pregnant women for the months-long rainy season, which is peak time for malaria transmission. This would significantly simplify seasonal malaria chemoprevention, which today involves taking a course of oral drugs monthly for up to five months. Alongside these innovations for malaria, child-friendly medicines for HIV and neglected tropical diseases are also helping address the gap of suitable medicines for children and reduce the burden of treatment for children and their caregivers.  Diagnostics for pre- and postnatal complications and infections Premature birth, birth complications, and neonatal infections are among the leading causes of neonatal deaths, often resulting from limited access to timely screening, particularly in low-resource settings that face a lack of health care services, poor laboratory infrastructure and insufficiently skilled health care workers. Low-cost, easy-to-use diagnostics that can be deployed at the point of care are helping ensure that mothers and babies everywhere have access to routine screening in the pre- and postnatal periods, helping catch and address complications and infections earlier. One innovation is a portable, user-friendly ultrasound developed by Philips that connects to a mobile app on a phone or tablet, helping bring high-quality diagnostics to the point of care. The technology is being rolled out in low- and middle-income countries with the support of the Gates Foundation and has already demonstrated its ability to improve informed decision-making when triaging pregnant women in rural underserved communities.Bacterial infections caused by pneumonia, sepsis, and meningitis are the primary cause of neonatal infections, and most could be averted with adequate access to preventive interventions, early diagnosis, and care. Almost half of sepsis cases occur in children under five. The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator has awarded funding to Melio, AstraDx, and QuantaMatrix to demonstrate feasibility of their rapid diagnostics for neonatal sepsis, all of which aim to provide results in mere hours, down from days. Early diagnosis is particularly important because the risk of death from neonatal sepsis increases about 7.6 percent every hour treatment is delayed. These innovations have the potential to help greatly reduce the risk of babies and mothers dying from complications and infections during the critical periods before and after birth.  Thirty-five years since the World Summit for Children, we’ve made tremendous progress in children’s health. Investing in innovative technologies specifically designed for children and striving to ensure children and their needs are considered and included in research across health areas will set up future generations for an even healthier future. 

An international research team led by the University of Liverpool has discovered an exciting new class of antibiotics with the potential to target some of the world’s most dangerous multidrug-resistant bacteria, including MRSA and Enterococcus faecium, as experts continue to warn of the growing global threat of antimicrobial resistance. The new class, Novltex, targets a key component of bacterial cell walls that does not mutate, meaning that, unlike traditional antibiotics, it could offer long-lasting protection against antibiotic-resistant strains of bacteria. Novltex is a synthetically designed platform, making it cheaper to produce and easier to scale up for global use than common antibiotics that utilize complex, expensive natural molecules. In studies so far, researchers found Novltex to be potent and fast-acting, even at low doses, and safe. A research team led by investigators at Weill Cornell Medicine has been awarded a five-year, $20.8 million grant from the National Institute of Allergy and Infectious Diseases at the US National Institutes of Health to further the preclinical development of a promising experimental HIV vaccine for pediatric populations. Experts agree that a preventive vaccine for HIV could help turn the tide of the pandemic. Previous studies of the experimental vaccine found that it was safe and may be effective in infants, although it seems to only work in young immune systems, not in adults. The new funding will support optimization of the vaccine in preparation for clinical trials in infants in parts of the world with a high burden of HIV. Researchers at Tulane University have designed an optimized CRISPR-based tuberculosis (TB) test that uses a simple tongue swab, which, if it continues to demonstrate promise, could be a breakthrough for TB testing. Current TB tests often rely on sputum, which can be difficult to collect and which not everyone can produce, and require sophisticated laboratory equipment and skilled health care workers, leading to gaps in diagnosis, particularly in low-resource settings. The researchers built on a previous CRISPR-based TB test, developing an assay that they found to be highly effective using tongue swabs and also highly sensitive in detecting TB in respiratory, pediatric stool, and spinal fluid samples. This painless and low-resource method could improve access to TB testing, particularly at the point of care in remote and low-resource communities. 

Last Wednesday, Emergent BioSolutions announced that it has received a $56 million contract extension with the Administration for Strategic Preparedness and Response to supply the US government with doses of a combined smallpox/mpox vaccine. The single-dose vaccine was first approved by the US Food and Drug Administration in 2007 for smallpox and, since 2024, has been one of two approved vaccines for smallpox and mpox. The vaccine doses, which will be stored in the Strategic National Stockpile, aim to boost US preparedness for future public health emergencies. A novel breakthrough method that utilizes artificial intelligence and advanced imaging technologies to more accurately and efficiently identify therapeutic antibodies could transform the way researchers develop treatments for infectious diseases, particularly emerging diseases, by significantly accelerating research. The method reduces the time needed to identify antibodies with potential from weeks to under a day and can also accomplish this task more efficiently, requiring far fewer researchers dedicating their time to screening thousands of candidate antibodies. This could allow researchers to move on from antibody discovery to the later stages of drug and vaccine development earlier and, ultimately, bring lifesaving products to patients faster. A recently published study found promising results for a new approach to developing broad-spectrum antivirals to treat existing viral infections and maybe even to prevent infections from emerging viral threats. Broad-spectrum antivirals are treatments that could combat a wide range of viruses by hitting essential targets that are common across many different viruses. While there are broad-spectrum treatments for bacterial infections, there has not been a similar broad-spectrum antiviral treatment approved for human use, in part because the proteins on the surface of a virus vary widely and change rapidly over time. The researchers focused on small molecules called synthetic carbohydrate receptors (SRCs), which earlier studies have suggested could be a potential target for antiviral medicines. They tested 57 different SRCs against six deadly viruses from three different viral families, including the viruses behind COVID-19, MERS, Nipah, and Ebola, finding several promising SRCs that blocked cells in the lab from being affected by all the viruses, some of which also later demonstrated promising safety and efficacy results in mice. 

Last week, Monod Bio, a spinout company from the University of Washington, announced that it has received a $1.5 million Gates Foundation grant to develop a urine-based rapid diagnostic test for tuberculosis (TB). The company aims to help fill the gap of non-sputum-based, rapid, point-of-care TB diagnostics that are suitable for low-resource settings. Most World Health Organization-recommended TB diagnostics rely on sputum, which not all patients can provide and can be logistically difficult for health care providers to collect, but current alternatives using urine and stool samples have low specificity, and current TB tests are also not available at the point of care in communities. The test will utilize the company’s NovoBodies, novel proteins designed to be highly specific and sensitive—diagnostics have primarily used natural proteins that are adapted for use in tests.  The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) last week announced that it is awarding $1 million to Zeteo Tech to further work on a rapid, non-invasive diagnostic test that can diagnose lower respiratory tract infections, like pneumonia, using breath samples. Lower respiratory tract infections kill 700,000 children under five annually. A less invasive, rapid test could help speed up diagnosis and guide more effective treatment, particularly of drug-resistant infections, helping reduce the prescribing of unnecessary antibiotics that can fuel the rise of antibiotic resistance. Zeteo’s platform analyzes the tiny particles and droplets in breath samples to distinguish between viral and bacterial infections and assess antibiotic susceptibility.  The results of a clinical trial released last week found that a common over-the-counter nasal spray that is used for seasonal allergies could work as an antiviral against a range of respiratory infections, including influenza, RSV, and the virus that causes COVID-19. The researchers suggest that the nasal spray, Azelastine, could help block COVID-19 infections by inhibiting a key enzyme that the virus uses to replicate or by preventing the virus from latching on to the point at which the virus enters human cells. If proven safe and effective in further studies, Azelastine could serve as an easily scalable, over-the-counter prevention method complementary to vaccines and other tools, especially when a virus is spreading among a community or in high-risk situations, like when traveling or attending crowded events. The research also provides another potential target for future vaccines. 

Last week, French biopharmaceutical company Osivax announced that it received a $19.5 million contract from the Biomedical Advanced Research and Development Authority (BARDA) to further develop its broad-spectrum influenza A candidate vaccine for pandemic and seasonal flu preparedness. The vaccine is designed to target a part of the virus that is highly conserved and less likely to mutate, hopefully offering a broader, more universal immune response, and it has demonstrated promising results in early clinical trials. BARDA’s funding will support ongoing clinical development and large-scale trial and manufacturing preparation, with potential options for future funding to support Phase 2b and scale-up activities. A team of researchers at the University of Nottingham successfully produced antibodies that kill the parasite that causes Chagas disease, an important step toward the development of a much-needed vaccine. Chagas is a serious neglected tropical disease, which causes 56,000 cases and 12,000 deaths every year. The researchers developed antibodies targeting two key proteins in the parasite that were found to be extremely effective at neutralizing the parasite in a crucial developmental phase when it’s capable of infection, which is key for vaccine development.   A University of Maryland research team developed an artificial intelligence (AI) tool that can quickly scan electronic medical records to identify high-risk patients who may be infected with H5N1 avian flu, as this year’s outbreak highlights the need for more robust screening interventions to ensure infections are not going undetected. The researchers used their generative AI large-language model to analyze the records of more than 13,000 adults who had visited the emergency department in Maryland in 2024 for acute respiratory illness and common H5N1 symptoms. With less than half an hour of human input, the model found 76 records that mentioned a lifestyle risk factor for H5N1, 14 of which were confirmed to have a recent exposure to animals that transmit H5N1. The tool highlights the shortcomings of the response to the latest outbreak and offers a potential new tool to help improve surveillance and testing in future outbreaks of H5N1 and other infectious diseases.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. Last week, the Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) announced that it is awarding $3 million to Baxiva to support the development of a vaccine that targets the most common serotypes associated with invasive Escherichia coli infections. E. coli is the leading cause of urinary tract infections around the world and a common cause of neonatal sepsis infections, particularly in low- and middle-income countries. The candidate uses new targets that haven’t been used in other vaccines and targets several bacterial strains. The company’s rapid development platform also enables it to quickly design vaccines targeting various types of bacteria.The Coalition for Epidemic Preparedness (CEPI) last week announced a global collaborative effort to carry out epidemiological and computational modelling projects to help strengthen scientific understanding of the Rift Valley fever and its impact across Africa, helping guide the planning of future clinical trials assessing much-needed vaccine candidates for the disease. Despite major Rift Valley fever outbreaks seen in Africa and beyond and global recognition of the need to boost R&D efforts, there are large gaps in our knowledge about the burden and spread of the disease. The two projects, led by institutions in Kenya and Tanzania and supported by global partners, will assess the impact of the disease across Africa, as well as forecast its future burden, providing crucial data for vaccine developers to plan large-scale trials when the virus is widely circulating.A new study from the University of Alabama at Birmingham demonstrates that an antibody designed to target the dengue virus could also block the spread of the Zika virus, an important finding that could inform the development of much-needed treatments for Zika. There are no approved treatments for Zika, a dangerous virus that can pose severe risks to pregnant people and their babies. In a novel preclinical in vivo model, the antibody helped stop the virus from spreading, particularly in vulnerable parts of the body, leading to improved survival rates.