Breakthroughs Blog

Over the past century, partnerships between the US government and scientists have led to major breakthroughs in global health research and development (R&D). Consistent US government funding, combined with the nation’s strong research universities and entrepreneurial spirit, has positioned America as a world leader. This investment has paid off in both health and economic gains. However, that foundation has recently weakened, as US government actions have sharply reduced research spending and limited global health research collaborations. These changes risk driving US researchers abroad and eroding the very strengths that made the United States such a scientific powerhouse. The result could be fewer breakthroughs, fewer economic benefits, and a costly brain drain for our nation. How we got here America’s global health research leadership is built on the backbone of decades of government investment by agencies like the National Institutes of Health (NIH), the Biomedical Advanced Research and Development Authority (BARDA), the Centers for Disease Control and Prevention, and the US Agency for International Development (USAID). This support built a top-tier talent pool at home and drew innovators from abroad—giving the United States a strong competitive advantage. That advantage is now in jeopardy. Recent policy and budget decisions have led to the termination of thousands of NIH grants, the wind-down of BARDA’s mRNA vaccine work, the closure of USAID and near-total cancellation of its health R&D awards, and the loss of more than 20,000 jobs at the Department of Health and Human Services. More cuts and restructuring are proposed.New federal rules are also disrupting universities—the backbone of US biomedical research. To stay eligible for federal funding, institutions must meet shifting requirements, adding costs and uncertainty. At the same time, new NIH policies have restricted international partnerships and broadly limited "dangerous gain-of-function research,” including studies that leading experts consider to be safe. Research on infectious diseases like tuberculosis has been disrupted as a result.  Funding for training young scientists is also falling. NIH’s main program for supporting early-career researchers saw its grant spending drop from $2.2 billion in 2024 to $1.7 billion in 2025. Without reliable support, many students and junior researchers may take their talents abroad—or abandon their field altogether. The international response In the wake of these changes, other countries are moving quickly to recruit US researchers.  In May, the European Union pledged $567 million over three years to “make Europe a magnet for researchers.” Similarly, the Netherlands launched a new fund to attract top international scientists, and Norway unveiled a £7.2 million program to recruit researchers. The head of Norway’s Research Council pointed to the pressure US researchers are facing on academic freedom as part of its rationale.  Individual universities are also joining in. Aix-Marseille University in France launched its Safe Place for Science program to attract US scientists who may “feel threatened or hindered in their research.” The Free University Brussels created a dedicated point of contact for US researchers interesting in continuing their work in Belgium. The message from abroad is clear: America’s loss can be their gain. So what?As the US retreats, the center of gravity for biomedical research may shift overseas. Data from Nature’s jobs board shows that in the first few months of the current administration, US job-seekers applying abroad rose sharply: 41 percent more to Canada, 32 percent to Europe, 20 percent to China, and 39 percent to other Asian countries compared to the same period in 2024.  Early-career researchers are being hit hardest. At the most innovative and impactful stage of their careers, opportunities are disappearing. Many have been left jobless or unsure if they have a future in their field. The US economy is also at stake. In 2024, NIH-funded research supported more than 400,000 jobs across all 50 states and generated $94.60 billion in economic activity—a return of $2.56 for every dollar spent. Reductions in US spending could shift those economic benefits to other countries. And since no other nation has the resources to fully replace the US’s historic role, we could see fewer medical breakthroughs for the world overall. Without strong and predictable federal research support, the United States risks losing its place as a top engine of global biomedical progress. Young researchers see more uncertainty than opportunity, and other nations are investing heavily to take the lead, accelerating a US brain drain. If US policymakers do not act to restore the foundational factors that rooted American scientific leadership, the future of science may unfold elsewhere—and the next generation of discoveries may not be American-led.  

A Phase 1 clinical trial testing an experimental monoclonal antibody for malaria found that the highest dose of the treatment studied was highly effective in preventing malaria. Researchers from the University of Maryland, Sanaria, the Vital Narrative consultancy, and the Gates Medical Research Institute tested the monoclonal antibody, MAM01, in 37 adults who had never been exposed to malaria, who received either a single dose of varying sizes or a placebo, and then were infected by the bites of infected mosquitoes. None of the three recipients who received the highest dose had malaria parasites in their blood after infection. The monoclonal antibody could prove to be an important treatment option that provides immediate, high-level, and long-lasting protection with just one doseA Japanese research team has isolated natural compounds from marine sponges that demonstrate an ability to kill parasites that cause leishmaniasis, which affects approximately 12 million people around the world. Current treatments are highly toxic, cause serious side effects, and can be prohibitively expensive, leaving patients, particularly in low-income communities, which face a disproportionate burden of leishmaniasis, unable to access or complete treatment. Drug resistance is also complicating leishmaniasis treatment efforts. These findings could guide research into new treatment approaches and could potentially also be applied to other protozoan diseases like Chagas disease and African sleeping sickness. A new CRISPR-based RNA test could help track the parasites that spread Human African trypanosomiasis, or sleeping sickness, which affects around 500 people per year and many more livestock animals. The test can identify parasite species living in an animal’s blood, helping public health workers carry out targeted treatment of infected animals and prevent transmission to humans. This One Health approach to zoonotic or parasitic diseases could also be rolled out to address other diseases, such as Chagas disease or leishmaniasis.  

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.