Breakthroughs Blog: Medicines

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. 

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. On Friday, IAVI announced a €1 million investment from the Dutch government to further clinical evaluation of the organization’s investigational vaccine candidate for Sudan virus disease, a severe disease with potentially long-term health consequences, for which there are no vaccines or specific treatments. It is important to continue advancing the vaccine’s development in between outbreaks, coordinating with partners to generate key data, accelerate and improve manufacturing for the later stages of development and production, and ensure that a trial can begin promptly following the identification of a new outbreak. IAVI’s vaccine was tested in a trial during Uganda’s Sudan virus outbreak earlier this year and previously demonstrated promising results in animal models and a Phase 1 clinical trial in the United States.Researchers at the University of Texas and Moderna recently announced that in a preclinical trial in rodents, their mRNA vaccine candidate provided full protection against Marburg disease, a deadly disease closely related to Ebola, for which there are currently no licensed vaccines or treatments. Marburg has caused several outbreaks in sub-Saharan Africa, including one in Tanzania earlier this year, and the frequency of outbreaks has increased in recent years, as behavioral shifts offer more opportunities for the virus to spill over from fruit bats to humans. If the vaccine proves successful in future human trials, it could help play a key role in curbing the spread of future outbreaks.Last week, Novartis announced that the first malaria treatment for babies and small children who weigh less than 10 pounds has been approved by Swiss authorities, offering a new tool in the fight against malaria, particularly to help reduce the high rate of malaria deaths in young children in sub-Saharan Africa. Coartem Baby, developed by Novartis and the Medicines for Malaria Venture, offers significant improvement over the previous option of treating these babies and young children with versions of malaria drugs formulated for older children, which raises the risk of toxicity and overdose. Novartis plans to introduce the drug on a largely not-for-profit basis, and it could be rolled out in African countries within weeks, with the eight countries that participated in clinical trials of the drug expected to be among the first to receive it.

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. A new study looking at data from two separate Phase 1 clinical trials shows that a targeted vaccine strategy can successfully activate early immune responses relevant to HIV, a significant advance in longtime efforts to develop an HIV vaccine. A persistent challenge in advancing HIV vaccines has been finding a way to train the immune system to produce antibodies that target many variants of the tricky, rapidly mutating virus. The study, conducted by an international team of scientists from IAVI and Scripps Research, included nearly 80 participants from North America and Africa, helping ensure a future vaccine has global potential. One of the trials used a stepwise vaccination strategy, administering two doses sequentially to guide the immune system to develop antibodies, while the second trial found that an initial dose could successfully activate the desired immune cells. Both vaccines used an mRNA vaccine platform, enabling faster production and clinical testing.A recently published study found that a new water-soluble formulation of the only drug available to treat helminths, or parasitic worms, can achieve the same effect as the currently available tablet form at half the usual dose. Praziquantel is currently available as large tablets that are bitter and difficult to swallow, especially for children and pets. The new formulation can be dissolved in water and was shown to be more efficiently absorbed, enabling patients to receive a smaller dose without sacrificing efficacy. Further clinical testing is needed before the new formulation can be brought to market, but if it continues to demonstrate success, the new formulation could be a game-changer in addressing helminths, which affect 400 million people worldwide.

In 2001, I traveled to Ghana as a graduate student working on a US Agency for International Development-funded decentralization program. I arrived armed with materials on local governance best practices. But the mayors I met were focused on urgent public health issues: clean water, HIV/AIDS, tuberculosis, and malaria. That trip, meant to be an early lesson in governance, became my first and most personal education in global health. It also nearly killed me. I was taking mefloquine weekly to prevent malaria. Toward the end of our stay, my partner and I set off to explore the country. Somewhere along the way, I developed what I believed to be giardia. That infection likely compromised the absorption of the mefloquine and left me dangerously vulnerable. One night in the village of Kokrobite, I woke with a high fever and chills so intense I could barely stand. A local man saw me and immediately recognized it as malaria. He arranged for a taxi to take me back to Accra. Before I left, he handed me a small, crumpled packet labeled in Chinese characters. Inside was a blister pack of pills. The only word written in English on the package was artesunate. He told me it would help, and, in that moment, sick, scared, and weak, I took it. At the hospital, a blood smear confirmed that I had cerebral malaria caused by Plasmodium falciparum, the deadliest strain. Cerebral malaria can cause brain swelling, coma, and death. I drifted in and out of consciousness for days. At one point, hospital staff told my partner that there was nothing more they could do. I should “go home” and be “as comfortable as possible.”My partner worked relentlessly to get me on one of the next flights back to the United States. I was so sick that the airline nearly refused to let me board. Once we arrived home, I was seen right away by an infectious disease doctor at Johns Hopkins Hospital. After reviewing my labs and the herbal remedy package, he said plainly: this saved your life. The artesunate had bought me enough time to survive the journey and begin treatment. At the time, the World Health Organization had not yet formally endorsed artemisinin-based combination therapies (ACTs) as the global standard for P. falciparum malaria. That came in 2006. But the effectiveness of artesunate alone was already well known in many parts of the world. That medicine saved my life.I missed several weeks of school but made a full recovery. Without that drug and the help of my partner (now my husband) and friends in Accra, I wouldn’t be here today. More than two decades have passed, and we've made tremendous progress. Insecticide-treated bed nets, rapid diagnostic tests, and ACTs have saved millions of lives. Two malaria vaccines, RTS,S and R21/Matrix-M, have been approved. Long-acting monoclonal antibodies are showing promise as seasonal protection for children. Newer insecticide-treated nets help counter resistance. And researchers are exploring RNA-based therapeutics and single-dose cures.Many of these breakthroughs are being developed by members of the Global Health Technologies Coalition, where I now serve as Executive Director. These tools represent our best chance not just to control malaria but to end it.But progress is not guaranteed. Cuts to foreign assistance and global health research and development are already slowing the progress of innovations in the pipeline. Some promising tools may be delayed or abandoned. That means more children at risk, more untreated infections, and more drug-resistant strains emerging. The parasite has evolved before. It will do so again if we let it. And malaria is no longer a disease that happens mostly outside of the United States. In the past two years, locally acquired cases have been reported in Florida, Texas, and Maryland. Climate change, global travel, and changing mosquito habitats are making malaria a threat we can no longer treat as distant. I know how thin the line is between surviving this disease and not. For me, it’s not abstract. It’s the memory of a fevered body, the generosity of strangers, and a medicine in the right place at the right time. This World Malaria Day, I urge leaders and policymakers to stay the course. The science is moving forward. But science alone isn’t enough. If we don’t finish what we’ve started, we risk letting malaria surge back and take more lives with it. Science has given us the tools. The question now is whether we will act in time or let the disease outpace us once again.