Breakthroughs Blog

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. Last week, BioVersys AG https://www.cidrap.umn.edu/antimicrobial-stewardship/partnership-aims-foster-development-drug-non-tb-mycobacteria announced a new agreement with Shionogi to jointly develop its novel drug for treating non-tuberculosis (TB) mycobacteria, which affects approximately 250,000 people per year, primarily in North America and Asia. Shionogi will provide $6.3 million as an upfront investment for the development of the drug candidate, BV500, through clinical studies and licensing. BioVersys has stated that the candidate has the potential to become a best-in-class therapeutic for non-TB mycobacteria infections, which are currently treated with a lengthy and complex regimen.Scientists at King’s College London have developed a new class of antifungal compounds that could lead to a promising new tool against Candida auris, a dangerous fungal pathogen and a growing global threat. The researchers modified an existing class of antifungal drugs called azoles (which can lose their effectiveness as C. auris becomes resistant to them) to be more impervious to resistance, an approach that has demonstrated promise in lab and preclinical studies. If the new antifungals continue to succeed in further preclinical studies, this research could pave the way for a new, first-in-class antifungal that continues to work in the face of resistance, helping reduce mortality and limit the spread of resistant strains of bacteria.A research team from the University of California, Riverside has made a significant advance in our basic understanding of the Plasmodium falciparum malaria parasite, a step toward the development of new and improved drugs for malaria. The researchers identified two key proteins within the parasite’s cells that control gene expression and used advanced genetic tools to reduce the expression of these proteins, which led to the death of the parasite, verifying their essential role and potential as a drug target. Crucially, these proteins also have no human counterparts. These findings help pinpoint essential targets for developing future drugs that are effective and have minimal side effects.

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. Newly published research suggests that a patient-centered approach, combining novel diagnostic tools with targeted therapies, could be a game changer in tuberculosis (TB) treatment. Current TB treatment requires a complicated, months-long regimen that can be hard for patients to adhere to, leading to treatment failure and the emergence of drug-resistant TB strains. The researchers behind the new study suggest that new diagnostic tools that can better distinguish between patients with less and more severe forms of TB combined with, respectively, shorter or longer, more resource-intensive treatment courses could improve patient outcomes, reduce the burden on health systems, and limit the emergence of drug resistance.Last week, Bavarian Nordic announced the start of the first of two clinical trials testing the use of its Jynneos mpox/smallpox vaccine in vulnerable populations, including infants and children two years old and younger and pregnant or breastfeeding women. If the vaccine demonstrates safety and efficacy, the trial data could help support expanded access to the vaccine for the most vulnerable populations. The trial is being led by the University of Antwerp and the University of Kinshasa and supported financially by EDCTP3 and the Coalition for Epidemic Preparedness Innovations.A recently published study found that a single-dose, long-lasting flu drug could provide protection for an entire flu season, offering longer and more effective protection than our current seasonal flu vaccines. The clinical trial followed 5,000 healthy adults for the entire duration of the most recent flu season, finding that the highest dose of the drug provided more than 76 percent protection compared with a placebo shot. The drug has also demonstrated efficacy against a wide variety of flu strains in a previous study in mice, compared to the season influenza vaccines, which are designed (prior to flu season based on expert assumptions) to protect against at least three of the seasonal strains that circulate each season and whose efficacy depends on how well they match the flu strains that end up circulating.

Over the past few months, the US administration has made sweeping changes at the Department of Health and Human Services (HHS), the backbone of America’s public health and health research infrastructure, which contains the National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and Biomedical Advanced Research and Development Authority (BARDA). Together, NIH, CDC, and BARDA, in conjunction with other agencies across the government, support the development of health technologies that save and improve lives in the United States and around the world, including a key role in combatting emerging infectious diseases and longstanding global health challenges like HIV/AIDS.   As the United States’ lead agency for medical research, NIH has long been at the forefront of scientific discovery, pushing forward innovations in health technology across a range of health areas. In compliment, BARDA supports the advanced development of medical countermeasures against diseases with pandemic potential to shore up the nation’s public health security, and CDC, alongside tracking the spread of diseases and other health threats, supports the development of new diagnostic, prevention, and surveillance technologies and evaluates the effectiveness of existing tools to inform future research and development (R&D) needs. Far-reaching cuts to HHS’s workforce and infrastructure and the terminations of thousands of research grants—with further budget reductions proposed—threaten to reverse decades of scientific progress, shrink the pipeline of next-generation health technologies, weaken the country’s biomedical workforce and industry, and make Americans and people around the world more vulnerable to longstanding and emerging health threats.  Widespread job losses and restructuring jeopardize key R&D functionsThe US administration has overseen the dismissal or abrupt exits of up to 20,000 federal employees across HHS—amounting to 25 percent of the workforce. Thirteen hundred jobs were eliminated at NIH and around 2,400 at CDC (while some jobs have since been restored, the administration’s pivots have made it hard to track the scale and permanence of these changes), including the removal of directors and gutting of divisions focused on infectious disease and global health partnerships. The departure of these staff—from seasoned leaders to young recruits—represents an immense loss of expertise and promise and could lead to severe disruptions in critical research and surveillance efforts.Additionally, HHS has also canceled thousands of research grants over the past few months—estimates of the value of terminated funding at NIH alone range from $1.8 to $2.7 billion (more than 1,000 grants terminated and 1,000 additional projects that had funding delayed) and billions more from CDC and BARDA, with research on COVID-19 and other emerging infectious diseases and HIV/AIDS particularly impacted. Moreover, a new NIH policy halting foreign subawards could upend international research collaborations and put thousands of additional grants at risk of termination, while a policy to cap NIH indirect research costs, which has remained blocked in the courts, could further erode available funding to support researchers.Beyond these initial layoffs and cuts, the administration is also proposing a restructuring effort and federal budget for fiscal year 2026 that would reduce funding for NIH, CDC, and BARDA by 43, 53, and 36 percent respectively and implement a new primary focus on chronic disease at HHS, shuttering key centers carrying out infectious disease research and forging global partnerships, including the NIH Fogarty International Center and CDC’s Global Health Center and National Center for HIV, Viral Hepatitis, STD, and Tuberculosis Prevention.While thoughtful and thorough reforms could serve to improve these agencies’ ability to carry out critical work and ensure American taxpayer money is well spent, the rapid pace and far-reaching scale of these cuts and changes (which have seen pushback in the courts) has aroused concern among many across academia, industry, and the medical research sector who fear long-term health and economic consequences and the risk of a US scientific workforce “brain drain,” as researchers leave the field or country altogether. Terminated research makes Americans more vulnerable to emerging health threatsFrom avian influenza to COVID-19, Ebola, and mpox, emerging health threats pose a growing risk to Americans in our increasingly interconnected world. We know all too well the significant toll of pandemics, both in terms of lives lost and health care spending and economic disruptions.NIH, CDC, and BARDA have historically played a crucial role in ensuring that Americans have the technologies to protect us against disease threats wherever they arise. The research that led to the development of the mRNA vaccines for COVID-19 and rapid diagnostic tests and the world’s first vaccine and treatments for Ebola were all supported by one or multiple of these agencies.Unfortunately, research on COVID-19 and other emerging threats have been among those areas most heavily impacted by recent grant terminations, caught in the crosshairs of various efforts to claw back COVID-19 funding; curtail funding for mRNA, gain-of-function, and vaccine research; zero out funding for specific universities; and prioritize chronic disease research.For example, at NIH, an initiative founded in 2022 to support the development of antivirals against a range of pandemic threats, including the viruses that cause Ebola, Zika, and measles, was defunded. Other NIH-funded projects to develop pan-coronavirus vaccines that could protect against COVID-19, MERS, SARS, and other yet-to-emerge coronaviruses, as well as drugs targeting a range of RNA viruses, including coronaviruses, avian influenza, and measles, were also cut.At CDC, $11.4 billion in funding allocated for the COVID-19 response was pulled back, including funding that was supporting essential surveillance and preparedness activities for COVID-19 and other emerging health threats. Meanwhile, at BARDA, grants supporting the late-stage development of mRNA vaccines for avian influenza were terminated over concerns about the safety of mRNA technology, despite scientific consensus that mRNA vaccines are safe and saved millions of lives during the COVID-19 pandemic.If enacted, the proposed HHS restructuring and president’s budget proposal would also eliminate key functions across NIH and CDC that serve as our first line of defense against pandemic threats. These include institutes and centers, like the NIH Fogarty International Center and CDC Global Health Center, that provide critical training, forge international partnerships, and monitor and respond to threats abroad.   If a new outbreak were to start (or if one of the several ongoing ones suddenly worsened) tomorrow or next month, the precious expertise, resources, and technologies in the pipeline that are now being lost or rolled back would mean Americans would be less safe and less prepared. In fact, the very ability to swiftly identify a new outbreak is now in question. Research terminations set back progress in the fight against HIV/AIDS and other longstanding global health challengesHIV has led to the deaths of more than 700,000 people in the United States since 1981, with 1.2 million Americans now living with HIV. NIH and CDC have played a crucial role in developing technologies and supporting programs that have reduced the number of new infections and enabled Americans and people around the world living with HIV to live longer and healthier lives.NIH has long been a global leader in HIV R&D, working with partners to isolate the virus for the first time and discovering a new class of antiviral drugs that helped turned HIV into a manageable chronic condition. HIV research supported by NIH has also led to technologies for other diseases and health conditions that affect millions of Americans, including cancer, heart failure, and even COVID-19. But efforts to drastically reduce funding for infectious disease research, including HIV research, at the agency could slow the discovery of future health breakthroughs.NIH has already terminated at least $759 million in grants involving HIV research, with many caught up in attempts to realign US investments away from diversity, equity, and inclusion efforts; zero out federal funding for several universities; crack down on funds going to foreign sub-awardees; and cut all foreign aid dollars for South Africa.  The latter two policies targeting research done outside of the United States are of particular concern for HIV R&D. While HIV continues to affect many Americans, to ensure new technologies are broadly effective, it is crucial to conduct research in diverse populations of people living with or at risk of HIV, including populations outside the United States—especially in South Africa, which remains the epicenter of the pandemic. The executive order ending US aid to South Africa has already threatened the continuation of at least 24 HIV trials being carried out there, including those advancing some of the most promising new tools. (The impact of these two policies also goes far beyond HIV/AIDS, with basic and clinical research on other longstanding global health challenges like tuberculosis and malaria also disrupted).NIH supports four global HIV clinical trial networks, which facilitate cross-sector and international partnerships furthering HIV R&D, all of which have seen their ongoing research or functions hampered in some way in the last few months by terminations and this collection of policies. They are now having to grapple with their future; one network has already scaled back ongoing studies, declined new research opportunities, laid off employees, and canceled at least six scheduled trials.NIH also notified two major consortia working on developing an HIV vaccine that it will not renew funding next year. While new long-acting HIV prevention tools represent major breakthroughs, most experts agree that a traditional, immunizing vaccine is still needed to sustainably end HIV/AIDS as a global health challenge. The consortia’s work has had tremendous success, furthering what are widely considered the most innovative, experimental vaccine candidates into clinical trials.  CDC has also played a complementary role in HIV efforts, leading surveillance and supporting research to help improve prevention and treatment efforts in the United States and globally. It is also a key implementing agency of the President’s Emergency Plan for AIDS Relief, or PEPFAR. Proposed budget cuts and restructuring at CDC would curtail domestic HIV prevention and surveillance activities, as well as eliminate the agency’s Global Health Center and, with it, a specific funding line for global HIV and TB activities (though the agency will likely continue to implement certain PEPFAR activities), undermining the ability of public health officials to track new infections and ensure patients can start treatment in a timely manner. They would likewise dismantle the NIH Fogarty International Center and, with it, programs that have long played a key role in training the next generation of HIV researchers.These changes threaten to set us back in the decades-long effort to develop a vaccine and other game-changing technologies to address HIV/AIDS, which were gathering momentum recently, with promising vaccine clinical trial results and the recent approval by the US Food and Drug Administration of an injectable HIV prevention drug (developed using evidence from NIH-funded research) that provides long-term protection—the next best thing from a vaccine. Delays and suspensions of ongoing research, as well as disrupted surveillance and services, could not only stall the development of the next generation of technologies, but also potentially lead to a resurgence of HIV in the United States and around the world.The bottom lineInvestments in global health R&D, including investments in emerging infectious disease and HIV/AIDS R&D, at NIH, CDC, BARDA, and other US agencies have demonstrated a huge return on investment, delivering 67 new technologies approved since 1999 for neglected and non-COVID emerging infectious diseases. Additionally, between 2007 and 2022, at least 86 percent of all funding that the US government directed to global health R&D was reinvested in American companies and institutions. This investment has created an estimated 600,000 new American jobs, spurred $104 billion in direct economic activity, and yielded scientific knowledge to inspire further innovations, which will ultimately generate $255 billion in long-term benefits to the American economy.  As the global heavyweight in biomedical R&D, NIH investments alone have generated hundreds of thousands of jobs and billions in new economic activity in every state (including the most rural ones), bolstering the American universities, research institutes, and businesses that make up the biomedical sector. And it pays off: every $1 received from NIH generates $2.46 on average in increased economic activity. In fact, we’re already beginning to see the dramatic economic fallout of this pullback in investment. These past investments have made America a global leader in biomedical innovation. As other countries start to pursue American researchers who have lost jobs and funding and China makes major investments and advances in life sciences R&D, for the first time surpassing the United States in drug clinical trials in 2024, America could fall behind. Investing in biomedical research is a tide that raises all boats and benefits cross-cutting health areas. In an era of great scientific opportunity but increased risks, this is the time to invest more and shore up the critical work at HHS agencies to boost the economy, America’s leadership on the world stage, and our ability to protect Americans and people around the world from an ever-increasing roster of longstanding and new health threats.