Breakthroughs Blog: HIV/AIDS

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 from a team at the Liverpool School of Tropical Medicine found that the drug nitisinone works as a potent contact insecticide for killing the mosquito species that spread malaria, dengue, Zika, and other emerging mosquito-borne viral threats, offering hope of a new approach to combating insecticide resistance. Nitisinone, which is currently used to treat a rare genetic condition in humans, kills mosquitoes by blocking an enzyme that is necessary for them to process human blood, which is not a pathway targeted by any currently used insecticides. The research suggests that the drug could be formulated for use in indoor residual spraying or insecticide-treated bed nets, helping eliminate mosquitoes even in settings where rising insecticide resistance has undermined the effectiveness of current vector control methods.Early last week, the Gates Foundation announced a $2.5 billion commitment through 2030 to accelerate research and development on women’s health. Specifically, the funding will help advance more than 40 innovations in five critical areas: obstetric care and maternal immunization, maternal health and nutrition, gynecological and menstrual health, contraceptive innovation, and sexually transmitted infections. These areas were selected based on extensive data about where innovation can save and improve the most lives and where there are persistently high rates of misdiagnosis, as well as direct insights from women in low- and middle-income countries about their needs, preferences, and challenges.Last week, IAVI; the Mutala Trust; ReiThera; and the Ragon Institute of Mass General Brigham, the Massachusetts Institute of Technology, and Harvard University announced that the first doses of an investigational HIV vaccine candidate were administered in their Phase 1, first-in-human clinical trial. The clinical trial is taking place at three sites in Zimbabwe and South Africa among 120 healthy adults, including people living with HIV. Carrying out the trial in sub-Saharan Africa, which faces the highest disease burden, will help ensure the vaccine is effective and relevant in the communities where it is most needed. The vaccine candidate is designed to stimulate the immune system, particularly CD8+ T cells, to recognize and target critical structural regions of the virus. Scientists believe CD8+ T cells could potentially target HIV-infected cells, so the trial will also assess whether the vaccine candidate could be part of future therapeutic or cure technologies.

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. Scientists have launched the first-ever library of adjuvants, which are substances that boost the ability of vaccines to create stronger, longer-lasting immunity. The repository, which will be housed at the United Kingdom’s Medicines and Healthcare products Regulatory Agency and funded and led by the Coalition for Epidemic Preparedness Innovations, will include 25 adjuvants available for use in developing new vaccines against epidemic and pandemic threats. The library will act as a matchmaking service, helping developers select the best suited adjuvants to increase the effectiveness and potency of their vaccines. The project is intended to support the 100 Days Mission, an ambitious global effort to develop vaccines against newly emerging threats in as few as 100 days.A new study has found that delivering a single gene therapy injection at birth could offer years-long protection against HIV. In the study, non-human primates who received the therapy within their first month of life saw protection for at least three years without the need for a booster—a potential signal that protection could last into adolescence in humans—, while those treated at 8-12 weeks saw a less effective immune response. The immune system is naturally more tolerant within a person’s first weeks of life, so it marks an optimal window for gene treatment that could be rejected at an older age. While questions remain as to whether these findings will translate to human infants and children, if successful, this treatment could offer a “one-and-done” multiyear solution to protect again mother-to-child HIV transmission that could be more cost-effective and feasible to implement in low-resource settings worldwide.The antiparasitic drug ivermectin was found to reduce the incidence of malaria by 26 percent in a trial conducted in Kenya, a finding that demonstrates its potential use for mass drug administration alongside other malaria control interventions. Invermectin is a drug commonly used to treat parasitic diseases like onchocerciasis and lymphatic filariasis, but scientists have been exploring its potential in malaria prevention, given the rise in insecticide resistance, which has rendered other prevention tools like insecticide-treated bednets and indoor residual spraying less effective. The trial, which involved more than 20,000 participants, compared the drug, administered in a single monthly dose for three months during the rainy season, with the antiparasitic albendazole—both alongside use of existing bed nets. It is the largest study of ivermectin for malaria conducted to date.

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 Drugs for Neglected Diseases initiative (DNDi) and AN2 Therapeutics announced a new collaboration to advance clinical development of AN2’s oral drug candidate for the treatment of chronic Chagas disease, which can remain asymptomatic for years while the infection wreaks irreversible damage in the body, potentially leading to the development of life-threatening conditions. The drug candidate, AN2-502998, is currently being tested in a Phase 1 first-in-human clinical study, scheduled for later in 2025, with a Phase 2 proof-of-concept study expected to commence next year. The partnership will leverage DNDi’s established network of clinical trial sites throughout Latin America and Spain, established through its previous Chagas clinical trial work.A team of researchers shared last week that they have developed a special kind of floss that could serve as a vaccine delivery method, delivering proteins and inactive viruses to the gums and triggering immune responses that can protect against infectious disease. While scientists have long been investigating alternatives to syringes like oral and nasal vaccine delivery methods, a challenge they’ve come across is that the mouth and nose have naturally tough defenses against foreign molecules. The flossing approach overcomes this challenge by targeting the pockets of gum between the teeth that are better at absorbing molecules. The approach proved successful in mice in multiple tests where researchers coated the floss in protein and inactive flu virus, and researchers have also tested the feasibility in humans, finding positive results. Further research will help determine if the technique will work in humans with a wide variation in gum health, including people with gum disease.Study results presented at the recent International AIDS Society Conference in HIV Science found that more than half of people living with HIV who received a pair of broadly neutralizing antibodies plus the drug N-803 experienced delayed viral rebound or maintained a low or suppressed viral load during interruption from antiretroviral treatment. While antiretroviral treatment can keep HIV suppressed indefinitely, the long-lasting viral reservoir maintained in the body has confounded researchers efforts to eliminate it, putting a cure out of reach. A small proportion of individuals naturally make unique broadly neutralizing antibodies that target key parts of the virus, which researchers have been trying to replicate with a manufactured drug. N-803, which is approved as a treatment for bladder cancer and is being studied in both cancer and HIV research, appears to be able to both reactivate dormant HIV and clear the virus out of hiding. Further research and an analysis across studies testing the approach will help confirm the underlying mechanisms of the effects researchers are seeing and determine whether the drug could be part of a future cure.

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.