Breakthroughs Blog: NTDs

“We might be tired of COVID-19, but it is not tired of us.” With those words punctuating his opening remarks, World Health Organization (WHO) Director-General Dr. Tedros Adhanom Ghebreyesus reminded World Health Assembly (WHA) participants that the ongoing pandemic continues to dominate both our lives and the agenda in multilateral fora. This year’s virtual assembly tried to continue to galvanize the global community around a coordinated response to the pandemic and reassert the importance of other key global health priorities. While this virtual assembly did not provide the same opportunities as years past to engage with stakeholders, GHTC tracked the proceedings and worked with partners to weigh in on key agenda items. Here are our top takeaways from this year’s forum:WHO and member states remain focused on pandemic response, health emergencies governanceWith the ongoing pandemic continuing to dominate the attention of the world, global coordination to address COVID-19 was a clear priority for WHO during the assembly. Delegates debated the challenges and gaps in the ongoing response efforts, as well as preparedness and response mechanisms for future health emergencies. The assembly also approved a resolution to bolster WHO’s work and strengthen countries’ preparedness capacities; while the resolution primarily focuses on health system strengthening, the International Health Regulations, and governance, it does include a provision that calls on member states “to facilitate investment in strong national research agendas and adequate infrastructures for research and development in support of new measures to counteract the impact of health emergencies, including non-pharmaceutical interventions.” The debate between member states over the resolution felt like a precursor for the more far-reaching reform proposals that may be in the offing next year, following the completion of multiple reviews over pandemic response that are now underway. Those reviews are looking at countries’ and WHO’s pandemic performance, WHO’s health emergencies program, and the International Health Regulations system itself. There appeared to be a slowly emerging political consensus shaping up around initiatives being advanced by both Germany and France to significantly strengthen the hand of WHO in terms of its emergencies powers to obtain data from member states and take action. Unfortunately, the proposals currently don’t formally integrate research and development (R&D) into their frameworks. WHO develops Immunization Agenda 2030  Member states expressed broad support during the assembly for the Immunization Agenda 2030, which outlines research and innovation as one of its seven strategic priorities, highlighting the need for “continued research into new and improved vaccines, technologies and vaccine manufacturing platforms, as well as innovations in immunization service delivery and program management.”Germany noted that the current strategy does not factor in the impact of COVID-19 on current immunization priorities and programs and that an additional evaluation to integrate pandemic response efforts will be necessary to adequately reflect the shifting landscape.WHO TB research and innovation strategy moves forwardThis year, WHO unveiled their new Global Strategy for Tuberculosis Research and Innovation, drawing on consultation with scientists, national tuberculosis (TB) program managers, and other officials from within and beyond ministries of health, including ministries of science and technology, representatives of civil society and affected communities, research funding institutions, and other TB research and innovation stakeholders. A resolution on this new strategy was passed by member states via silence procedure in the summer and formally adopted during the assembly.The strategy outlines four major areas for action: 1) creating an enabling environment for TB research and innovation; 2) increasing financial investments in TB research and innovation; 3) promoting and improving approaches to data sharing; and 4) promoting equitable access to the benefits of research and innovation. “This global strategy will guide member states and other relevant stakeholders in translating political commitments on TB research and innovation into concrete action backed by investment, as an integral part of efforts to end TB,” said Dr. Tereza Kasaeva, director of WHO’s Global TB Program. The WHO strategy also promotes the sharing of data arising from publicly funded research, with the ultimate goal of ensuring that the public benefits from public investments in science. The resolution also calls for WHO to provide technical and strategic assistance to member states, urges countries to strengthen efforts for TB research and innovation as a complement to broader cooperation to tackle antimicrobial resistance at all levels, and pushes for increased coordination across United Nations agencies and greater collaboration with the private sector.While the strategy is a welcome development, further investment for TB research must follow from member states. Global leaders must close the US$1.3 billion funding gap for TB R&D annually if the 2030 targets are to be achieved. WHA adopts new road map for neglected tropical diseasesWhile member states did not spend much time debating the topic, they did adopt a new road map on tackling neglected tropical diseases (NTDs) at the assembly. The NTD road map sets global targets and milestones to prevent, control, eliminate, and eradicate 20 NTD groups.One of the key components of the road map is a shift away from single-disease vertical programs to integrated approaches, which promote improved coordination and collaboration. Another distinct feature is to drive greater ownership by national and local governments, including communities. The overarching 2030 global targets include a 90 percent reduction in the number of people requiring treatment for NTDs and a call to eradicate dracunculiasis—also known as Guinea-worm disease—and the chronic skin disease yaws, which affects mainly children under 15 years old.Research and innovation will be a critical component to ensure that these targets are met, and several member states called for greater investment for new tools to combat these diseases.Civil society engagement with WHO remains limited Civil society engagement with WHO continues to be a challenging process, and after several disappointing meetings this year, this month’s assembly was no different. The often hectic and oblique communication, or lack thereof, on how civil society organizations (CSOs) could meaningfully participate in these meetings heading into the assembly was yet another sign that the trend line on productive collaboration between WHO and CSOs is heading in the wrong direction.CSOs were also prohibited from delivering statements on certain agenda items, and like member states, they were forced to consolidate a number of agenda items into a single statement as WHO pulled together several topics into one session, creating a disjointed dialogue in which not all issues received adequate airtime.Moving forward, WHO needs to provide new avenues for CSO input around these fora, whether through separate listening sessions, online consultations, or other mechanisms that allow civil society to bring its expertise to bear and provide technical feedback to various processes.This year’s assembly felt largely more ceremonial than substantive at times, given the lack of debate on many agenda items and WHO speeding through the list of topics. The pandemic will continue to be a major focus in 2021 for WHO and member states, but other health areas that have been neglected through underinvestment on the WHA73 agenda must also re-emerge as key priorities.

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 team of Brazilian researchers are leading an international consortium to discover new drug candidates for malaria, visceral leishmaniasis, and Chagas disease. The organizations involved in the consortium include FAPESP (São Paulo Research Foundation), Medicines for Malaria Venture (MMV), Drugs for Neglected Diseases initiative (DNDi), Universidade Estadual de Campinas, and the University of São Paulo. The consortium has received US$10.7 million from the participating organizations for the next five years and will prioritize selecting and training researchers from São Paulo and investing inA new study has found that delivering the BCG (bacille Calmette-Guérin) tuberculosis (TB) vaccine intravenously better protected monkeys against infection than the standard administration method of injecting it into the skin. The BCG vaccine, which is the only licensed TB vaccine, is effective in preventing early-childhood forms of TB but provides limited protection against pulmonary TB, the dominant form in adults. Researchers have suspected that delivering the vaccine via other routes of administration, such as inhaled aerosolized vaccine or IV, could improve efficacy. In this study, researchers used rhesus macaque monkeys to compare the potency of intravenous needle injection to aerosolized and intradermal routes of vaccine delivery. Remarkably, nine of the ten monkeys that received the intravenous injection were highly protected from exposure to TB six months after vaccination—and six of the monkeys showed no signs of exposure. local institutions. DNDi and MMV will provide frameworks and resources for drug discovery, including MMV’s database of molecules.A new study has found that delivering the BCG (bacille Calmette-Guérin) tuberculosis (TB) vaccine intravenously better protected monkeys against infection than the standard administration method of injecting it into the skin. The BCG vaccine, which is the only licensed TB vaccine, is effective in preventing early-childhood forms of TB but provides limited protection against pulmonary TB, the dominant form in adults. Researchers have suspected that delivering the vaccine via other routes of administration, such as inhaled aerosolized vaccine or IV, could improve efficacy. In this study, researchers used rhesus macaque monkeys to compare the potency of intravenous needle injection to aerosolized and intradermal routes of vaccine delivery. Remarkably, nine of the ten monkeys that received the intravenous injection were highly protected from exposure to TB six months after vaccination—and six of the monkeys showed no signs of exposure.Researchers at the Institut Pasteur and the Centre national de la recherche scientifique have discovered a new route for developing antimalarial drugs. In studying the Plasmodium parasite and its ability to adapt between host environments in humans and mosquitoes throughout its life cycle, the researchers realized they could exploit this adaptability. The parasite uses a process called DNA methylation to modify which genes are expressed during different stages of its life cycle in humans and mosquitoes. The researchers tested 70 methylation-inhibiting molecules and found many that were effective, including some that killed the parasite within hours. The researchers also found that the most promising molecules were able to kill artemisinin-resistant malaria strains. This research could lead to new treatments in the future against treatment-resistant malaria strains.

This week, months of tense negotiation between leaders in the Democratically-controlled US House of Representatives, the Republican-held Senate, and the White House concluded with the congressional passage of two massive spending packages encompassing all federal government spending for fiscal year (FY) 2020 —nearly three months behind schedule. Two continuing resolutions, passed by Congress to allow additional time for the process to play out after the start of the new fiscal year on October 1, have kept the government operating at FY19 levels, while from week to week, it was anyone’s guess whether the stalemate over funding levels and contentious items like border wall funding could be broken to avoid another government shutdown.A long process to produce two very long billsIn November, the appropriations “cardinals”—chairs and ranking members of both chambers’ Appropriation Committees—announced they had reached a deal on overall spending for each of the 12 appropriations bills, figures known as 302(b)s. This marked a critical step in the negotiations, given that at the time the House and Senate passed a two-year budget deal over the summer, each chamber had been working off of different overall blueprint funding envelopes: The House had already passed its appropriations bills assuming much higher funding levels than were ultimately included in the deal, while the Senate was developing its bills under the more modest final caps.Once the gulf was bridged and the 302(b)s were set, the leaders and their staff worked quickly to develop final compromise appropriations bills, which they grouped into two “minibus” funding packages released on December 16: a “national security appropriations” minibus and a “domestic priorities and international assistance appropriations” minibus, which included the bills for Labor, Health and Human Services, and Education (LHHS) and State and Foreign Operations (SFOPS).Funding increases included for several key accounts The later minibus encompassing LHHS and SFOPS was a mixed bag for accounts that support global health research and development (R&D). In the LHHS division of the minibus, overall funding for the National Institutes of Health (NIH) was boosted 6.7 percent from FY19 enacted levels—including increases of 6.7 percent for the National Institute of Allergy and Infectious Diseases, 3.3 percent for the National Center for Advancing Translational Sciences, 3.4 percent for the Eunice Kennedy Shriver National Institute of Child Health and Human Development, and 3.4 percent for the Fogarty International Center. The joint explanatory statement accompanying the division also notes “an increase of no less than $25,000,000 over the fiscal year 2019 level for HIV and AIDS research” at NIH.The Centers for Disease Control (CDC) saw even greater overall growth than NIH, with an increase of 8.7 percent; however, GHTC-priority accounts that finance global health R&D were mostly flat-funded. While the Center for Global Health (CGH) received a 16.8 percent bump, the entirety of the increase went to Global Public Health Protection—which funds global health security activities—and a new Global Tuberculosis line—which represents level funding of an amount previously transferred annually from a domestic tuberculosis (TB) line to support global TB activities. All other CGH lines were flat-funded. The National Center for Emerging and Zoonotic Infectious Diseases got a modest 1.6 percent bump. The final GHTC priority account in the LHHS bill, the Biomedical Advanced Research and Development Authority (BARDA), was flat-funded.The SFOPS division of the minibus increased overall funding for Global Health Programs at the State Department and US Agency for International Development (USAID) by 2.9 percent. This is slightly lower than what had been proposed in the Senate SFOPS bill earlier this fall and significantly lower than the House proposal to increase by 5.2 percent, but overall still a positive trend reflecting strong support for global health among appropriators, who were working under the constraints of a modest overall SFOPS allocation in the final agreement. At USAID, most health area-specific allocations were flat-funded or increased by about two to four percent—the big exception being the Global Fund for AIDS, Tuberculosis, and Malaria, which was increased by 15.6 percent to reflect the higher pledge made by the US government at the Global Fund’s international pledging conference in October.Report language signals policy support for global health R&DGHTC successfully advocated for the inclusion of several sections of report language, included in the original House and Senate SFOPS bill reports, that will hold under the final agreement. Report language serves as guidance from Congress to US agencies on how it would like to see funding implemented. Language that will hold for FY20 includes GHTC-supported language on USAID’s important role in and reporting on health-related R&D and inter-agency collaboration and coordination on global health product development, as well as support for “CDC’s continued and enhanced work in research and development aimed at creating new tools to respond to health threats at home and abroad” and BARDA’s contributions to emerging infectious disease outbreaks and other naturally occurring threats.The “domestic priorities and international assistance minibus” not only included some positive funding increases and report language, but also advanced a bill supported by GHTC—the End Neglected Tropical Diseases (NTD) Act, which supports R&D activities by USAID to control and eliminate NTDs.The House passed both packages Tuesday afternoon, with the Senate following suit Thursday and now the bills are awaiting signature by the President. GHTC applauds Congress for their strong continued support for global health R&D evidenced by the final deal and looks forward to building on this foundation for our advocacy as we enter a new decade full of opportunities for global health innovation.

This blog was authored by Marissa Chmiola, Ansley Kahn, and Julien Rashid.Two thousand nineteen was a tumultuous year for the global health community. From a major reorganization of the World Health Organization (WHO), to steps forward and backward in quashing the smoldering Ebola epidemic, to welcome approvals of new desperately needed health tools, 2019 took us on a topsy-turvy ride.Though the landscape may be constantly shifting under our feet, we at GHTC feel heartened by the clear evidence of our community’s shared progress and impact—and we remain emboldened by our belief that science serves and science saves.So, as the year comes to a close, we are taking a look back at the top global health innovation stories and news that shaped 2019. We hope they will energize and inspire you just as they have energized and inspired us.1. A breakthrough new treatment is approved for drug-resistant tuberculosisIn August 2019, a breakthrough new drug, pretomanid, was approved by the US Food and Drug Administration (FDA) as part of a revolutionary new treatment regimen for extensively drug-resistant tuberculosis (XDR-TB)—the most difficult-to-treat form of the disease. XDR-TB has a treatment success rate below 40 percent, but the BPaL regimen promises to dramatically improve this historically grim prognosis. In clinical trials, it successfully treated 89 percent of patients with XDR-TB. The regimen is also drastically shorter and simpler for patients. It’s taken orally and lasts just six months, compared to prior treatments which can involve injections and take up to two years.The BPaL regimen includes bedaquiline, pretomanid, and linezolid. Bedaquiline and linezolid have been included in older regimens, but this is the premier of pretomanid, which is only the third new compound in the last 40 years approved by the FDA to treat TB. Developed by TB Alliance, BPaL brings new ammunition to the global fight against this ancient, intractable killer, which today claims more lives than any other infectious disease.2. New tools bolster the fight against EbolaThough the Ebola outbreak continued to smolder in the Democratic Republic of Congo (DRC) in 2019, with ongoing conflict hampering response efforts, new innovations brought increased firepower and renewed optimism to the fight. In November, Merck’s Ebola vaccine Ervebo —which has been used on an emergency basis throughout the current outbreak—became the world’s first licensed Ebola vaccine after the European Commission granted it marketing authorization. Shortly thereafter, WHO prequalified it, paving the way for the vaccine’s use in countries at high risk. Meanwhile, a second experimental vaccine produced by Johnson & Johnson, which requires two doses, was introduced in the DRC alongside Ervebo to complement response efforts.On the treatment side, final data from a landmark clinical trial of four experimental Ebola therapies in DRC showed that two of the drugs dramatically reduced the risk of dying from the disease, especially in those who started treatment quickly after the onset of illness. The two treatments, which have also been used on an emergency basis in the outbreak, led to a survival rate of about 65 percent in treated patients compared to 33 percent in the overall outbreak. While there is still work to be done to quash this devastating disease, it is truly incredible that while five years ago we had no vaccine or treatment for Ebola, today, we can say Ebola is both a preventable and treatable disease.3. The world commits to achieving Universal Health Coverage, recognizing R&D’s roleThis September, in a rare moment of unity, the world came together at the United Nations to commit to achieving universal health coverage (UHC)—the concept that everyone, everywhere should have access to quality health care services and products without financial hardship. It’s a pledge that holds promise to radically transform the health and lives of millions around the world. Within this declaration of political will, we were pleased to see world leaders recognize the role research and development (R&D) plays in driving UHC. Many people think to deliver quality health care, we simply need to make sure everyone has access to existing tools. But in reality, we are still without essential technologies to combat many diseases and conditions impacting the world’s poorest; and where tools do exist, they are often not suitable for all types of people and locations. It’s clear to actually deliver the “quality” and “all” in “quality health for all,” we must intensify investment in health R&D.This year’s declaration represents a milestone moment in this quest, but it also marks the start of a mission, not its end. We now hope world leaders will back up their promise with concrete action and financing to realize this bold, shared goal. The health of our world depends on it.4. World’s first malaria vaccine rolled out in sub-Saharan AfricaEarlier this year, the world’s first approved malaria vaccine was introduced in Malawi, Ghana, and Kenya as part of a large-scale pilot program in which 360,000 children annually will be vaccinated. The vaccine, RTS,S, is the first, and to date only, vaccine to significantly reduce malaria in children. RTS,S took more than 30 years and more than US$500 million to develop, and is the result of an international collaboration between GSK, PATH, WHO, US government agencies, other research partners, and a network of African countries. In large scale trials, the vaccine prevented 4 in 10 cases of the disease. RTS,S is a promising new tool in our arsenal to combat malaria, and used alongside other interventions, has the potential to make a substantial contribution to malaria control efforts in sub-Saharan Africa and save tens of thousands of children’s lives.5. A new science division is established at WHOThis year, WHO announced wide-ranging reforms to the organization’s structure intended to help it achieve its “triple billion” targets—including one billion more people benefiting from UHC, one billion more people better protected from health emergencies, and one billion more people enjoying better health and well-being. Among the reforms is the creation of a new Science Division, headed by Chief Scientist Soumya Swaminathan, formed “to strengthen WHO’s core scientific work and ensure the quality and consistency of WHO’s norms and standards.” The new Science Division will serve as the primary hub coordinating R&D activities across WHO and set the innovation agenda for the organization. Though there remains some ambiguity on how this new division will interact with other WHO programs, including the prequalification program and essential medicines and diagnostics list functions, which currently sit outside the Science Division’s structure, this reform creates fresh opportunity to strengthen WHO’s work to accelerate new health technologies from first regulatory approval to widespread availability to people in need worldwide.6. Funding for neglected disease R&D reaches all time highAccording to the 2018 G-FINDER Report, which was launched in January this year, we learned that global funding for neglected disease R&D in 2017 reached the highest level ever recorded. This increase is cause for celebration as it will bring us one step closer to closing the R&D financing gap to meet the world’s greatest health challenges. At $3.56 billion, the 2017 investment was $232 million—or 7 percent—higher than 2016 and the largest year-to-year bump in investment since 2009. It was also the first time that investment grew for two consecutive years.Though other nations contributed most of this new growth, the United States government continued its leadership as the world’s largest funder of neglected disease R&D, adding a fresh $23 million. This bump happened despite the President requesting a fiscal year 2017 budget that hung a blade over global health and medical research programs. That this funding emerged uncut is a strong signal of continued congressional support for global health and medical innovation—even in a moment of deep political tumult and fierce budget battles.7. Race for a chikungunya vaccine shifts into overdriveChikungunya is a debilitatingly painful viral disease spread via mosquito, for which no cure or vaccine currently exists, but with several vaccine candidates now in clinical trials, this could soon change. The three frontrunner candidates are from Themis Bioscience, Emergent BioSolutions, and Valneva, each with clinical-stage candidates. Themis Bioscience raised funding in September from the private sector to launch phase 3 clinical trials of its candidate, MV-CHIK, in the next few months. Emergent BioSolutions is also preparing for phase 3 clinical trials with its candidate, CHIKV-VLP, after revealing positive phase 2 clinical trial results in November. Valneva’s candidate, VLA1553, completed a phase 1 clinical trial and has received funding from the Coalition for Epidemic Preparedness Innovations to further its development. All three frontrunners have received Fast Track designation from the FDA as of the end of 2019, but only time will tell which company might finish that track first.8. President signs two vital bills supporting global health innovationWith impeachment news dominating Washington as we head into the holiday season, you may be surprised to learn Congress and President Trump found some common ground in 2019 in a somewhat unexpected area—strengthening R&D for global health challenges. On January 3, the President kicked off the year by signing into law the Global Health Innovation Act, a bipartisan bill, championed by GHTC, to support efforts by the US Agency for International Development to develop affordable, appropriate technologies to advance global health.Later in June, President Trump signed the Pandemic and All-Hazards Preparedness and Advancing Innovation Act, a vital piece of legislation reauthorizing the work of the Biomedical Advanced Research and Development Authority (BARDA), an agency charged with developing medical countermeasures against both bioterror and naturally occurring health threats. BARDA is a critical piece of the architecture of US support for global health R&D, having advanced lifesaving products for infectious disease threats like Ebola and Zika in recent years; and this legislation, which GHTC influenced, reinforces the agency’s authority and mandate to pursue research against a range of naturally occurring threats, including emerging infectious diseases, pandemic influenza, and antimicrobial-resistant pathogens.9. Researchers pursue creative ways to combat mosquitoesIf there had to be just one antagonist in the grand narrative of human health, it would doubtless be the small, but lethal mosquito. After battling this tiny creature for millennia, it’s possible that in this century, humans might finally grasp victory. Two thousand nineteen brought fresh news of new potential tools to aid in this fight. From novel bednet treatments, to mosquito birth control, to radiation and genetic sterilization techniques, to new larvicides and techniques for their application, to next-generation technologies to improve R&D, it’s clear numerous breakthroughs are within reach, which wielded together, may signal the third act of this ancient drama.10. Drug and vaccine delivery get innovativeFrom the R&D news of 2019, it’s clear that one of the most flourishing frontiers in global health innovation is not only what vaccine or drug you could receive, but also how you will receive it. Delivery issues from maintaining vaccine cold chains to ensuring patients adhere to difficult treatment regimens have proven to be major barriers to care in the world’s poorest places. Thankfully researchers are pursuing creative and innovative ways to overcome these challenges.In the vaccine world, scientists are pursuing wild new ways to make vaccines easy and pain-free, including microneedle patch stickers and other needle-free instruments, as well as pursuing approaches to making them heat stable, like freeze-dryingor transforming them into a dissolvable film similar to breath strips. For HIV/AIDS, new technologies designed to aid patient adherence are expected to come to market soon, including a fruit-flavored drug formulation designed for children and a long-acting, monthly injectable antiretroviral treatment. In the future, taking your TB medicine and contraceptive, could be as easy as inhaling it or absorbing it by wearing your favorite necklace or earrings. Some of these ideas may seem crazy and fantastical, but if history is any indication, science so often finds a way.For other fascinating global health news delivered to your inbox weekly and to stay abreast of developments in 2020 and beyond, sign up now to receive GHTC’s R&D News Roundup newsletter.