Research Roundup: Once-a-month birth control pill, experimental typhoid fever vaccine, and release of 2019 World Malaria Report

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.

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.  Indian generic drug manufacturer Cipla and the Drugs for Neglected Diseases initiative have developed a new, palatable pediatric formulation of HIV medicines priced at US$1 day, which experts say could save the lives of thousands of children each year. The drug, called Quadrimune, contains four HIV drugs—ritonavir, lopinavir, abacavir and lamivudine—and comes in strawberry-flavored granules the size of grains of sugar that can be mixed with milk or sprinkled on baby cereal. The granules are also coated with a polymer that doesn’t melt until it hits the stomach, eliminating the need for cold transport or storage of the drug, which can be inconsistently available in rural areas, especially in sub-Saharan Africa. Quadrimune is still under review by the US Food and Drug Administration and, if approved, will likely lead to rapid certification by the World Health Organization. Each year, approximately 80,000 babies and toddlers die of AIDS in part because their medicines come in hard pills or bitter syrups that are very difficult for small children to swallow or keep down.Final data from a landmark clinical trial of four Ebola therapies in the current outbreak in the Democratic Republic of the Congo show two of the drugs dramatically reduce the risk of dying from the disease, especially in those who started treatment quickly after the onset of their illness. Findings of the PALM trial show that a monoclonal antibody cocktail called REGN-EB3, made by Regeneron Pharmaceuticals, and a single monoclonal antibody developed by the US National Institute of Allergy and Infectious Diseases called mAb114 led to a survival rate of about 65 percent in treated patients compared to 33 percent in the overall outbreak. The other two products tested in the trial, an antibody product called ZMapp and the antiviral drug remdesivir made by Gilead Sciences, did not perform as well, saving only half the patients who were treated with one of those two therapies. The PALM trial began November of last year but ended early in August of this year because an interim analysis showed REGN-EB3 and mAb114 were statistically better than ZMapp and remdesivir.When babies are born with HIV, starting treatment within hours to days is better than waiting a few weeks or months, which is the norm in many countries. The findings from a small study in Botswana, funded by the US National Institutes of Health, show that very early treatment limits how HIV takes root in a newborn’s body, shrinking the “reservoir” of the virus that hides out waiting to rebound in case the children stop taking their medication. The study also demonstrated that the earliest-treated children had more normal functioning of some key parts of their immune system. These findings could influence care in regions hard hit by the epidemic and offer clues as scientists pursue a cure. Though it has become routine practice to give pregnant women a cocktail of anti-HIV drugs to prevent mother-to-child transmission of the virus, it is estimated that 300 to 500 infants are infected every day in sub-Saharan Africa.