Marissa Chmiola

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.

Faces of Innovation—a new GHTC project that features scientists on the front lines of research and development on new global health tools and technologies—profiles Hannah Wu, who we met at the American Society of Tropical Medicine and Hygiene (ASTMH) Annual Conference, who is working to develop vaccines against the neglected tropical disease schistosomiasis at Brown University.My Name: Hannah WuWhere I work: Brown University Medical School in Providence, Rhode IslandI’m funded by: NIHMy research: We do research on neglected tropical diseases, for malaria and schistosomiasis…I’ve been working on schistosomiasis for a long time. I am lucky enough I can not only work in the lab, but also go to the field to see people. Even though schistosomiasis is more neglected than malaria, there are people in the world suffering from this disease. It’s caused by a trematoda—this is a worm. If we have a vaccine for it, that might be much better to cure the disease or to prevent the disease. So that’s what we are working at. We look at immune mechanisms from the population, and we are trying to find vaccine candidates. For the shistosomisis species, that I am specifically working on…it is also a one health strategy that we should use….We hope that we will have more funding to do other experiments and research on this vaccine, to be available in the livestock.For parasitic diseases, animals are a very important reservoir host. Some people are in touch with animals every day, in their daily life. So we are not only making vaccines for the humans, we can actually make vaccines for, in my case in schistosomiasis, for water buffalo which account for 70 to 90 percent of transmission. So that’s the concept of one health.Motivation: When I was in graduate school, I was not very clear what I wanted to do, but I think I was lucky enough, I got to see the people who had the disease…Because of their everyday life, they have to be in contact with the pathogen, so they cannot really avoid to have the disease, and they don’t have other means….Research has a curiosity I always like. And I was curious about finding new things…but if at the same time I can…contribute to this world by finding a vaccine to help people, that would be great. So that motivates me.Why federal support is critical: We are human beings. We’re not isolated…We care about other people, so that’s one basic reason we can do this research for the world, to improve human health. And I really believe that everyone deserves an opportunity to get health care and to live a more healthy life.When I’m not in the lab: I am involved with my church a lot. Through the church I work with young people. I tell them science is about curiosity. We have the privilege to do science, so we should do real science for real people. We’re not doing science just for the sake of science, we’re doing science for the betterment of human life.

Faces of Innovation—a new GHTC project that features scientists on the front lines of research and development on new global health tools and technologies—profiles Dr. Heinz Weidenthaler, who we met at the American Society of Tropical Medicine and Hygiene (ASTMH) Annual Conference, who developed a smallpox and monkeypox vaccine with Bavarian Nordic.My name: Heinz Weidenthaler, MDWhere I work: Bavarian Nordic, which is headquartered in Denmark and has offices in Morrisville, North CarolinaI’m funded by: Biomedical Advanced Research and Development Authority, National Institutes of Health, Department of Defense, Centers for Disease Control and PreventionMy research: I am a medical doctor by training working for a company called Bavarian Nordic…We have done quite an extensive program in developing a smallpox and monkeypox vaccine with support of several federal agencies of the US, mostly BARDA, who has funded most of our clinical trials and helped us really tremendously throughout the development of this product. There were other contributions, NIH NIAID [National Institutes of Health National Institute of Allergy and Infectious Diseases] provided support in specifically running several of the clinical trials that finally led to licensure of the product. The Department of Defense was incredibly helpful in running the key pivotal phase III trial, via USAMRIID [US Army Medical Research Institute of Infectious Diseases]. We also have a collaboration with CDC [Centers for Disease Control and Prevention], who is using our product in a study right now in the Democratic Republic of Congo [DRC].It is intended to be a safer alternative smallpox vaccine, which means it can protect the public, of course, both in America, but also on a global basis. Smallpox is a global threat, not a national threat at a given country…We received US licensure not only for smallpox, but also for monkeypox. There the global impact really comes in because the current epidemiology of monkeypox is endemic in several African countries like the DRC and Nigeria. This is something that, there will be an impact as soon as it is able to be deployed there. This is running right now with the CDC study in DRC, that could really save lives by preventing infections in these areas.Motivation: I’m a medical doctor by training and joined the pharma/biotech industry quite a while ago, and instead of just working on another blood pressure pill that would be generic a few years later, I think working in the pox virus vaccine sphere really has an impact on saving lives. And doing that with the help of government funding, in terms of a really successful public private partnership here, I think is a tremendous success, and being part of that, and helping in the clinical development of our vaccine is really a motivating thing because it just has an impact of peoples’ lives.Why federal support is critical: Global health is affecting all countries, including the US. Last year, we had these cases from Nigeria, people who were bringing cases of monkeypox, transmitting them to the UK, to Singapore, to Israel. The world is so globally connected, and infectious diseases that are endemic in sub-Saharan African countries can be anywhere in the world 24 hours later. I think this has a really huge impact on being prepared for all scenarios, and is of critical importance for the health of individuals worldwide, including the US.When I’m not in the lab: I take care of my family. I have three kids back home. I try to be there as much as possible, which is not always easy, but I enjoy the time back home with my kids. I try to do some kinds of sports as far as I find the time for that. I play volleyball quite frequently.

Faces of Innovation—a new GHTC project that features scientists on the front lines of research and development on new global health tools and technologies—profiles Dr. Geoffrey Siwo, who we met at the American Society of Tropical Medicine and Hygiene Annual Conference, who develops mathematical models to accelerate product development at the University of Notre Dame in Indiana.My Name: Geoffrey Siwo, PhD.Where I work: University of Notre Dame in Notre Dame, IndianaI’m funded by: National Institutes of Health and National Science FoundationMy research: My research is in developing computational methods that impact the way we do science, especially with respect to global health….Basically the goal is to ensure that we can accelerate the way new medicines are brought into the market for diseases like malaria, and so this is why our project is to arrange computation methods, such as artificial intelligence that can have a rapid impact on the way we translate some of the science findings intro practical applications for global health….We believe that artificial intelligence can cut down the time that is needed to do this. One aspect [of our research] is to understand drug resistance, especially for malaria. So for the case of malaria where there is resistance to [the malaria drug] artemisinin, we are inviting data scientists to develop computational models that predict the mechanisms of the resistance and that will help in potential surveillance of resistance.Motivation: I am from Kenya originally, and I am motivated to do this research because malaria is a big problem in Africa and the only way to make a difference is to develop the therapies that directly address the disease.Why federal support is critical: This research has the potential to affect millions of lives. We know that disease has no barriers, and the solutions that we develop for diseases in any part of the world can have an impact on any other part of the world, including the US.When I’m not in the lab: I like reading some novels, especially regarding future technology and the future of the world.

Faces of Innovation—a new GHTC project that features scientists on the front lines of research and development on new global health tools and technologies—profiles Dr. Mark Kortepeter, who we met at the American Society of Tropical Medicine and Hygiene (ASTMH) Annual Conference, who does research on emerging infectious diseases at the University of Nebraska in Lincoln.My name: Mark Kortepeter, MD, PhD.Where I work: University of Nebraska in LincolnI’m funded by: Assistant Secretary of Preparedness and Response and Centers for Disease Control and Prevention.My research: I’m an infectious disease and public health physician and I work for the University of Nebraska Medical Center. I lead what’s called the special pathogens research network, so this is a network, we have ten centers across the country, that are tied together as a research network for response to outbreaks, especially ones that are related to high consequence pathogens like Ebola. What we realized after the 2014/2016 outbreak [of Ebola], where there were patients coming into the US, was that there were a number of different investigational products that were used to treat the patients. We realized in the end that we really didn’t understand what was beneficial and what wasn’t in terms of the treatment because they were all given as individual investigational products. So the whole goal was to have a network that was tied together so we can learn more if this happens again—whether it’s Ebola or some other disease like Nipa virus or some novel influenza—so at the end of an outbreak, we would actually be able to compile data, be working off the same sheet of music, off the same protocol, to essentially advance science in understanding what works best for patients.If there are novels products that are still investigational, we would track with those—so we have some Ebola countermeasures that we have been working with…as well as if there was something new that popped up and a need to study something in real time, we could do that. In addition, if there is something for which there is no therapy, we also have the ability to follow the natural history of the disease and what clinical interventions also seem to be useful.Motivation: I got into this because I had a travel bug, and I liked the idea of doing work in infectious diseases because there is a travel component with it because you’re dealing with exotic diseases across the world. So that’s what really got me into it. I just found it fascinating working with people of other cultures.Why federal support is critical: It’s important to invest in this because we really don’t know where the next outbreak will come from…It’s possible that something will come out of left field and surprise us. So it’s very important to have some preparedness in mind, such that you can be ready for anything that comes. If you really think about it, we were all caught up with Ebola in 2014/2016, and then suddenly there is Zika virus, or we were surprised when West Nile showed up on the US shores. So we have to have a global mindset, we have to understand what’s going on outside the country as well as what can land on our shores, because we just never know what’s going to happen next. We’re all kind of one global world now in terms of infectious diseases. Infectious diseases have no boundaries or borders.When I’m not in the lab: The thing I’m really excited about right now is that I wrote a book about my experiences dealing with bioweapon defense when I worked at Fort Detrick, Maryland at the US Army laboratory there…It’s called “Inside the Hot Zone: A Solider on the Frontline of Biological Warfare.” It just came out this week. It’s written like a medical thriller, but it’s also very educational, and it gives an insider’s perspective on things like the anthrax attacks, preparation for Ebola outbreaks, dealing with Ebola in the laboratory, things like that. 

One of the great things about working at GHTC is that every day we get to read stories about incredible innovations in global health. Most of the technologies we read about are inventive, some are revolutionary, and a few are so unusual, one might think they were ripped from the pages of a science fiction novel. So, to enjoy a bit of fun and mischief this Halloween, we’ve created a new game, TRICK or TREATment, to test our readers’ knowledge of global health technologies. For each question, we will present a description of a global health technology. Choose TREATment if you believe this innovation exists and has market approval. Choose TRICK if you think we are trying to pull one over on you and, instead, the product is still in development or just a figment of our imagination.  Loading... (function(i, s, o, g, r, a, m) { var ql = document.querySelectorAll('A[quiz],DIV[quiz],A[data-quiz],DIV[data-quiz]'); if (ql) { if (ql.length) { for (var k = 0; k