Research Roundup: Vaccines without chemicals, FDA fast track for Vanelva's chikungunya vaccine, and Marburg virus found in bats in West Africa

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 at the University of Texas Medical Branch have developed an experimental, single-dose drug that has shown to successfully protect non-human primates against all known strains of the Ebola virus. "Our experimental drug can protect against all forms of Ebola known to harm people, suggesting that it will continue to protect people if the Ebola viruses evolve over time," noted scientist Thomas Geisbert. In contrast to many other therapeutics developed to protect against Ebola, a single-dose, broadly effective drug could provide protection against the virus quickly and efficiently during an outbreak while reducing the burden on health care workers in the field. Further studies could explore the efficacy of lower doses of the drug, which could allow for easy-to-administer treatment via auto-injectors.Researchers at the University of Arizona have taken a major step forward in developing a “mosquito birth control” drug—which targets a unique protein in female mosquitoes—to help curb the spread of malaria and other mosquito-borne diseases. This unique protein is critical for a female’s offspring to hatch and when blocked causes defective shells, leaving the embryos inside to die. Developing a drug that targets this specific protein could provide a way to reduce mosquito populations without harming other insects, such as bees. One scientist noted he hoped this discovery could lead to the development of new insecticides within the next five years, which could be applied to bednets and used in anti-mosquito sprays. Researchers at the Royal College of Surgeons in Ireland have developed an experimental new treatment for tuberculosis (TB), which is administered using an inhaler. The treatment—which reduces the bacteria in the lungs that causes TB while helping the patient’s immune system fight the disease—uses all trans retinoic acid (atRA), a derivative of Vitamin A that has been shown as an effective treatment for TB. To deliver the treatment, researchers packaged atRA into safe-for-consumption particles small enough to use in an inhaler. The particles significantly reduced TB-causing bacteria and associated lung damage, which supports the treatment’s potential for clinical testing.

Two thousand eighteen was a turbulent year in global health. Between reemerging crises, breakthrough scientific discoveries, and the continued looming threat of health research funding cuts, 2018 took us on a roller coaster ride of peril and promise. Now as the year comes to an end, GHTC is taking a moment to reflect on the global health innovation stories and news that shaped 2018.Here are the highlights from our year in review:1. A long-neglected disease sees a breakthroughA patient suffering from sleeping sickness visits a hospital in DRC. PATH/Georgina GoodwinIn 2018, a revolutionary treatment emerged for a neglected tropical disease, sleeping sickness. The first all-oral drug for sleeping sickness, developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, received a positive scientific opinion from the European Medicines Agency—paving the way for the treatment to be rolled out in endemic countries in 2019. This groundbreaking treatment, known as fexinidazole, is a tablet taken once daily for ten days that treats the most common form of sleeping sickness, a disease spread by tsetse flies, which causes neuropsychiatric symptoms and can be deadly if left untreated. Current treatments for sleeping sickness require a complicated series of infusions and pills, are costly, and involve long hospital stays. Fexinidazole is a simple once-a-day pill that could reduce the logistical burdens to treating a disease for which approximately 65 million people in sub-Saharan Africa are at risk. 2. TB captures the spotlight as new research demonstrates promiseThe world’s largest infectious disease killer finally received some well-deserved attention this year as world leaders convened the first-ever United Nations High-Level Meeting on Tuberculosis, at which member states committed to closing the US$1.3 billion annual gap in funding for tuberculosis (TB) research. While time will tell whether this moment will prove a powerful catalyst or an empty promise, the science continues to advance as we wait for the politics to play catch up. This year we saw several exciting new developments in TB research. Aeras and GlaxoSmithKline released the results of a phase 2b clinical trial that showed their candidate vaccine prevented TB from developing in half of those who received it. If it succeeds in additional testing, this vaccine would represent an improvement over the existing vaccine in use and would be the first new TB vaccine developed in over a century. On the treatment side, TB Alliance and partners launched the SimpliciTB trial, to test a new all-oral drug regimen for treating both drug-sensitive and drug-resistant TB, known as BPaMZ, which could slash treatment times for TB by one-third or more and is projected to be markedly less expensive than current therapy. Additionally, TB Alliance was honored this year by GHTC with an Innovating for Impact Partnership Award for the development of child-friendly TB medicines, which are now available in 86 countries and counting.3. A single dose cure for malaria is approvedA patient in Zambia receives a malaria diagnostic test. PATH/Gena Morgan This year, the global health community witnessed regulatory approval of the first new treatment for Plasmodium vivax malaria in 60 years by the US Food and Drug Administration (FDA). The new drug, tafenoquine, is a single-dose treatment developed by GlaxoSmithKline and Medicines for Malaria Venture. P. vivax malaria causes around 8.5 million infections every year in Asia, Latin America, and parts of Africa. It’s tricky to combat because the P. vivax parasite can lie dormant in the liver, where it reactivates, and can cause new episodes of malaria weeks, months, or even years after the initial infection. In a study of the treatment, 60 percent saw no relapse six months after taking the drug. Tafenoquine has been submitted for approval to the Australian regulatory authority and is slated to be submitted for approval in malaria-endemic countries. Experts hope this new treatment will accelerate the path to elimination of this mosquito-borne disease.4. Innovations show promise while the battle against Ebola rages onOne of the most disheartening stories of 2018 was the reemergence of Ebola in the Democratic Republic of Congo (DRC). Ongoing conflict in the region has hampered response efforts, with the outbreak now reaching 500 confirmed cases. Yet, amidst this crisis, there is one reason for optimism: An experimental vaccine appears to be helping those it reaches. Close to 45,000 people have received the experimental rVSV-ZEBOV Ebola vaccine—the largest use of the product since a clinical trial in 2015 showed it worked. While the vaccine’s effectiveness during this outbreak has not been formally assessed, World Health Organization (WHO) officials believe it is having a major impact. The vaccine’s developer, Merck, announced last month that it has begun the process of submitting evidence to seek FDA approval, a crucial step to bring the vaccine to market. Meanwhile, new preliminary research also suggests experimental treatments deployed during the outbreak are improving survival rates, and researchers have begun clinical trials in eastern DRC to test the effectiveness of four of these experimental drugs. This research raises new hope that science may soon offer us both a vaccine and treatments to combat Ebola.5. Congress pushes back against cuts to global health and medical researchWhile the Trump Administration proposed in its fiscal year (FY) 2018 and 2019 budgets to slash funding for global health and medical research programs, in the end Congress pushed back. In March, Congress passed the FY18 omnibus, which protected, and in many cases increased, funding to key US agencies and programs that advance global health research and development (R&D). In September, Congress passed an FY19 spending bill covering the Defense, Labor, Health and Human Services (LHHS), and Education departments. The bill boosted funding for medical research across the Department of Health and Human Services, with the National Institutes of Health receiving a $2 billion increase and the US Centers for Disease Control and Prevention and the Biomedical Advanced Research and Development Authority also receiving modest increases.These funding bills represent a forceful rebuke by Congress of the Administration’s proposals to draw back support for global health and medical research programs and demonstrate that Congressional policymakers on both sides of the aisle want to see the US continue its leadership in advancing innovation to improve the health of those in need around the world. Given that funding for the US Agency for International Development (USAID) was maintained through a continuing resolution that expires on December 21, we now await further action by Congress to determine how FY19 funding for global health accounts at USAID will shake out.6. New vaccines give us a better shot at saving livesA healthworker in Laos prepares a vaccine. PATH/Aaron Joel Santos Two thousand eighteen was a year of innovations in vaccines. In what could be the first new polio vaccine in more than 50 years, researchers have developed a way to freeze-dry an inactivated poliovirus vaccine (IPV) so that it can be stored at higher temperatures and reconstituted for use without losing its potency—potentially offering a new tool to help eradicate the disease. In other vaccine news, Johnson & Johnson (J&J) unveiled promising early-stage results for a tetravalent mosaic-based HIV preventative vaccine regimen. Currently in large-scale clinical trials in sub-Saharan Africa, the vaccine marks only the fifth time in history an HIV vaccine candidate has advanced to human clinical trials, offering new hope for a breakthrough in HIV prevention. Additionally, WHO prequalified new vaccines to combat typhoid and rotavirus, which is the most common cause of severe diarrheal disease in children worldwide. In January, WHO approved a new, highly effective and low-cost typhoid vaccine for global use. In the same month, it prequalified Rotavac, an oral rotavirus vaccine, and later in the year, approved Rotasiil, a first-of-its-kind thermostable vaccine to prevent rotavirus.7. Innovators strive to put greater choice in the hands of patientsOne of the exciting themes we saw across so many global health stories this year was the power of innovation to put greater choice in the hands of patients. People have unique needs, and our toolbox of global health technologies must reflect that. Several scientific developments this year promise to expand family planning choices for women and their families. The first-of-its-kind, self-insertable and removable one-year contraceptive vaginal ring received regulatory approval from the FDA; new research from Uganda showed a self-injectable form of contraception can help women overcome barriers to consistent use; and the first clinical trial was launched for a male contraceptive gel, a technology that could bring more equality to the burden of family planning. In addition, researchers are also advancing long-acting injectable forms of medicines to provide an alternative for individuals who struggle to take frequent pills. For example, HIV-positive patients may soon be able to choose whether they want to use long-acting injectable antiretroviral treatments or daily pills as a form of Pre-Exposure Prophylaxis, and scientists are also working on injectable formulations of medicines for malaria prevention. 8. Scientists pursue wild and weird ways to fight deadly diseases A dog in India. PATH/Gabe BienczyckiWinston Churchill once said, “No idea is so outlandish that it should not be considered.” That’s clearly a motto researchers have taken to heart, as evidenced by the number of stories we read this year about unusual and creative ways scientists are trying to fight infectious diseases. Here’s a sample of some of our favorites. One could say it’s a bacteria-eat-bacteria world out there, with researchers studying whether germ-eating microbes could be used to combat drug-resistant infections. Man’s best friend may also be man’s diagnostic tool, according to new research showing that dogs can be trained to detect malaria in people. In the future, administering a vaccine could be as easy and pain-free as putting on a Band-Aid if microneedle patches in development come to fruition. The contents of your takeout dinner might be the next frontier in HIV prevention, as scientists have developed a technique to modify a strain of rice to produce HIV-neutralizing proteins. While these ideas may seem fantastical, history has taught us that groundbreaking discoveries can come from unusual places. For other fascinating global health news delivered to your inbox weekly and to stay abreast of developments in 2019 and beyond, sign-up now to receive GHTC’s R&D News Roundup newsletter.