Breakthroughs Blog: Medicines

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 led by US Food and Drug Administration researchers last week published findings suggesting that three vaccines, already approved and stored in the Strategic National Stockpile, targeting earlier H5N1 clades that circulated in the early 2000s could provide some cross-neutralizing antibodies against the currently circulating 2.3.4.4b clade, which continues to infect dairy herds and some farm workers across the United States. Federal officials are already implementing a plan to produce 4.8 million doses of an updated vaccine targeting the current clade, with the first doses expected to be delivered this month, but, before being administered to people, the shots would still need to get regulatory approval. These new study results offer hope that the old vaccines could provide some protection if there is a need to start vaccinating humans before the updated vaccines become available.Recently published results from a Phase 2 clinical trial conducted in Sudan by the Drugs for Neglected Diseases initiative and the Institute of Endemic Diseases at the University of Khartoum found promising results for a new and improved treatment for people with post-kala-azar dermal leishmaniasis (PKDL), a serious condition that can develop after treatment for visceral leishmaniasis. The current treatment for PKDL is an injectable drug given for two to three months, which can cause life-threatening toxicity and must be administered under close supervision in a hospital. The new treatment is safer, shorter, cheaper, more effective, and more patient-friendly, only requiring patients to be admitted to the hospital for 14 days followed by an oral treatment that can be completed at home, offering hope of a new and improved tool for PDKL for affected populations, like in Sudan where nearly 20 percent of patients treated for visceral leishmaniasis will develop PKDL within six months post-treatment, the highest rate worldwide.The Sabin Vaccine Institute and the Makerere University Water Reed Project recently launched a Phase 2 clinical trial testing Sabin's vaccine against Sudan ebolavirus, which can cause deadly disease, as seen in the recent outbreak in Uganda that ended only last year. Although Sabin’s vaccine was one of three candidates chosen to be included in a trial during the outbreak, the outbreak ended before the trial could begin. The vaccine candidate, which is based on the same platform as Sabin’s Marburg vaccine candidate that is already undergoing its own Phase 2 trial, was found to be safe and elicit rapid, robust, and long-lasting immune responses in Phase 1 clinical and nonclinical studies. There is currently no approved vaccine for Sudan ebolavirus.

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 Wednesday, the World Health Organization (WHO) announced that it had prequalified an at-home, over-the-counter assay for hepatitis C virus (HCV), the first self-test to be prequalified for HCV, which causes an estimated 1 million new infections globally each year. The prequalification of the assay, manufactured by OraSure Technologies, extends the 2017 prequalification of the company’s HCV test for professional use. Self-testing is recommended to complement existing testing services based on evidence demonstrating that it increases access to and uptake of services. The prequalification status will hopefully enable procurement in low- and middle-income countries, expanding access to HCV testing and treatment services and helping to contribute to the global goal of HCV elimination.The Safety of Antimalarials in First Trimester consortium, made up of scientific and social research experts on malaria in pregnancy has kicked off preparation for the first-ever Phase 3b clinical trial testing the efficacy and safety of antimalarials in women in their first trimester of pregnancy, which will be cosponsored by Medicines for Malaria Venture and the Liverpool School of Tropical Medicine. Pregnant women are typically excluded from clinical trials out of fear of causing harm, leaving few medicines for malaria treatment and no medicines for prevention available for women in their first trimester despite the significant health risks associated with malaria in pregnancy. The data from this trial could enable pregnant women and all women of childbearing age to safely take these antimalarial medicines without risk of causing harm, as well as support the design of future trials targeting other infectious diseases affecting pregnant women in low- and middle-income countries.  New research has led to the discovery of a potent adjuvant, PVP-037, that could induce a greater, more durable, and broader immune response to vaccines. Adjuvants are compounds that enhance the immune response, which are key to vaccine development, particularly for inactivated virus vaccines. Researchers identified the compound by screening a library of more than 200,000 small molecules found in human immune cells. In mice, an optimized form of the adjuvant boosted antibodies against flu and SARS-CoV-2 when given alongside disease-causing antigens. The compound is stable, easy to work with, and can be formulated in most drug delivery systems, which would make it easier to integrate it into vaccine development. Further research will be needed to study how the adjuvant performs in the presence of actual pathogens, assess its impact on the durability of immune responses, and test whether it works across age groups.

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 Global Health Innovative Technology (GHIT) Fund awarded a two-year grant to the Drugs for Neglected Diseases initiative (DNDi) and its partner Mitsubishi Tanabe Pharma Corporation to continue lead optimization work on a novel compound to target the Trypanosoma cruzi parasite that causes Chagas disease, a neglected parasitic disease that affects an estimated 6 million people globally every year. Current treatments for Chagas, which were developed decades ago, need to be administered for eight weeks, can have serious and potentially toxic side effects, and have not been proven effective in people with severe chronic symptoms. The partners hope their work will lead to preclinical development candidate compounds and, eventually, a new medicine for Chagas disease that is safe and effective for all patients, including pregnant individuals.Researchers have developed an experimental vaccine that has the potential to protect against a broad range of coronaviruses, including known and as-of-yet-unknown varieties. The experimental vaccine was made by taking harmless proteins shared across different coronaviruses and attaching them to minuscule nanoparticles that are then injected to prime the body’s immune system to broadly target both known and unknown viruses in the same family. The vaccine induced a broad immune response to coronaviruses in mice, including to the pathogen that caused the 2003 Sars outbreak, even though proteins from that virus were not added to the vaccine. If proven safe and effective in humans, this and other proactive vaccines designed to target separate pathogens could be stockpiled by countries along with clear plans to quickly scale up production if a new pandemic-potential pathogen emerges. The universal vaccine can be made in existing facilities for microbial fermentation, and the researchers are already working with industrial partners on how to scale up production. There would need to be new processes designed with medical regulators before approval because there are no current procedures for the regulation of proactive vaccinology.Last Monday, the Biomedical Advanced Research and Development Authority announced the launch of the next generation of its accelerator network, which will include a diagnostics and medical devices program and a therapeutics hub. The former will be called the International Consortium for Research, Engineering, Acceleration of Technology Excellence and will support the development and acceleration of new diagnostics and medical devices aimed at improving health security. Along with three more hubs expected to be announced later this year, these hubs will create a global network to foster the growth of the health security innovation ecosystem, including support across the entire process, from development and evaluation to validation and commercialization of new 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. As reports of H5N1 bird flu circulating in dairy cow herds across the United States continue, the experts in charge of preparing and responding to pandemics are now considering our ability to quickly develop a vaccine if human transmission were to occur in the future. The world has decades of experience producing, regulating, and updating flu vaccines, as well as the recent memory of the incredibly rapid development of vaccines during the COVID-19 pandemic. However, current global production capacity for flu vaccines would not yield enough doses quickly enough to vaccinate the large portion of the world’s population that would be necessary to quell a bird flu pandemic. Adjuvants—compounds that boosts immune response to the active vaccine antigen —or the introduction of an mRNA vaccine could, respectively, stretch supplies and speed up production, but it is not clear that there is sufficient production capacity for relevant adjuvants, and it is unclear how close producers are to advancing mRNA flu vaccines to market. Additionally, current flu production capacity is highly concentrated in high-income countries with no flu production in Africa, underlining the potential of another highly inequitable pandemic response.Last week Medicines for Malaria Venture (MMV) and Novartis announced positive data from their Phase 2/3 trial in Africa demonstrating the efficacy and safety of a novel formulation of the Coartem® malaria medicine developed for babies weighing less than 5 kilograms. Previously, this population lacked access to an evidence-based treatment option developed specifically for them, with this trial becoming the first evidence-based trial conducted to evaluate a new antimalarial dose and regimen for all infants under 5 kilograms with acute uncomplicated malaria. This population is currently treated with tablets meant for higher-weight children that are simply adjusted for weight, which can lead to overdose and toxicity in smaller kids. The data have been submitted for regulatory review, and if approved, the partners aim to make the treatment available as soon as possible.The Drugs for Neglected Diseases initiative (DNDi) and partners have initiated a Phase 2 clinical trial in Ethiopia testing an innovative, safer, simpler, and patient-friendly medicine to treat visceral leishmaniasis, the world’s deadliest parasitic killer after malaria. Visceral leishmaniasis is currently treated with painful injections that must be given at the hospital daily for 17 days and can cause rare but life-threatening side effects. The hope is that the new drug, which is administered via oral pills, will be efficacious, less toxic, and more accessible than the currently available treatment regimen, ensuring earlier access to treatment. A similar Phase 2 trial is also being conducted in South Asia to test if patients in the two endemic regions respond differently to the treatment.