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. 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.

Malaria is one of the world’s oldest and deadliest diseases. Thanks to international efforts to develop and scale up prevention tools and treatments, significant gains have been made over the past few decades in fighting this dangerous disease. But challenges, such as growing drug and insecticide resistance, climate change, and disruptions from COVID-19, have hampered control efforts. While both malaria cases and deaths steadily declined from 2000 to 2019, since then, cases have been on the rise and reported deaths have not returned to pre-pandemic levels—indicating progress against the disease has stalled and underscoring the need for continued research and development of new tools.In recognition of World Malaria Day, test your knowledge of this deadly disease and the innovations being advanced to end malaria:

Neglected tropical diseases (NTDs) are a group of infectious diseases that exert a crushing burden in vulnerable communities around the world. These diseases can blind, disable, and disfigure people, robbing them of health and opportunity and trapping them in poverty. While breakthroughs in recent years have brought new resources to the fight against NTDs, significant gaps remain in the arsenal of tools needed to control and eliminate these diseases. In recognition of World NTD Day on January 30, GHTC is testing our readers’ knowledge with nine questions about NTD innovation. See if you can beat our quiz below, and join the fight to #BeatNTDs and #EndTheNeglect.