Breakthroughs Blog: NTDs

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 Drugs for Neglected Diseases initiative (DNDi) and AN2 Therapeutics announced a new collaboration to advance clinical development of AN2’s oral drug candidate for the treatment of chronic Chagas disease, which can remain asymptomatic for years while the infection wreaks irreversible damage in the body, potentially leading to the development of life-threatening conditions. The drug candidate, AN2-502998, is currently being tested in a Phase 1 first-in-human clinical study, scheduled for later in 2025, with a Phase 2 proof-of-concept study expected to commence next year. The partnership will leverage DNDi’s established network of clinical trial sites throughout Latin America and Spain, established through its previous Chagas clinical trial work.A team of researchers shared last week that they have developed a special kind of floss that could serve as a vaccine delivery method, delivering proteins and inactive viruses to the gums and triggering immune responses that can protect against infectious disease. While scientists have long been investigating alternatives to syringes like oral and nasal vaccine delivery methods, a challenge they’ve come across is that the mouth and nose have naturally tough defenses against foreign molecules. The flossing approach overcomes this challenge by targeting the pockets of gum between the teeth that are better at absorbing molecules. The approach proved successful in mice in multiple tests where researchers coated the floss in protein and inactive flu virus, and researchers have also tested the feasibility in humans, finding positive results. Further research will help determine if the technique will work in humans with a wide variation in gum health, including people with gum disease.Study results presented at the recent International AIDS Society Conference in HIV Science found that more than half of people living with HIV who received a pair of broadly neutralizing antibodies plus the drug N-803 experienced delayed viral rebound or maintained a low or suppressed viral load during interruption from antiretroviral treatment. While antiretroviral treatment can keep HIV suppressed indefinitely, the long-lasting viral reservoir maintained in the body has confounded researchers efforts to eliminate it, putting a cure out of reach. A small proportion of individuals naturally make unique broadly neutralizing antibodies that target key parts of the virus, which researchers have been trying to replicate with a manufactured drug. N-803, which is approved as a treatment for bladder cancer and is being studied in both cancer and HIV research, appears to be able to both reactivate dormant HIV and clear the virus out of hiding. Further research and an analysis across studies testing the approach will help confirm the underlying mechanisms of the effects researchers are seeing and determine whether the drug could be part of a future cure.

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 new study looking at data from two separate Phase 1 clinical trials shows that a targeted vaccine strategy can successfully activate early immune responses relevant to HIV, a significant advance in longtime efforts to develop an HIV vaccine. A persistent challenge in advancing HIV vaccines has been finding a way to train the immune system to produce antibodies that target many variants of the tricky, rapidly mutating virus. The study, conducted by an international team of scientists from IAVI and Scripps Research, included nearly 80 participants from North America and Africa, helping ensure a future vaccine has global potential. One of the trials used a stepwise vaccination strategy, administering two doses sequentially to guide the immune system to develop antibodies, while the second trial found that an initial dose could successfully activate the desired immune cells. Both vaccines used an mRNA vaccine platform, enabling faster production and clinical testing.A recently published study found that a new water-soluble formulation of the only drug available to treat helminths, or parasitic worms, can achieve the same effect as the currently available tablet form at half the usual dose. Praziquantel is currently available as large tablets that are bitter and difficult to swallow, especially for children and pets. The new formulation can be dissolved in water and was shown to be more efficiently absorbed, enabling patients to receive a smaller dose without sacrificing efficacy. Further clinical testing is needed before the new formulation can be brought to market, but if it continues to demonstrate success, the new formulation could be a game-changer in addressing helminths, which affect 400 million people worldwide.

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 new study published in The Lancet Infectious Diseases ahead of World Malaria Day last week found that adding a low dose of the widely available antimalarial drug primaquine to artemisinin-based combination therapy is safe and effective at blocking Plasmodium falciparum malaria transmission in young children in areas of low- and moderate-to-high transmission. The new study fills a gap in knowledge of the safety and efficacy of this approach for young children, who face the highest burden of clinical malaria and are particularly at risk of artemisinin-resistant strains but could face adverse complications from adult formulations of primaquine. The findings highlight the need for child-friendly formulations of primaquine and support the expanded use of the drug in regions with a moderate-to-high transmission rate and the threat of artemisinin partial resistance.The Drugs for Neglected Diseases initiative, the National Health Service University Hospitals of Liverpool Group, and the University of Liverpool have initiated a clinical trial in Liverpool, United Kingdom to test an oral drug for visceral leishmaniasis, one of the world’s deadliest parasitic diseases. Current treatments for visceral leishmaniasis, which is almost always fatal if left untreated, include daily injections for up to 17 days, which can cause immediate pain, as well as nausea, vomiting, and more serious complications. The new oral drug, DNDI-6899, is easier to store, transport, and administer than current treatments. If proven successful, the drug could help make treatment more accessible to those who need it most.DioSynVax and ACM BioLabs have signed a new deal to jointly develop a next-generation avian flu vaccine with the potential for mucosal vaccine delivery, as the virus continues its worrying spread. The mRNA vaccine targets all major clades of the H5 avian flu subtype and could be scaled for rapid distribution globally in the case of a pandemic. The vaccine is also designed to be delivered via nasal spray, which could help increase uptake; to induce mucosal immunity, which helps fight the pathogen before it starts circulating the body; and to be temperature stable, enabling rollout in lower-resource settings.

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 research team at the Children's Hospital of Philadelphia has discovered a key process that could provide new insight into designing more effective antimalarial drugs, as rising antimicrobial resistance complicates the ongoing global fight against malaria by rendering current drugs less effective. One promising strategy to combat poor absorption or retention of drugs in the body is the use of prodrugs that can offer a more targeted attack. However, these prodrugs need to be activated, typically by an enzyme. By identifying the primary enzyme that activates multiple antimalarial drugs, these new findings could enable the development of prodrugs for malaria, which, by design, would also be less likely to lead to the emergence of drug resistance.New research shows that wastewater sampling, including in sewer systems and on airplanes, could predict new viral and bacterial outbreaks and the emergence of new variants of pathogens weeks or even months before they become widespread, in addition to being less invasive, more efficient, and faster than current methods. A new study from the US Centers for Disease Control and Prevention demonstrates how it was able to use wastewater sampling to detect the presence of avian flu in 2022 weeks before the virus was detected in poultry and then wild bird populations. Widespread and regular wastewater sampling would enable public health authorities to better surveil population and community health and, on a more micro level, help health care providers provide more accurate diagnoses and care for patients.  Last week, the Pediatric Praziquantel Consortium announced the first administration of its new pediatric schistosomiasis treatment to a preschool-aged child in Uganda, marking a critical step toward closing the long-standing treatment gap for young children for this parasitic disease, which affects millions of children in sub-Saharan Africa. The innovative therapy, designed specifically for children between three months and six years, is also child-friendly, palatable, and heat-stable, making it especially well-suited for this population and region. The rollout is part of a program aimed at integrating the treatment into health care systems and preparing for its broader rollout across the region, starting with Côte d’Ivoire, Kenya, Tanzania, and Senegal.