Breakthroughs Blog: Child health

The Gates Foundation has awarded ProFound Therapeutics up to $35 million to find new drug targets for preeclampsia, a high blood pressure condition during pregnancy that can be severe for mother and child and is a significant contributor to maternal and fetal deaths globally, particularly in low- and middle-income countries. ProFound will utilize their vast repository of proteins and data to improve scientific understanding of how preeclampsia is developed in the body, with the goal of identifying promising approaches for future drug development. Once targets are identified, the company will explore their features using an AI tool and then evaluate them in laboratory experiments.Last week, the Coalition for Epidemic Preparedness Innovations (CEPI) announced that it will invest up to $16.5 million to advance the development of an African-developed vaccine candidate for the Bundibugyo strain of Ebola, which is behind the fast-growing outbreak in the Democratic Republic of the Congo. CEPI’s support will help advance the candidate through preclinical development and into an early-stage clinical trial in Africa. The vaccine candidate uses a technology that is utilized by a licensed vaccine against smallpox and mpox and has elicited long-term immunity in a vaccine targeting the Zaire strain of Ebola. It is the fifth Bundibugyo-specific vaccine candidate supported by CEPI in response to the current outbreak and the only of the five designed to induce two major parts of the immune system, both B- and T-cells, for comprehensive and long-lasting immunity.A novel tuberculosis (TB) vaccine candidate failed to match the current, more-than-100-years-old vaccine in preventing TB infections in newborns, according to the recently shared results of a Phase 3 trial conducted among 6,897 healthy newborns in sub-Saharan Africa. TB remains the world’s leading infectious disease killer and there is an urgent need for a new vaccine—the current TB vaccine, BCG, is effective at preventing some types of TB in infants, but it offers inconsistent protection in adults against pulmonary TB. One of 17TB vaccine candidates in the pipeline, the vaccine in the trial, VPM1002, is a genetically modified recombinant BCG vaccine developed by the Serum Institute of India. While disappointing, the results of the trial do provide significant insights about how best to design robust, feasible efficacy trials for future vaccine candidates.

Last Thursday, the World Health Organization (WHO) announced that, with support from Gavi, the Vaccine Alliance, it will allocate 70,000 doses of an approved Ebola vaccine to the Democratic Republic of the Congo, which is facing the largest Ebola outbreak in the country’s history. The vaccine, Ervebo, targets Ebola Zaire, the most common Ebola strain, not the Bundibugyo strain behind the current outbreak—for which there are no approved vaccines —but early laboratory and animal data suggest it may provide some cross-protection. Twenty thousand doses will be used in a Phase 3 trial to evaluate whether the vaccine offers cross-protection for Bundibugyo, while the remaining doses will be allocated to frontline health care workers, serving the dual goals of providing some protection for at-risk populations against severe illness and death during the current outbreak while gathering critical data to inform use of the vaccine in future Bundibugyo outbreaks.Researchers at the Walter and Eliza Hall Institute of Medical Research have developed a rapid blood test that could improve detection of iron deficiency, a widespread health challenge disproportionately affecting mothers and children in low-resource settings where access to conventional laboratory-based screening methods are limited. Using a finger-prick blood sample, the test provides results within 15 minutes without the need for specialized laboratory infrastructure and highly trained health care providers. It has demonstrated strong correlation with gold-standard laboratory methods used in high-income countries and includes a built-in reference line to make the results easier to interpret. The test is currently undergoing evaluation in sub-Saharan Africa.The Coalition for Epidemic Preparedness Innovations (CEPI) shared last week that it will dedicate $4.17 million to four research projects designed to assess whether additional vaccines currently or previously licensed against Ebola Zaire could offer some cross-protection against severe illness and death during the current rapidly growing outbreak of Bundibugyo Ebola. Specifically, the funding will go to teams at the University of California, Los Angeles in the United States; the Institute of Tropical Medicine in Belgium; the University of Oxford in the United Kingdom; and the MRC-University of Glasgow Centre for Virus Research in the United Kingdom. The research teams will use blood samples from vaccinated individuals to assess immune responses and potential cross-reactivity of antibody responses, helping establish and standardize antibody tests for Bundibugyo, inform interpretation of clinical trial results, and guide future strategies. These research initiatives will support trials during the current outbreak and complement ongoing efforts to develop Bundibugyo-specific vaccines. 

An international clinical trial led by the Global Antibiotic Research and Development Partnership (GARDP) seeking improved antibiotic treatments for newborn sepsis has expanded to India. Neonatal sepsis is one of the leading causes of newborn deaths worldwide and is becoming harder to treat due to rising antibiotic drug resistance. Newborns have long been underrepresented in antibiotic research, leaving health care providers without an evidence-based standard-of-care treatment. The lack of a standard treatment has in turn complicated neonatal sepsis research because there is no obvious treatment to use as a comparison in trials. The trial aims to test and rank multiple potential antibiotic combinations for neonatal sepsis.Last week, the World Health Organization announced that a clinical trial evaluating two potential antiviral therapeutics for the Bundibugyo strain of Ebola will be launched in the Democratic Republic of the Congo next week amid the ongoing outbreak. There are currently no approved vaccines or therapeutics for the Bundibugyo strain, which is only known to have caused three outbreaks in history. The trial will evaluate the ability of Mapp Biopharmaceutical’s MBP134 and Gilead Sciences’ remdesivir to reduce mortality in patients, alone and in combination. The United States and Gilead Sciences are donating all the doses that will be used in the trial.The US Food and Drug Administration (FDA) has approved the first generic version of rifapentine as part of combination treatment regimens for active and latent pulmonary tuberculosis (TB). Rifapentine was first approved by FDA in 1998 to treat pulmonary TB. For active pulmonary TB cases in patients 12 or older, the generic antibiotic is approved only in combination with one or more anti-TB drugs, while for latent TB cases, the generic is approved in combination with isoniazid to treat patients 2 or older at high risk of progressing to TB disease.

A new US National Institutes of Health-funded study conducted in seven high tuberculosis (TB)-burden countries found that a new and improved near point-of-care TB test platform was highly accurate, effective, and scaleable. Pluslife Biotech’s MiniDock MTB test platform is battery operated, low-cost, provides results in 30 minutes, and can be used with tongue swabs in addition to sputum samples, which certain groups, including babies and people living with HIV, cannot produce. The new test is well-suited for low-resource, smaller, or more rural health clinics, which may not have access to current resource-intensive, expensive molecular testing options. The University of Oxford and the Serum Institute of India have signed a license agreement to support the development, large-scale manufacturing, and potential future commercialization of the malaria vaccine candidate R78C for use in clinical settings. R78C combines two Plasmodium falciparum blood-stage antigens, designed to target the malaria parasite at multiple points in its lifecycle, offering longer and more effective protection. This new license, which builds on a long-standing collaboration between the partners on malaria vaccine work, will initially help accelerate the candidate into clinical evaluation. The Serum Institute of India’s global manufacturing capabilities will also help ensure that resulting successful malaria vaccines can be manufactured at scale and deployed affordably to malaria-endemic countries. The Medicines for Malaria Venture (MMV) and Novartis recently announced that the World Health Organization (WHO) has prequalified Coartem® Baby, the first and only antimalarial drug developed specifically for newborns and young infants weighing between 2 and 5 kilograms. This treatment will help address a long-standing gap in malaria care for this group, who are often treated with antimalarial formulations intended for use in older children, which can increase the risk of toxicity or underdosing. The prequalification designation enables countries and global procurers to start purchasing and deploying the treatment in malaria-endemic countries; Novartis is making the treatment available on a largely nonprofit basis in malaria-endemic regions.