Paris/Geneva – Clinical trial results presented in Paris at the Union World Conference on Lung Health today showed that a once-daily dose of a widely available antibiotic over six months cut a child’s risk of developing multidrug-resistant tuberculosis (MDR-TB) by more than half. Funded by global health organization Unitaid and led by Stellenbosch University, the research is the first of its kind to investigate how to prevent this dangerous form of TB in children who suffer some of the most severe outcomes from the disease.
MDR-TB is a form of the disease that has developed resistance to two or more of the first-line drugs used against it, making treatment particularly lengthy and difficult to complete. Because only two in five people access appropriate treatment and even fewer get cured, the disease continues to spread. Children are at particularly high risk, often becoming infected through close contact with an adult with the disease. Each year, an estimated two million children become newly infected with MDR-TB, which, left untreated, can develop into active disease.
Though several existing treatment regimens can prevent the progression to TB disease in both children and adults, evidence to-date into MDR-TB prevention has been limited since no randomized controlled trials had previously been conducted. The TB CHAMP clinical trial recruited nearly 1,000 healthy children who had been exposed to MDR-TB in their homes in communities with high burdens of TB and MDR-TB across South Africa.
“Children often wait years for an adult medicine or intervention to be adapted to their needs,” said Dr Philippe Duneton, Executive Director of Unitaid, the leading multilateral funder of TB research and development for children, which backed the development of the first and many subsequent TB medicines for children. “This research is remarkable because, if the approach is recommended by the World Health Organization, not only do we have the high-quality evidence to protect children from the most dangerous form of TB, but we have already developed a fruit-flavored, dispersible form of the treatment for children – a critical but frequently neglected last step in ensuring children will take the medicine.”
“Once it progresses to active disease, multidrug-resistant TB treatment is long and harrowing for children and their families to endure. This research provides high-quality evidence to cut that risk in half. But what’s more, in avoiding difficult treatments, we found the intervention to be cost saving for national health systems,” said Professor Anneke Hesseling, Director of the Desmond Tutu TB Centre and the Principal Investigator of the TB-CHAMP trial, at Stellenbosch University.
Because just 8% of all public funding for TB research and development is dedicated to children’s specific needs, research to advance solutions for children typically lags years behind adult interventions. However, the TB-CHAMP results were presented alongside the V-QUINN trial, which investigated a similar intervention for adults. Both pieces of research are expected to guide World Health Organization (WHO) recommendations for preventing MDR-TB in children and adults anticipated in the coming months. If the WHO issue a recommendation, it could be the first time in the history of TB that a child intervention is developed concurrently to an adult treatment.
Additionally, because child medicines require additional research into adequate dosing, and need to be adapted to make them palatable and easy for children to swallow, the Unitaid-Stellenbosch University program worked in parallel to develop a child-friendly formulation which is already available for purchase. This often-overlooked element of children’s care is particularly critical in TB, as a child’s refusal to swallow bitter pills could leave the door open to the development of serious disease. This research, coupled with child medicines means that, if recommended, optimal MDR-TB prevention for children could be rolled out without delay – though funding is urgently needed to support the contact tracing necessary to find children at risk.
This news comes alongside another major advance to MDR-TB treatment, presented at the Union Conference yesterday. The endTB clinical trial, funded by Unitaid and led by Partners in Health, Médecins Sans Frontières and Interactive Research and Development, found three new, all-oral, shortened regimens for drug-resistant TB that were safe and effective across all populations – including children, pregnant women, and people with common comorbidities. A fourth regimen showed to be a valuable alternative for patients who cannot tolerate the key drugs that are included in nearly every current World Health Organization-recommended regimen for MDR-TB.
These regimens could provide a much-needed complement to the currently recommended shorter treatment regimen, which is highly effective but not suitable for all groups. And because the drugs used in the endTB regimens are already registered and widely available, they could quickly translate into new alternatives for care for countless people with DR-TB, pending WHO review.
“With more than 400,000 people newly falling ill with MDR-TB each year, we need safe, effective treatment options for every patient. We are extremely hopeful that the four treatment regimens from the endTB clinical trial will help ensure that all populations can access high-quality, shortened treatment,” said Carole Mitnick, ScD, Partners In Health Director of Research for the endTB project, Co-Principal Investigator of the study, and Professor of Global Health and Social Medicine at Harvard Medical School.
The endTB trial enrolled more than 750 patients from Georgia, India, Kazakhstan, Lesotho, Pakistan, Peru, and South Africa. It included historically excluded populations like adolescents and those with comorbidities like substance-use disorders and retained participants who became pregnant during the trial.
Together, the two studies represent major advances in the treatment and prevention of MDR-TB. These additional therapeutic options should greatly reduce sickness and death in all people at risk of this most deadly form of TB.
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