On June 6, key stakeholders came together in Seattle to introduce the GHIT Fund and celebrate its achievements. Among numerous special speakers, the Gates Foundation’s Trevor Mundel, President of Global Health, and Ken Duncan, Deputy Director, Drug Discovery, shared their perspectives and reflections.

Dr. Mundel, who serves on GHIT’s Council, recalled his first introduction to GHIT in 2011, when Tachi Yamada introduced the idea of creating the GHIT Fund in Japan. “For the Gates Foundation,” he said, “this was a great opportunity to enlist Japan in the global health architecture. GHIT launched in 2013 and since then has evolved with an extremely strong portfolio.”

Importantly, he added, GHIT’s partnership with the Japanese government and industry set an example for other countries’ engagement in global health R&D. He noted that “It’s entirely due to GHIT that we have the RIGHT Foundation in Korea. It's rare to create effective new organizations in global health, especially in science investments, but GHIT has succeeded.”

Dr. Duncan, a member of GHIT’s Selection Committee, described the story of one of the GHIT Fund’s key successes – the development of a potential new treatment option for schistosomiasis in preschool-aged children, recently pre-qualified by the World Health Organization (WHO). The journey began in 2013 with the Pediatric Praziquantel Consortium's proposal to address schistosomiasis in preschool-aged children. Schistosomiasis affects over 240 million people globally, including an estimated 50 million preschoolers in Africa.

The 600 mg tablet of the standard-of-care treatment is used as the first-line treatment for schistosomiasis; this treatment is safe, effective and suitable for school-aged children and adults.  However, an appropriate drug for preschoolers children has been elusive. The Pediatric Praziquantel Consortium was formed to address this treatment gap. Summarizing the scientific challenges and opportunities the consortium grappled with, Duncan explained that the original treatment is a mixture of two enantiomers – as in left- and right-handed forms – but only the left-handed form is active, and it was thought that pure L-PZQ (levopraziquantel, the biologically active PZQ enantiomer) is less bitter than the mixed version. Reformulation was still not straightforward as children's metabolism and pharmacokinetics differ from adults, requiring dose adjustments.  A full clinical program informed the development path and confirmed safety and efficacy in preschoolers, including final dosage.

Led by Merck (Germany), the public-private partnership currently has 14 partners and collaborators, including Japanese pharmaceutical company Astellas, and public institutions in endemic countries and in Europe. Duncan explained that Astellas was a key founding partner, responsible for a large part of the product development work.

The first challenge was producing the pure, left-handed version of the standard treatment on a large scale. Eventually, Astellas developed a child-friendly, taste-masked tablet that can be taken whole or dispersed in water, and is stable in high temperatures and humidity—critical, Duncan noted, for the environments in which it would be used. A suitable dose was selected, and Phase 2 and 3 studies confirmed its effectiveness. In December 2023, the European Medicines Agency granted the positive scientific opinion to the new pediatric treatment option for preschoolers (aged 3 months to six years); in May 2024, this was included in the World Health Organization (WHO) list of prequalified medicines.

GHIT’s long-term support at every stage, including supporting work looking at new procurement access mechanisms and deliverability of the drug has been essential, Duncan reflected. “What this really illustrates is how a consortium can come together involving multiple partners, each of which brings different skills, all of which are essential to making the project successful.”

From left to right: Dr. Trevor Mundel, President of Global Health, Bill & Melinda Gates Foundation and Dr. Ken Duncan, Deputy Director, Drug Discovery, Bill & Melinda Gates Foundation