PATH receives GHIT Fund grant to expand drug research for soil-transmitted helminths

Contact:
Elena Pantjushenko, 415.341.4140, epantjushenko@path.org.

Seattle, March 31, 2016–PATH announced today a two-year, US$1,002,996* grant from the Global Health Innovative Technology Fund (GHIT Fund) in Japan to develop a new treatment for soil-transmitted helminths (STH) infections. PATH will lead the international partnership that includes two Japanese companies–Meiji Seika Pharma Co., Ltd. and Ajinomoto Co., Inc.–and the University of Massachusetts Medical School to pursue the development of the protein Cry5B as a new therapeutic option for STH management.

More than 880 million children are in need of treatment for STH worldwide, with the heaviest burden placed on the poorest and most deprived communities with poor sanitation. Transmitted by roundworm and whipworm eggs, and hookworm larvae, infections can cause diarrhea, abdominal pain, general malaise and weakness, and chronic blood loss. STH infections can cause mortality, but their greatest impact is thought to come as a result of morbidity associated with anemia and malnutrition, which lead to physical and cognitive stunting.

"Infections caused by intestinal parasites are considered among the most pressing of global health problems, thought to parasitize some 2 billion people worldwide–nearly half of them children," said Dr. David Shoultz, who leads PATH's Drug Development program. "Further development of Cry5B will help us investigate its full potential as a valuable tool to help reduce the burden of STH infections and protect children worldwide."

The drugs currently used to treat STH infections as part of mass drug administration campaigns, such as albendazole and mebendazole, are threatened by the rise of resistance. A significant gap in the profile of all of the currently used anthelmintic drugs is that they are contraindicated for women in their first trimester of pregnancy, leaving the mother and her fetus vulnerable to the effects of STH-induced anemia and malnutrition and out of reach of a therapeutic intervention.

Cry5B is a powerful, naturally occurring anthelmintic protein from the soil bacterium Bacillus thuringiensis. It has demonstrated efficacy in animal models, and its safety profile could permit its use in young children and women during their first trimester of pregnancy. There is also evidence that resistance to the protein would be developed more slowly compared to current treatments, which could allow for decades of intensive use.

Supported by the investment from GHIT, the partnership's effort over the next two years will focus on 1) the development of appropriate microbial strains to express Cry5B, 2) the development of appropriate fermentation processes for growing Cry5B-expressing strains, and 3) research of needed processes for the purification of Cry5B from the production strains.

The combination of a Cry5B-based product with other existing anthelmintic drugs could result in further improved efficacy and spectrum, protect the therapeutic agents from the development of resistance, and support the global health strategy to expand the reach of anthelmintic mass drug administration campaigns to reduce the global burden of STH.

PATH's Drug Development program works to develop and ensure availability and accessibility of safe and effective new medicines for diseases disproportionately affecting people in developing countries. For more information, please visit http://sites.path.org/drugdevelopment/.

The first of its kind in Japan, the GHIT Fund is a public-private partnership between seven Japanese pharmaceutical companies, the Japanese Government, the Bill & Melinda Gates Foundation, the Wellcome Trust, and the United Nations Development Programme. Launched in April 2013 with an initial commitment of more than US$100 million and now with capital of approximately US$140 million, the organization taps Japanese research and development to fight neglected diseases. The GHIT Fund invests and manages a portfolio of development partnerships aimed at neglected diseases that afflict the world's poorest people. GHIT mobilizes Japanese pharmaceutical companies and academic and research organizations to engage in the effort to get new medicines, vaccines, and diagnostic tools to people who need them most, with Japan quickly becoming a game-changer in global health.

For more information, please visit www.ghitfund.org.

* All amounts listed at the exchange rate of USD1 = JPY100.