PATH Drug Solutions

PATH’s Drug Development Global Program grew from our affiliation in late 2011 with the nonprofit drug development group OneWorld Health. We’re working to discover, develop, and deliver safe, effective, and affordable new treatments for diseases disproportionately affecting people in the developing world. By combining entrepreneurial energy, pharmaceutical expertise, and an innovative product development partnership model, we challenge the assumption that pharmaceutical research and development is too expensive to create the new medicines that the developing world desperately needs.
Descriptions of a few of our projects follow. Learn more about our initiatives on the OneWorld Health website(http://www.oneworldhealth.org/) .
Targeting diarrheal diseaseAn estimated 800,000 children under five years of age die every year from diarrheal disease, making it the second most common killer of young children after pneumonia. Survivors who are inadequately treated suffer long-term health problems. With about 2 billion cases of diarrheal disease globally every year, the world has an urgent need for safe, effective, and affordable treatments.
We are working to address the burden of cholera and other diarrheal diseases. In 2011, we received approval from the US Food and Drug Administration for phase 1 clinical trials of an investigational new drug called iOWH032 to treat acute secretory diarrhea resulting from diseases like cholera. This first-in-class synthetic drug is designed to be used in conjunction with oral rehydration therapy, providing faster relief of diarrhea symptoms and encouraging wider adoption of and compliance with the therapy. The drug candidate is scheduled to enter phase 2 trials in 2012.
We continuously evaluate new treatment opportunities for infectious diarrheal disease and establish partnerships to move forward promising candidates for development. Currently, we are collaborating with Anacor Pharmaceuticals to find antibacterial compounds for treating bloody diarrhea (shigellosis) and with the Center for World Health and Medicine to identify potential anti-secretory drug candidates.
Ensuring the supply of malaria treatmentMalaria is a life-threatening disease transmitted by mosquitoes infected with parasites of the genus Plasmodium. Every year, malaria infects an estimated 216 million people, causing 655,000 deaths—most of them young children in sub-Saharan Africa. With proper surveillance and care, malaria can be prevented and treated.
Artemisinin-based combination therapy is the gold standard of malaria treatment. However, the supply of artemisinin derived from plants can be unstable, resulting in shortages and high cost of therapy.
To meet this challenge, we have formed a partnership with synthetic biology innovator Amyris Inc. and leading French pharmaceutical company Sanofi to develop a semisynthetic form of artemisinin. The synthetic biology technology is based on pioneering inventions licensed from the University of California, Berkeley and the National Research Council Plant Biotechnology Institute (Canada).
The partnership, known as the Artemisinin Project and supported by the Bill & Melinda Gates Foundation, aims to create an additional source of nonseasonal, high-quality, affordable artemisinin to supplement the current botanical supply, making artemisinin-based combination therapy more accessible to the hundreds of millions of impoverished people who contract malaria each year.
In 2011, after successful completion of the scientific work necessary to enable production of semisynthetic artemisinin, the Artemisinin Project entered the production scale-up phase. Integration of semisynthetic artemisinin into the supply chain and therapy is expected in the next few years.
Treating and controlling kala-azarKala-azar, also called visceral leishmaniasis or VL, is a potentially fatal infectious disease that is transmitted through the bite of a sand fly. It affects the visceral organs, causing chronic fever, weight loss, and anemia. VL is endemic in 79 countries, primarily in the developing world; the population at risk is estimated at 200 million. If left untreated, VL is nearly always fatal. The disease causes 20,000 to 40,000 deaths each year.
We developed the paromomycin intramuscular injection (PMIM) to be a safe, effective, and affordable treatment for VL. A full course of treatment costs less than US$20. PMIM was designated by the World Health Organization for inclusion on its Model List for Essential Medicines and has been approved for the Essential Drug Lists of Bangladesh, Ethiopia, India, Nepal, Sudan, and Uganda. PMIM is currently registered in India, Nepal, and Uganda.
In collaboration with other global health partners, we are establishing new methods for controlling and treating VL. In 2011, we joined forces with the Drugs for Neglected Diseases initiative and the Special Programme for Research Training in Tropical Diseases, on a major project aimed at establishing and implementing new treatment modalities as successful tools to work toward elimination of VL in South Asia.  Learn more about the project (423 KB PDF)(http://www.oneworldhealth.org/pdf/OWH%20role_Visceral%20leishmaniasis.pdf) .
We also work to ensure that the treatments we develop for VL are acceptable and available to patients at an affordable cost through private- and public-sector markets and health care systems. We expand access to drugs and diagnostics by conducting epidemiologic and market research to identify barriers to access and evaluating delivery models designed to overcome them.