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Institute For OneWorld Health And Novartis Launch Innovative Collaboration To Discover And Develop Novel Therapy To Combat Diarrheal Disease

The Institute for OneWorld Health, the US-based non-profit pharmaceutical company that develops drugs for people with neglected infectious diseases in the developing world, today announced that it has launched a collaboration with global pharmaceutical leader Novartis to discover and develop a novel therapy for secretory diarrhea, a deadly disease that kills more than 1.6 million children in the developing world each year. OneWorld Health and the Novartis Institutes for BioMedical Research will join forces to discover drugs to treat infectious diarrhea via inhibition of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) chloride channel. Under the three-year agreement, Novartis is responsible for all research and associated costs and OneWorld Health is responsible for preclinical and clinical development that may result from the collaboration. Secretory diarrhea is the second leading cause of death in early childhood in developing countries. The pediatric death toll due to diarrheal illnesses exceeds that of AIDS, tuberculosis, and malaria combined. In poor countries, diarrheal disease is second only to pneumonia in causing the deaths of children under five years old. Every week, 31,000 children in low-income countries die from diarrheal diseases - approximately 4,500 deaths every single day. Therapy for infectious diarrhea today involves oral rehydration, effective if begun early enough in the course of illness. Because infectious diarrhea is caused in many cases by over-activation of the Cystic Fibrosis (CF) channel, a CF channel blocker could be a powerful addition to currently available treatments, directly reducing fluid and electrolyte loss. "We are delighted to forge this innovative partnership with Novartis in pursuit of a new therapy to treat one of the world"s most pervasive killers of children around the globe," said Dr. Richard Chin, M.D., OneWorld Health"s Chief Executive Officer. "OneWorld Health greatly appreciates Novartis" commitment to addressing unmet medical needs in impoverished communities and their willingness to dedicate their knowledge and expertise to the fight against childhood deaths from diarrheal diseases." Cystic Fibrosis and secretory diarrhea have a shared disease mechanism involving the Cystic Fibrosis Transmembrane conductance Regulator. CF is caused by mutations that stop the proper function of the CFTR protein that leads to a build up of mucus in the lungs that make it difficult to breathe. In secretory diarrhea, infectious agents or toxins can induce CFTR hyperactivity that results in excessive loss of water from the body via the gastro-intestinal tract. The loss of water leads to dehydration that can result in death if left untreated. Both parties bring expertise to bear. Through their extensive work in cystic fibrosis research, Novartis scientists have gained insights into the structure and function of CFTR, which will be applied to discovering a CFTR inhibitor for secretory diarrhea. OneWorld Health has worked in developing an anti-secretory therapy since 2006, with a $47 million grant from the Bill and Melinda Gates Foundation, based on their collection of compounds and based on research conducted at the University of California, San Francisco. Institute for OneWorld Health


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