Aim is to develop a CFTR ion channel blocker to help prevent fluid loss.
Novartis and the Institute for OneWorld Health signed a three-year collaboration to develop a treatment for secretory diarrhea that targets the cystic fibrosis transmembrane conductance regulatory (CFTR) chloride ion channel. Novartis will carry out all research and assume associated costs, with OneWorld Health having responsibility for subsequent preclinical and clinical development.
OneWorld Health has been working on an antisecretory diarrhea therapy since 2006, funded by a $47 million grant from the Bill and Melinda Gates Foundation. The work was based on research carried out at the University of California, San Francisco. The new collaboration will now take advantage f Novartis’ knowledge in the area of CFTR structure and function.
In cystic fibrosis, the CFTR gene mutation leads to a buildup of mucus in the lungs, but in secretory diarrhea the infectious agents or toxins induce CFTR hyperactivity that results in excessive loss of water from the body via the gastrointestinal tract. OneWorld believes that because infectious diarrhea is often caused by over-activation of the CF channel, an inhibitor could represent an effective addition to currently available treatments, which are based on oral rehydration, by helping to reduce fluid and electrolyte loss.