Mode of action of candidate drugs for treatment of Cystic Fibrosis
Disease-modifying drug discovery for the treatment of Cystic Fibrosis including translational aspects to patients.
Cystic Fibrosis (CF) is a genetic disease (more than 100.000 patients worldwide) and is caused by a defect in a protein (CFTR). Each protein in our body is a chain of amino acids that must be properly folded in the cell to perform optimally. That folding of the CFTR protein is a complicated process. In many patients with CF this folding process is impaired and therefore an effective protein cannot be produced. This is disrupting the salt and water resources in all cells that produce mucus and hence tough slime is created.
Recent research results show that the basic defect in the cells in CF can be tackled with medicines. Because the current medications are insufficiently working or only working for a group of patients with specific abnormalities in the genetic material (mutations), Galapagos is one of the companies developing candidate medicines to target these basic defects.
Researchers from Utrecht University Department of Chemistry, Pharmaceutical Company Galapagos and the Nederlandse Cystic Fibrosis Stichting (NCFS) have jointly performed a project to better understand the disease mechanisms of Cystic Fibrosis and the mode of action of new candidate drugs being developed by Galapagos, in order to bring better drugs faster to patients with CF. By using the techniques that have been developed by the Lab of Professor Ineke Braakman to study the mode of action of the Galapagos candidate drugs, this project has gained much more insights into the basic defect in the cell at different mutations, how candidate medicines of Galapagos are acting thereon and how the cells and auxiliary proteins respond to them.
These results shall be used in the further development of the best candidate medicines by Galapagos and other companies. In already granted follow-up projects the same consortium partners will further unravel the mechanism of the folding of the protein and how to improve this in order to contribute to the ultimate objective: a cure for people with CF.