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HEALTH 2007, October 16   Cystic fibrosis: The molecule of hope     The team directed by Frédéric Becq, a physiology professor at Poitiers University, has discovered a molecule that could potentially allow cystic fibrosis patients to lead a normal life. In late September, initial clinical trials took place in Spain. “For the time being, we don’t know how to treat the gene responsible for cystic fibrosis”, emphasizes Professor Becq. That is why, along with his team at the physiology and cellular biology institute, his first objective has been to treat the damaged protein produced by this defective gene. Through its 2005 discovery of a molecule known as Miglustat that is capable of regenerating the protein, a laboratory belonging to both the French CNRS and Poitiers University has rendered genuine hope to the many patients suffering from a rare disease that was long ignored by the large-scale pharmaceutical industries. “Concretely speaking, Miglustat comes and blocks the enzyme deteriorating the protein, which will resultantly be able to regain its place in the cell and assume its role in the transport of chloride, sodium and water”, explains Frédéric Becq. While treatment based upon this molecule would not cure cystic fibrosis, it would nonetheless prevent overproduction of the mucus causing obstruction of the different canals of an organism’s secretory glands; this obstruction first appears at the digestive and then, quite rapidly, at the respiratory level. A drug for 2011? A drug may be hitting the market as soon as 2011; that said and as of now, so indicates Professor Becq, “failure remains possible”. The date may sound surprisingly soon; after all, it generally takes ten to fifteen years for a molecule to be given a concrete application. The short time lapse is explained by the presence of Miglustat in treatment of another rare pathology, Gaucher’s disease. “This has allowed us to gain time by avoiding Phase 1 toxicological examinations on the animal”, the researcher notes. The first clinical trials on humans began in Spain on September 28th. Conducted by Actelion, the Swiss pharmaceutical laboratory developing a drug for Gaucher’s disease, these tests shall determine the actual efficacy of the molecule on the 25 patients having been selected. At the outset of 2009, if matters proceed apace, a large-scale Phase 3 trial will get underway. In France, one newborn baby out of 200 is affected by cystic fibrosis, and at this time, 6000 people suffer from the disease; they may be said to invest limitless hope in Professor Becq’s team. Though avowedly confident, when mentioning this discovery he “hedges his bets”. Having worked on cystic fibrosis since 1993, he is well aware of the fact that “in the event of failure”, there would be “enormous disappointment”. 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