This is exciting news for us. Meghan has the form DF508.
This new medicine will potentially put her form of CF into a remission stage.
CF Foundation Announces Initiation of Phase 2 Trial of VX-809
March 25, 2009 The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis—a faulty gene and its protein product, called CFTR. Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a “corrector” and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would. The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients. "Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We are encouraged by Vertex’s rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF.” "The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."
VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.