The remarkable progress of VX-809 is a reminder of why it is so important to remove barriers that prevent people with CF from participating in clinical trials. Passing the “Improving Access to Clinical Trials Act of 2009” will allow people with CF who are on public benefits to participate in important research without losing their health coverage. We continue to work with Congressional supporters to chart the best path forward for the bill. There are two options to pass the bill:
Option #1: On its own
Non-controversial, bi-partisan and no-cost legislation like the clinical trials bill may be considered under special procedural rules. Thanks to your hard work, 110 members of the House, and eight members of the Senate have signed on as co-sponsors, meeting the requirements to provide the bill the opportunity to advance on its own.
Option #2: As part of a health care reform bill
Despite the shift away from health reform in past weeks, our sponsors – several of whom are key negotiators on health reform – continue to consider it as an option and the best opportunity to move the bill sooner rather than later.
Read more about what H.R. 2866 would do.
The bill has two viable opportunities because you made it clear to your members of Congress that this is a priority for you and should be for them as well. Ask your members of Congress to keep the "Improving Access to Clinical Trials Act" on their agenda until it passes.
Right now, because of eligibility rules regarding compensation for clinical trial participation, many people with CF are forced to choose between taking part in important clinical trials and keeping their health care coverage.To help promising drugs move swiftly from the research and testing phase to the people who need them, more people with cystic fibrosis are needed to participate in clinical trials. This important legislation will remove that barrier to allow more people to do so.
CF Investigational Drug VX-809
Shows Encouraging Results in Phase 2a Trial
February 3, 2010
Vertex Pharmaceuticals Incorporated announced today results from a Phase 2a trial of VX-809, an oral investigational drug that aims to correct the basic defect in cystic fibrosis. VX-809 was found to be well-tolerated and to reduce sweat chloride levels — a key indicator of CF.
The 28-day, Phase 2a trial of VX-809 examined the drug in cystic fibrosis patients who have the Delta F508 gene mutation, the most common mutation in CF. The study focused primarily on the safety and tolerability of the drug and changes in sweat chloride.
A reduction in sweat chloride levels in the Phase 2a data suggests that VX-809 may improve the function of CFTR, the faulty protein in CF.“These are very exciting and important results in our effort to find ways to treat the basic defect in cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “The VX-809 data further supports our hypothesis that small molecules can be used to treat the underlying genetic cause of cystic fibrosis.”
The data from this trial pave the way for future studies of VX-809, including testing the therapy in combination with VX-770. Also developed by Vertex, VX-770 is an oral investigational drug that showed encouraging Phase 2 results in restoring the function of CFTR in patients with the G551D mutation of CF.
Research in the laboratory suggests that using two therapies in combination may increase CFTR function in cells with the Delta F508 mutation when compared to using a single therapy alone.
The first trial examining VX-809 and VX-770 in combination in cystic fibrosis patients is currently in the planning phases, and is expected to begin in the United States in the second half of 2010.
In addition, Vertex is further reviewing the Phase 2a data and may explore the option of studying VX-809 as a single drug in higher doses in a separate clinical trial.
Through its Therapeutics Development Program, the CF Foundation collaborated with Vertex to discover and develop VX-809 and VX-770, investing approximately $76 million in the effort. This represents the largest single investment in CF drug discovery by the Foundation.
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