The Beginning of the End?

by on February 3, 2010
in CFTR, quality of life, Vertex

It’s been a weird day.  I had planned to write about meditation and why having a “lung disease” doesn’t mean you can’t meditate.  This was something I used to actually believe, since beginning meditators are often instructed to “follow the breath.”  I tried this…I really did!  It only made me completely anxious and sure that I was suffocating!

I was going to wax prolific on how it is actually easy to use other objects to “anchor” the mind, and how wonderful it is to start each day with a relaxing (usually) and centering meditation practice, without even thinking about the breath.

Then, as I always (sadly) do before I started to write, I checked my email.  There I saw the following press release , and I literally lost my breath.

Vertex Announces Results from Phase 2a Trial of VX-809 Targeting the Defective Protein
Responsible for Cystic Fibrosis

-VX-809 was well-tolerated at all dose levels when dosed once daily for 28 days-
-Statistically significant changes observed in measurement of sweat chloride suggest increased CFTR
activity-
-Data support planned combination trial of VX-809 and VX-770 in second half of 2010 for CF patients with
the F508del mutation-
CAMBRIDGE, Mass., Feb 03, 2010 (BUSINESS WIRE) — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced results from a preliminary analysis of data from a 28-day Phase 2a clinical trial of VX-809 in patients with cystic
fibrosis (CF) who are homozygous for the F508del mutation. VX-809, an oral investigational Cystic Fibrosis Transmembrane
Conductance Regulator protein (CFTR) corrector, was well-tolerated across all four dose groups studied. In the trial, VX-809
showed a statistically significant decline in sweat chloride at both the 100 mg and 200 mg once-daily doses, suggesting that the
activity of the CFTR protein was increased in patients during dosing. Additionally, VX-809 demonstrated a dose response in
change in sweat chloride across the four dose groups. On the basis of these results, Vertex plans to initiate a combination trial
of VX-809 and VX-770, an investigational CFTR potentiator, in the second half of 2010. VX-809 and VX-770 were developed
with support from Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation.
“This Phase 2a trial evaluated the potential effect of an oral compound to improve trafficking of the defective CFTR protein,
and its results represent an encouraging step forward in the development of new therapies to treat the underlying cause of CF
in patients with the most common CFTR mutation, known as F508del,” said J.P. Clancy, M.D., Director of the Pediatric
Pulmonary Center at the University of Alabama at Birmingham and Principal Investigator for the VX-809 Phase 2a trial. “In the
trial, VX-809 was well-tolerated across the dose groups, and statistically significant changes in sweat chloride, an important
biomarker of CFTR activity, were observed at certain dose levels. There is high interest in the CF community in new
approaches to CF therapy, and we look forward to the future exploration of VX-809 and VX-770 as part of a novel combination
regimen aimed at treating the majority of CF patients.”

IT WORKS!

Ok, first if you need a review of what this all means, read an earlier post here.  The big news here is that Vertex 809 somehow “corrects” the trafficking problem of the dF508 defective protein…at least enough to cause a change in sweat chloride concentration.  This is huge! We know that another Vertex drug, Vx 770, potentiates  (enhances) the effectiveness of the chloride channels present at the apical membrane in the G551D mutation, and is now being tested on people with the dF508 (most common) mutations as we speak (read, write, whatever we are doing).  So, if Vx 809 gets the protein up there, and Vx 770 opens it….

Is this the beginning of the end of CF as we know it?

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