Wednesday, September 2, 2009

New Drugs Update...and request

As many of you may remember from a previous post a year ago, we wrote about some exciting new drugs that were on their way to helping those with CF. One of those drugs is now in clinical phase trial. Elena's Grama, Susie Goodrow, sent out a prayer request with the following info:

We thought we would pass this info on to you for prayer of this new drug treatment for CF. It looks quite good.

Elena, however, is a Delta F508 patient and this drug will focus on G551D. Hopefully, the VX-770 or VX-809 will also work to help Elena in the future. See the info below and in red the area about Delta F508 and this drug.

Either way our prayers will not only help Elena but others and every step of research for CF leads in the right direction we all want to see.

For more information or answers to questions you may have go to

Please pass on to your family, friends, and prayer groups.

Praying Grandparents,
Susie and David

VX-770 Trial Begins in Children as Part of Phase 3 Program
August 5, 2009
Today, the CF Foundation announced the initiation of a clinical trial in children as part of the Phase 3 registration program for VX-770. VX-770 is an investigational oral therapy aimed at treating the underlying cause of cystic fibrosis. The drug is being developed by Vertex Pharmaceuticals with support from the CF Foundation.
This is the second of three clinical trials underway in the Phase 3 registration program for VX-770. The registration program is designed to generate data that the U.S. Food and Drug Administration can use to determine if VX-770 is safe, effective and acceptable for approval.
“This potential drug is one of the most promising therapies in our pipeline that aims to treat the underlying cause of cystic fibrosis,” said Robert J. Beall, Ph.D., president and chief executive officer of the CF Foundation.
“We’re excited that enrollment is open for this second trial. The initiation of this study is an advancement in our efforts to bring a new therapeutic option to CF patients.”
The trial is a two-part study of VX-770 in patients with CF age six to 11 years who have the G551D mutation of CF. It will enroll approximately 30 children with CF.
The first trial, which was initiated in May, is examining VX-770 in people with CF age 12 and older who have the G551D mutation of CF.
Why wasn’t the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?
Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770. Most Delta F508 protein is not at the cell surface and therefore it is unclear whether the patients with this mutation will benefit. The Phase 3 registration program of VX-770 will help determine if it is effective in this patient population.
It is possible that Delta F508 patients may need to have the location of their CFTR protein corrected to achieve the most benefit from VX-770. Vertex is developing another compound, VX-809, which is currently being studied in patients homozygous for the Delta F508 mutation. VX-809 is designed to increase the concentration of CFTR proteins at the cell surface.

Joel followed up with:

Thanks, Mom, for sending this out. Here are a couple other notes...

-Notice that these groundbreaking drugs are being developed with heavy support from the CF Foundation. In fact, this work would not be possible without the CFF pouring over 90% of every dollar it receives into this type of research. Drug companies will not do it alone because it costs too much to develop a medicine that only a few thousand people will need to buy. For this reason, Julie and I thank everyone for their support of events that benefit the CFF.

-Phase 3 FDA trials of these drugs could last many many years. We need to pray for the proper political support and leadership that can accelerate the process.

-Elena is too young for most clinical trials, but it's possible that she could be on the cutting edge before we know it. Prayer is needed that she be at the right place at the right time with the right criteria to potentially benefit from (and tolerate) the trial.

This is all very exciting news. LOTS of information to digest, but wanted to be sure all of Elena's support is aware of the awesome strides that are being made because of the prayers and donations you have all made over the past five years. We are MOST grateful!


1 comment:

  1. Praying for that strong little girl and her family daily.


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