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VLC 2014: State of the Science Update

Saturday, April 12, 2014

I was honored to be invited to attend the Cystic Fibrosis Foundation's Volunteer Leadership Conference in Bethesda, Maryland again this year.

I tried to capture as much of the information as possible to blog about for the benefit of other CF families.  So, sorry in advance for the crazy amount of slides! :)

I have divided up my experience at VLC in three different blog posts.  The first one is from the "State of the Science Update."  Always, my favorite!!


Bob Beall, Ph. D., President and CEO of the Cystic Fibrosis Foundation addresses the crowd to welcome us to VLC 2014.


This room was full of people who are determined to do whatever it takes to find a cure for CF.


Michael Boyle, M.D., FFCP, Director, Adult CF Program/Associate Professor of Medicine @ John Hopkins Hospital (seated left), Bob Beall (middle) and Preston Campbell, M.D. is the CFF's Executive Vice President for Medical Affairs (seated right) gave the State of the Science Update.


This slide shows major projects of the CF Foundation over the last 50 years.  As the slide shows, it is in the last decade that the CF  Foundation has been focusing on therapeutic development (the development of medications).


If we break down the Therapeutic Era, we can see that it has been in the last 4 years, the CFF has worked on First Generation CF Modulators.  The top three arrows show the focus of the CF Foundation of developing therapies in the next 15+ years.

With regard to expanding Kalydeco to other mutations, the CF Foundation said "probably 15% of population will benefit, hopefully, by 2016."


This slide shows how effective Kalydeco has been versus some of the other therapies.  It is really amazing to see how much more effective Kalydeco is, compared to the medications many CF patients use daily such as TOBI, Azithromycin and Pulmozyme.


This slide shows that scientists have seen significant change in psuedomonas for those who have been on Kalydeco.   This is super promising for all of those with Cystic Fibrosis!  We have typically thought that once a CF patient gets psuedomonas, they cannot get rid of it.  But now, with Kalydeco, there is real hope that even though one might acquire psuedomonas, they might be able to get rid of it.


This slide shows how Kalydeco has demonstrated to positively affect lung function, even after nearly three years!


What about the most common mutation, F508del?   Fifty percent of patients carry two copies of the F508del mutation and 90% of patients carry at least one copy.  We are looking for a combination therapy of VX-809 and Kalydeco (VX-770) to work.  At this time, it shows that although it does not work as effective as Kalydeco does for G511D, but the combination drug still shows very positive results!


This slide shows some of the Phase 2 clinical trial results for those taking VX-809 and Kalydeco.

At the conference, on our table, were some postcards with a distinct message from the CFF.  I thought I would share these messages here...

"Phase 3 Combination Trial Core Messages:
* We know there are high expectations in the CF community about the Phase 3 combination trials of ivacaftor (Kalydeco) and lumacaftor (VX-809), which are being studied in people with the most common mutation of CF, F508del.  However, drug development is a lengthy and unpredictable process.  The trials are still underway and it's too early to speculate about the results.

* The research process for a new drug (or combination of drugs) must follow the gold standard for how clinical trials are run, evaluated and reported to the medical community and the FDA.  This process is essential to ensuring that the new drugs are proven to be safe and effective when used in the broader population.

* This combination trial is only the first step in finding more therapies to treat the most common mutation of CF.  Based on what we already know from laboratory studies and earlier clinical research, we'll likely need to continue to improve the effectiveness of these therapies and perhaps use more than two therapies to achieve the full beneficial effect we all desire.

* The CF Foundation is fighting CF from ever angle and not relying on any one potential therapist to win the battle.  The Foundation is actively pursuing the development of other promising compounds with some of the best biotech and pharmaceutical companies in the world, including Pfizer, Genzyme and Vertex."

We hope to know the results of the Phase 3 VX-809 and Kalydeco clinic trials later this year.  The trial will last 6 months and includes more than 1000 patients.  There is hope that by this time next year, these two medications will be available for those with two F508del mutations.


What about patients that only have one copy of F508del?  This is where Bennett is and the place we are most interested in.  Phase 2 trials are underway with both VX-809 and VX-661.  There is hope that this will come along in 2016.


This is a great slide.  It shows all of the current pharmaceutical companies working towards finding a cure for CF.  When I first attended VLC 3 years ago, there were only a few of them.  Now, the list has grown.  That means we have even more reasons to be hopeful.  Others are seeing the financial potential and possibility of success.  For me, that means a cure is coming!!


This is the CFF lab in Boston.  See more about it here: http://youtu.be/xE4bXjbr3_c


The CF Foundation is exploring new technology.  (I apologize.  My notes got lost on the way home from the conference so I don't have specifics on this slide.  But, when I find them, I'll post up more information here.)


While Kalydeco is able to help only 4% of the CF population right now, the CFF believes that Kalydeco in addition to a corrector medication will ultimately be able to help 95% of the CF population.  That will leave a remaining 3% of those with nonsense mutations and 2% without nonsense mutations.


So, what about nonsense (X) class 1 mutations?  The CFF is determined to leave no one behind.  Although Ataluren showed poor results in its last clinical trial, there is a belief that inhaled medications may have caused Ataluren to be less effective.  So, a new phase 3 trial is planned to start this year!


There are new screening programs for nonsense mutation therapies.  This is very hopeful news for those with CF whose genes end with an X.


The CFF wants to remove those X's (fix the genetic mutations) on the DNA.


Of course, even though the CFF is focusing on a cure, attention to other areas such as addressing CF symptoms and the unmet need of CF antibiotics has not been lost.  There is a need for antibiotics that treat some of CF's worst culprits: pseudomonas, B cepacia, MRSA and mycobacteria.

At the end of the session, someone made a comment to the presenters that they were disappointed by the update.  The person indicated they wanted someone to "wow" them with the news.

Dr. Campbell explained that there was news to be "wow"ed by, but he cautioned, "it would be irresponsible for us to promise success without bumps along the way...we are not making an Egg McMuffin here."

Bob Beall added, "This is not an easy problem.  We're curing a genetic disease!"

But, Dr. Boyle said, “It’s an exciting time. We realize there are challenges. We will get there; it’s just a question of how long.  The key to improving the lungs is fixing the underlying problem. Ivacaftor does that. It’s a wow!"

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