Wednesday, May 14, 2014

Hope for Tomorrow

A lot can happen in the course of a day, a week, a month, a year, or a lifetime.  I often think about the things my grandparents have seen change in their lifetime: electricity, cars, farming, indoor plumbing, the advancements in medicines and vaccines, and means of communication.
The advancements in CF treatments have made it possible for me to still be breathing.  There is so much hope for the future.  Each day is a new chance at a life saving breakthrough. 

Yesterday's blog post was Part I of II entries by pharmacist, Stacy Peters, again to whom I am so grateful.  She is constantly researching new therapies and is on the forefront of CF drug development.  In Part I, she discussed the defective protein in CF and its complex genetic challenges.

The Future and Hope for a Cure
In 2O12 a breakthrough oral medication called Kalydeco was released by the FDA.  This ground breaking new drug targets the underlying cause of CF for people with the mutation G551D: only about 4% of people with CF are eligible to reap the benefits of Kalydeco.  Even though I do not have the right mutation for this miracle drug, it gives us all huge hope in the fight against CF and the future. There will come a day when CF no longer steals anyone's breath. 

Here is Part II written by Stacy Peters:

"A new class of medications referred to as CFTR “modulators” has been in development for the last several years.  CFTR “modulators” work by:  1) increasing function of the CFTR protein at the cell surface (i.e. Kalydeco), 2) transporting the CFTR protein to the cell surface (i.e. lumacaftor or VX-661 – currently in clinical trials), or 3)  help the body “overlook” errors in the DNA that make the CFTR protein (ataluren – currently in clinical trials).  Unfortunately, since there are different reasons for why the CFTR protein/gate doesn’t work, there isn’t a “one size fits all” medication for everyone with CF.  While not a cure, the advantage with this class of medications as a whole is that they target the underlying defect in CF, whereas other treatments such as Pulmozyme® and TOBI® all target the aftermath such as the thick mucus and bacteria in the airways.
Kalydeco (Ivacaftor) is currently the only CFTR “modulator” approved, it works for people with a mutation called G551D and other class 3 mutations (only ~4% of those with CF).  Kalydeco works by activating the CFTR channel or “gate” and helps normalize water and salt transport.  Since it only works by activating the “gate” on the cell surface in a very specific way, it doesn’t work for those who have other classes of mutations. 
There are several other CFTR modulators in clinical trials.  Some are using 2 drugs to attempt correcting the CFTR protein.  For example, in people who have delta F508, the most common mutation, there are 3 new medications being studied.  Lumacaftor in combination with Kalydeco, VX-661 in combination with Kalydeco, and N6022 which is in very early development.  The lumacaftor or VX-661 works by moving the CFTR protein to the cell surface, then Kalydeco will come in and open the gate. 
Ataluren is also in clinical studies for those with class 1 mutations.  It works by causing the cell to “overlook” the error in the mutated CFTR gene, allowing for the CFTR protein to be made. 
The goal of the CF Foundation is to ensure there is a CFTR modulator for EVERY mutation.  This will be quite a challenge given the variety of mutations out there.    
While CFTR modulators are all the rage in CF research, there are other very important medications and treatment approaches being evaluated.
·       New inhaled antibiotics to help suppress bacteria such as pseudomonas and MRSA.
·       New anti-inflammatories targeting inflammation in the airways and body.
·       New delivery devices that decrease the time it takes to nebulize medications.
·       Evaluation of existing therapies to determine if there are ideal combinations and treatment durations to maximize the effectiveness of the current approved medications.

·       For more information visit:

While there are no guarantees that medications in clinical trials will be proven effective, the rapid advance in technology and progression through clinical trials is promising."

New developments in treatments and the fight against CF are crucial, not only for the daily fight against CF, but for the discovery of a cure.  Treatments that have extended my life thus far are losing their effect: my CF is becoming resistant and less responsive to treatment.  The advancements in my lifetime alone have been truly amazing, and I cannot wait to see what the future holds.  Again, thank you to Stacy for sharing her amazing gifts making it possible for us all to breath.  I am so grateful to each of you who so passionately have fought and continue to fight to add tomorrows for everyone with CF.  I wouldn't be here without you.  Love to you all. 

What changes have you seen in your lifetime?

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