Looking forward and beyond for people with Cystic Fibrosis and their treatments

With great excitement (and extreme fear), I'd like to start this blog off with stating Bobby officially has been listed on the transplant list.  So what this means for us is that at any moment from this point forward, no matter the time of day, Bobby can get a call to go into the hospital for surgery. We are super excited because this means we could have another 10 years together, BUT we are very scared because of all the risks and complications that statistically are likely.  Long story short, our lives just got a bit more complex.

                                                           (CREDIT: Pinterest)

But what does this mean for people with CF? Is this their last option?  Hopefully, the answer one day will be no.  Right now, a company called Vertex Pharmaceuticals has created a medication that compensates for the defective gene in CFers, which reduces the levels of mucus inside the body. (Check out http://www.nhs.uk/Conditions/cystic-fibrosis/Pages/treatment.aspx for more information.)  In essence, it contradicts the defective gene.  While it's not a cure but it is about as close as you can get!  Unfortunately, in this early stage, the medication is only effective in people who have one type of rare mutation, (which is thought to be about 1/25 people with CF, or 4%).

                                            (Credit: Vertex Website)

Vertex is working on other versions of this medication, which you can see are in various phases of development.  You can find more information on Vertex here.  Hopefully soon we will get more information from these! (However, it will come too-late for my husband because once he has the transplant, he will no longer require these medications for his lungs.)

Another hot topic lately is the stem cell treatment for CFers.  While I understand the perspective the U.S. medical field has taken, I do NOT agree with it. I've always been of the opinion that pharmaceutical companies own the medical field, and they make a financial windfall off of people who are chronically ill.  I do not think enough is being done here to truly utilize stem cell research towards developing cures for these and other diseases.

Many doctors I've spoken with push away from the subject, and I'm curious why.  Is it from an ethical stand point or a true bias?  Adult stem cells can be donated without harm, and can be utilized in the same manner as "embryonic," thereby avoiding any ethical dilemmas. The more information I gather behind the medications necessary for chronic conditions, the more clear it's becoming to me that drugs are being pushed over potential cures.

With this small rant over, I do firmly believe that stem cell transplant treatments are on the horizon, and will certainly be a viable option to people, like Bobby, who are struggling with CF.  And one day soon, CF will stand merely for CURE FOUND.

 

(CREDIT: Pinterest)