Monday, March 24, 2014

Hope whispers, "Try one more time"

When the world says, "Give up,"

Hope whispers, "Try it one more time."
~Author Unknown

I have a hard time leaving the fate of my kid up to researchers. I know and appreciate that a ton of great research is being done to find new and novel treatment options to help patients with Cystic Fibrosis, but it's not happening fast enough and that's not okay with me. 

Two years ago, when I attended my very first NACFC, I found somewhere deep within the program book of abstracts ad article on Gallium and the potential for its use in treating pseudomonas infections. Six months later, at an unrelated meeting in DC, I heard the president of the CFF mention Gallium as something that would probably be a pretty good thing for CF the future. Fast forward to 2013 when Drew acquired Achromobacter, the miserable multi-drug resistant bacteria that has been causing him problems for over a year now. One of the first thoughts that I had was "gallium". Then, this past fall when I attended the 2013 NACFC, Gallium was presented during one of the Plenary Sessions. I must try gallium.

I'm no scientist. I'm sort of like a doctor with far less textbook training but way more real life experience. (I kid! (sort of)) My understanding of the way gallium works in CF is this: When a bacteria like pseudomonas starts to grow in your airways, it binds together creating something called a biofilm, which makes it incredibly hard for antibiotics to penetrate. Achromobacter, like pseudomonas, also creates a biofilm. It is even more resistant to antibiotics than pseudomonas. In fact, the kind Drew has is completely resistant to all antibiotics. Studies have show a 16% decline in lung function ever 48mo when a patient is colonized with achromobacter. On one hand that sucks and is terrifying. On the other hand, this is just a research paper. Either way, this is not okay with me. 

I started to push the envelope a little, emailing Drew's doctor more, asking everyone I know who has more medical knowledge than me about gallium and it's uses and its potential, inside and outside of CF. I've asked about dosing and forms (inhaled vs injected) and how I can get my hands on it to try it. The information that I found on the current clinical trials for gallium are all looking at the bacteria pseudomonas. They're also suggesting that the estimated study completion date is September 2018. That is more than 4 years from now! That's 4% for us. And that also is the study on pseudomonas. I completely understand and appreciate that steps are taken to understand the safety and efficacy of different drugs before they become available to patients, but there has got to be a better way. This drug is already available to patients for a number of different things. We need to find ways to work together and use the testing that they've already done that brought this drug through FDA approval to lessen the time that it would take for others to benefit from it's use. 

I don't know if gallium (aka Ganite) will help Drew, but I'm going to find out. If you're a doctor or scientist whose reading this, tell me what you know about Ganite. Help me to piece these things together to discover who in this community could benefit from this drug. Are there different ways to administer it that could limit any harmful effects? I am playing scientist now and I need people to help me collect everything available on this drug so that I can make sense out of it. 

It's easy for a doctor or researcher to say to me "just be patient" when it's not their kids life on the line. I know that there are tremendous new therapies available and soon to be available to CF patients and I pray that Drew will find benefit from them. However, I need him to be well when he starts them. My job as his parent is to keep him alive and well, and I will do that. I cannot sit back and wait for someone else to determine the fate of my child. I will talk about this and talk about this and talk some more, collecting resources, making connections, finding what I need to either prove or eliminate this drug as a reasonable treatment option for my kid, not because I want to stir the pot or because I don't have anything better to do, but because his life depends on it. His life depends on me and I'm going to find a way to save him.  

I will leave you with this, stolen shamelessly from another fantastic CF blog. "My heart reminded me the race in the battle against cf is NOT a sprint based upon numbers. It’s a MARATHON of decisions based upon individual knowledge, circumstances and needs." That's the truth. It's exhausting, fighting this way, but when you have no choice, hope keeps you going. 

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