Monday, July 20, 2015

Exciting News

WOW! We have had a crazy past 24 hours. I am so tired, but too excited to not share this news. Let me fill you in...

We left yesterday (Sunday) after church to go to Dallas for a routine neurology check up the following morning (Monday). Since Steele's diagnosis, we have gone every six months to this particular doctor for routine check-ups and exams. The appointment we had today was scheduled a little over six months ago.

About a year ago, give or take, we found out that a clinical trial would be opening up sometime this year in an effort to develop a treatment for SMA. Now that doesn't mean that the trial just started. In fact, this particular trial has been through a "pipeline". Basically, in my own simple terms, in order for any drug to be approved by the FDA it has to travel through this pipeline. It must be developed and put through pretty intense testing in order to even make it to human trials. They told us at the conference last year that for about every 10,000 drug ideas ONE makes it into the first step of the pipeline. Once they get to that point, about 10% of those make it through. Its a tough system, but we are thankful it is because that's how we make sure the drug is safe and effective. So, we heard that a trial would be opening up and it seemed as if Steele just MIGHT be able to be included. The study is to include children with his "type" of SMA and he fit in the age window.

When we heard that Dallas was a possible site for the trial we got very excited and immediately started praying.We went into our neurology check up in January hoping to hear about the trial criteria, and if we might be included. But it wasn't the right time. Dallas wasn't quite ready to start screening possible participants. So we waited. And waited. And waited. I can't tell you that I haven't been impatient at times. And then other times I have been optimistic. And still others I have had the feeling of just totally putting it out of my mind because I didn't know if that day would ever come.

Let me get sort of off topic for a bit.. I promise I have a point!!

Not quite two months ago Steele had surgery on his tonsils. The surgery went well and we are so thankful we did it. What you don't know is that only days before Steele's surgery we were faced with an extremely difficult decision. We were informed that if Steele had surgery, he would not be eligible for screening for the trial for two months. I went into panic mode. Why? Well my mind thought, "What if we have this surgery and then they finally open the site and we aren't able to participate?! What if we miss our shot?" I was shaken. I felt like I had to make a huge decision that could completely affect the rest of Steele's life- no matter what we chose. So, we prayed. And sought out wise counsel. And prayed some more. As you know we decided to go ahead with the surgery. This meant that until August 2nd, Steele would not be eligible for screening. It was a risk, but we took it.

And then today happened. At our appointment today we found out that the Dallas site is officially open for the clinical trial we have been praying about. Then we found out that Steele's name is on the list, and they are hoping to get us in for an appointment.....get this.....wait for it.....THE.FIRST.WEEK.IN.AUGUST. WOW!!

What this means: Steele will be screened to determine whether or not he is eligible to be a participant in this study. He will have to go through a "gauntlet" of tests. His participation in this trial will be a direct result of how the researchers determine he does on those tests and if he meets all of the inclusion criteria.

What this does not mean: Participation in the study does not guarantee results- positive or negative. It actually doesn't even guarantee he will get the drug. He has a 1/3 chance of getting the Sham procedure which is no drug at all. It doesn't mean that this drug will definitely be approved by the FDA. It is not considered a "treatment" for SMA. It is a trial- they are exploring the effects of this drug in hopes to eventually have some sort of treatment one day.

You may totally agree with this research. You may totally disagree. Either way is ok with us. In fact, we aren't even totally sure we totally agree OR disagree! That is ok. Our motivation for participating in this study is to further the research and hopefully advancement in medicine for people with SMA. If they can develop or decide against a treatment for SMA based on these trials, and we can help at all in that process we are all for it. I pray there is a CURE for SMA one day, and I pray that we can help each other to get there.

No matter what happens from this point forward we know that God is sovereign, and has a plan for our lives, and we are so thankful.

Romans 8:28~ And we know that God causes everything to work together for the good of those who love God and are called according to his purpose for them.

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