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.

Tuesday, July 7, 2015

Cure SMA Conference - Day 3

Day 3

Our third day at the conference was similar to day 2 in that we got to choose between several workshops to attend. We had three different workshop times that day. Beau and I split up in order to gain as much information from as many different workshops as possible. I attended a nutrition course for oral feeders, a yoga therapy class, and a physical therapy class while Beau went to a breathing class, an orthopedic class, and toy adaptation. Beau actually stayed in the "toy adaptation" class for over an hour past the time of the workshop to help other families put their toy together. I was a proud wife that day...he was definitely using his gifts. That's conference for you though, we all come together to help one another. To join together as a family and work toward a common goal. That is the mindset of the Cure SMA conference.

After the workshops were completed we had the opportunity to sit in a "Researcher Q & A". This is where the researchers come sit on a panel and explain what their research is focused on, where they are currently in their research, and then they allowed the families to ask them questions. This was very interesting to me because I learned that we are closer now than we have ever been to a treatment or maybe even a cure for this disease! What an amazing day that will be!!

The day finished off with a PJ party and movie night. We were invited to wear comfy pj's and grab some pillows, blankets, and snacks and watch a movie (this year it was Bug's Life). This is just more time to sort of "hang out" with the other families. Steele enjoys any activity that includes popcorn, so he was completely content.

Overall, the conference was better than great. I know I have repeated that over and over again, but we live with SMA everyday. SMA is not great everyday, in fact, it really sucks a lot of the time. It is so hard as parents to watch your child struggle. Don't get me wrong, Steele is joyful most of the time and that really helps. It just doesn't seem fair that when he falls he can't always get back up. Its not fair that it is so hard to feed himself with eating utensils- so hard in fact that at almost 3 years old I'm still feeding him 95% of the time. I hate that nagging voice in my head that worries about what Steele's future will look like- what will he be able to do? What won't he be able to do? Will he get scoliosis? Will he need surgery? Will he always be able to eat by mouth? Will SMA take him from me? The fears could go on and on. So, when we get to go enjoy some aspect of SMA, we do it wholeheartedly. In most cases, you don't get to choose your family. That's how it is with our SMA family...its not a family we chose but chose us. I am proud of my son everyday for how he battles, how he has chosen to go through this life.... JOYFULLY. The Lord is using him to minister to me. We can't live in fear everyday, we weren't created to. We will celebrate each day we are gifted together.

When the conference was done we decided to extend our stay and take a couple days to relax and unwind our minds after all that had gone on that weekend. We had such a great time, but I couldn't help but feel like something was a little "off". For whatever reason I just didn't have the same feeling when we walked into and through our beautiful hotel. Then, on the side of one of the buildings downtown, I saw this quote by Walt Disney- "You can design and create, and build the most wonderful place in the world. But it takes people to make the dream a reality". And it hit me. That's what was off. When the families left the conference the feeling of the conference left too. We were right back to our normal which is to not be normal- if that makes sense. Needless to say we can not wait for next year's conference.

Giving Thanks

It wouldn't be right if I didn't stop and thank my dad, who generously provided this trip for our family. Our financial situation can get pretty sticky sometimes with all of the medical bills and necessities we pay for on a regular basis. So without his help, we wouldn't have been able to go on this trip and have these experiences. Thank You Dad!! We love you.

And once again I would like to thank you- whoever you are reading this and for whatever reason it is you read- we are thankful. It is our prayer that this blog would not only educate others about SMA, but also to encourage others in their journey. I pray that our life and testimony would encourage others to keep going, keep fighting, to love the Lord with all their hearts, and to do all things with JOY and THANKS :)  Always be full of joy in the Lord. I say it again-rejoice! Philippians 4:4

God Bless!!!

More Pictures

PJ party and movie night

Steele making friends at the conference


Riding Roller Coasters at Worlds of Fun Park

Union Station

Dave and Buster's Arcade Fun!

Kansas City Zoo

Snacking while riding the train at the zoo