SPINRAZA

So what in the heck is Spinraza? Well, if you're associated with the SMA community in any way, you already know the answer. If not, allow me to educate you. Spinraza is a drug that treats SMA. Correction, it's the ONLY drug ever developed to help fight SMA. Hard to believe this drug is a reality. When Colby was diagnosed almost 15 years ago, there was no cure and no treatment. I honestly never thought we would see hope in a medication to help Colby in his lifetime. Well, guess that'll teach me not to dream big huh? Of course we've all prayed for something to help in any way, and there's a good possibility Spinraza just might help him.

The majority of the research and drug trials for this drug have been done in newborns and infants. Spinraza shows amazing results in reversing the symptoms of SMA. But what does that mean for Colby? SMA has ravaged his body for years, leaving him with very weak muscles that despite our best efforts are starting to contracture, along with being vent dependent. Colby can usually spend 2-3 minutes off his vent before his oxygen starts to drop. Will Spinraza “fix” all this? Will Colby be able to walk and talk soon? No. He won't. However, this drug shows some promise with the older SMA Type I kiddos who have been in trials. If Spinraza could stop SMA from progressing and making Colby any weaker, I'd be happy. If the drug could help Colby gain back some strength and movement that SMA has taken from him, I'd be over-the-moon thrilled. Spinraza is not a fix-all for Colby, but at this point, it's the ONLY hope we have for Colby in treating his SMA. Let's do this!

Not so fast. It's not that easy, of course. Never is with Colby and/or SMA. First was the process in getting the drug approved through insurance. The drug was approved by the FDA on December 23, 2016. Being a brand new drug and treating such a small patient population, the drug is outrageously expensive. The first thing Colby's insurance required was a pre-treatment PT evaluation, which we did back in Cincy on April 19^th. Next insurance required genetic testing that wasn't done back in 2002 when Colby was diagnosed. Check. Got that done as soon as the insurance company asked for it. Then on June 1, Colby's private insurance changed, meaning we had to submit this information to his new coverage and see what they had to say. They approved! Colby has been given the okie dokie to start receiving the one and only drug on the market to treat SMA. Exciting news, I know. But I'm also very apprehensive for several reasons.

You don't just pick up Spinraza at the Kroger pharmacy, crush up a pill and flush it down Colby's g-tube. Oh no. The drug is administered via lumbar puncture. Honestly, this scares me to death. A possible side effect could be a migraine if there is any fluid leakage during the procedure. I've lived with migraines for years, and that is a pain and discomfort I wouldn't wish on anybody, especially my sweet boy! So that's a concern. There's also a worry that since Colby's spine is now fused, finding an opening large enough to administer the drug might be an issue. We won't have any idea until we get to Cincy Childrens and the IR (interventional radiologist) looks to see the access availability for Colby to get Spinraza. And at the moment, Colby's blood clotting factors are abnormal, so there's no way he's getting this drug until that issue is fixed. Colby had a ton of nutrition labs done last month, ordered by his neurologist. She also ordered a PT/PTT/INR as pre-procedure labs. And of course they were off because the blood was drawn from his port and not from a peripheral. The next step was to get an order for a peripheral lab draw. Hoping that this clears up the clotting issue. Just got the order for that yesterday. Tried to find a company to come out and do the blood draw here at home. Unsuccessful. So tomorrow morning we'll be dragging Colby up to LabCorp to get his blood draw done. I'm also a nervous wreck about this, since Colby is a horrible stick! I mean absolutely horrible! I'm dreading it big time. Hoping I don't thunder punch anybody during the lab draw tomorrow.

Wow, this is a lot of info in a short blog. That's where things stand with Colby getting this drug. It's pending basically. Once everything is a go, we'll hit the road to Cincy. Colby will receive the drug on day 1, day 15, day 30 and day 120, then every 120 days forever and ever or until we decide to stop the injections. Honestly, I'm pretty damn nervous about Colby getting Spinraza. The drug is brand new, so who even knows any long term side effects. Plus I hate the idea of Colby getting this done by lumbar puncture. There are some SMA families who jumped as soon as the drug was approved. They wanted Spinraza for their child immediately. That's not me. There are some SMA families who aren't currently considering the drug for their child. They want more information. That's not me either. I'm in the middle I guess. Scared to death of what this drug will or won't do for Colby, but at the same time I feel I owe it to him to try. He's come so far and done so much beyond what doctors ever thought he would, why not push the envelope and see if now his SMA can maybe be reversed. Wish us luck! This is a very, very, very big deal for Colby. The process needs to go as smoothly as possible so I don't lose what little sanity I have left. Who would have ever thought I'd be blogging about a treatment for SMA?! How awesomely awesome is that?!