Cautious Hope

A lot of exciting things have been going on since our last post.  Two days before Christmas, we were on our way to pick up Alexander’s new companion dog, a dog we hope will be a great service dog for him, when we received the amazing news that Nusinersin (now being marketed as Spinraza) was approved by the FDA for treatment of all types of SMA!  I cried many tears of joy, and still get emotional just thinking about it.

This news provided us with a wonderful Christmas full of hope.  To add to the excitement, Charlie (new puppy name) is fitting in well with our family.  He is super smart and I believe he will learn quickly when it comes time for training; usually starting around 6 months old.

Our excitement was quickly shocked back into reality when we discovered, along with many other SMA families, that the price for this new treatment would be $750,000 for the first year and $350,000 for every subsequent year for the rest of Alexander’s life.  Each patient receives 3 injections the first month and then an additional injection every 4 months indefinitely.  The drug is administered as a spinal injection, and because SMA is a rare disease, we were expecting a hefty price tag.  We certainly weren’t expecting it to cost 3/4 of a million dollars the first year alone!  We certainly hope price isn’t a barrier for receiving this life saving treatment and we were hopeful that Alexander’s neurologist could help us justify the treatment to insurance.


We had an appointment with our neurologist on January 3rd to discuss our desire to get him treatment as soon as possible.  The front desk assistant checked us in and gave us the typical medical questionnaire form; it felt good when we wrote “Spinraza!!” in dark ink in the “Why are you here?” box.

Spinraza’s approval is so fresh that the neurologist’s office was unaware that it was available to be prescribed.  Heck, the pharmaceutical company, Biogen, was at that very moment on a conference call with CureSMA.org detailing the early processes being developed to get patients signed up for treatment.  After the NP and doctor were away for a few moments, we suspect researching Spinraza, they returned to explain to us that Nationwide Children’s Hospital in Columbus, OH may be better prepared to treat Alexander, at least more promptly.  We were a little discouraged by this, mostly because of the fear of our WV state insurance not covering the drug for Alex outside of WV, but we were told that Nationwide may have better charity assistance to ensure Alexander gets his injections.  That coupled with the possibility of expedited treatment was enough for us to leave satisfied.

The very next day, we received a phone call from their office in which they informed us that they were having a meeting with the Biogen drug rep that following Friday!  We had left our appointment the day before thinking that we would have to go to Columbus to get treatment.  We were certainly excited to learn how quickly things were moving at the WV location!  I received a follow up call after that meeting and was asked if we wished to go ahead and submit to our insurance for a pre-authorization for the treatment!   I’m shaking as I type this, thinking about my little boy getting access to this drug.  Of course, his fate is now in the hands of our insurance company.  We have to wait to find out if it has been approved.  I’m nervous but I am also expecting them to deny it, at least at first (we will appeal if that happens). If they do approve it, Alexander will be one of the first four in our state to receive the treatment.  They expect the hospital pharmacy to have the formulary around the first of February and start injections around mid February if insurance is settled.  We can’t wait to update everyone when he receives his first treatment!

He deserves (needs) this treatment

We can’t believe how much has changed!  When we received his diagnosis last April, the outlook was pretty grim.  We were told there is no treatment, there is no cure.  We would see our little boy’s muscles waste away over time and helplessly watch him lose the ability to move, swallow and breathe.  That we should prepare to make him happy and comfortable for as long as possible.  While this drug is not a cure (It does not correct the condition), it is a treatment that can stop the progression of the disease.  We don’t expect to see too much improvement in his motor abilities, but the treatment should stop the deterioration.  This means he will be continue to be able to eat by mouth, be able to move his arms (he still has limited range of motion), breathe on his own and maintain head control.

We are hopeful.  Hopeful that this treatment will  prolong his strength until the next big breakthrough (a complete cure).  It’s a feeling that many parents of sufferers never got to experience.

Alex is such a bright child; he already knows all of the alphabet by sight, all of his colors, numbers, shapes etc.  He can figure out games and puzzles with ease.  It is hard to watch such a smart little boy struggle to do something simple like lift a juice to drink or have to ask for help with lifting his head when it falls forward.  His body is failing him and, up to this point, we have felt completely helpless.  The best we can do is to continue with his regimen of weekly therapies, keep him as strong as possible, keep his legs and arms flexible while we hold out hope in anticipation that he will gain at least a little strength after being on this treatment over the course of time, and ultimately until a cure is found!

We are going to fight for this.  We are going to fight for Alexander’s future.