1. The big news in these parts is that the boys are finally getting Spinraza!
The hospital scheduler was not prepared for my display of emotion.
me: (sees CHOP on the caller ID, gets hopes so damn high) Hello?!?!?
scheduler: Yes, can I speak to the parent or guardian of Fulton Mantoan?
me: (voice a bit louder) YES, THAT’S ME!!!
scheduler: Great. Fulton’s name was added to our list for Spinraza treatments so I wanted to call and schedule him….
me: (falling to my knees and interrupting) AAAAAAAAAAA! OH MY WORD YOU HAVE MADE MY DAY! YOU HAVE MADE. MY. DAY! YOU HAVE NO IDEA HOW LONG WE HAVE WAITED FOR THIS CALL!!! (Might have professed my undying love for her too but I’m not sure.)
scheduler: (weirded out but keeping it together, replies in a friendly tone) Uh, yeah, you’re welcome. Haha. Yeah, so, we have Friday the 18th open if….
me: (trying not to burst into tears of joy as I skip around the house) WE’RE AVAILABLE ANY TIME!!! WOOOOOO!!!
And you get the idea.
2. But let’s back up so we’re all on the same page. First, it helps to understand a bit about Spinal Muscular Atrophy, the neuromuscular disease both Fulton and Teddy have. I’ve blogged about it here, and the Muscular Dystrophy Association has a good summary too.
3. Now, Spinraza is the first FDA approved drug to treat folks with SMA. That means that after years of testing (and some pretty incredible results) the Food and Drug Administration said, yes, this drug is safe and more effective than a placebo and there for may be prescribed to kids and adults with SMA, opening the door to all those with SMA to approach their doctors and insurance companies and ask for the treatment. If you want to understand how Spinraza helps people with SMA, just watch this video. It explains everything much clearer than I ever could.
4. Shortly after the FDA announced approval in December 2016, we made an appointment at the Children’s Hospital of Philadelphia to learn the eligibility of the boys. It was determined at that visit that Fulton would need to have the drug administered by interventional radiology since the curvature of his spine would make it tricky for the regular neurologists to do the lumbar puncture. It was also determined that Teddy could have his doses administered in the OR by neurology (who, having run some of the drug trials for Spinraza, were already familiar with the drug and how to administer it). During the procedure a bit of spinal fluid is withdrawn and the drug is injected back into the place of the spinal fluid. The only major side effect of the lumbar puncture is headaches and/or pain at the sight of the injection.
Then we had to wait. The boys’ insurance released their initial guidelines for Spinraza coverage effective April 1. Unfortunately, under those guidelines, Fulton would not be covered because he uses breathing support overnight. But we knew CHOP would take the lead in fighting for treatment, so we continued to wait. Thankfully, the insurance guidelines were updated and Fulton became eligible, however, the hospital and insurance company still were “in negotiations” for quite some time over some other details.
5. The boys will need four doses over eight weeks, then follow-up doses every four months as the medicine breaks down in their bodies. The first four doses, or loading doses, cost $750,000 for each child. Each follow-up dose is $150,000. If insurance said no, this was not something we were going to be able to hold a bake sale for. However, since the drug is showing such promising results, which will lead to less medical interventions in the long run for those with SMA, insurance companies are covering Spinraza for most cases (I believe. I don’t personally know anyone who hasn’t been able to get it yet, though many of us have had to wait or fight to get to this point. And I’m not sure of the overseas availability yet.)
6. After originally scheduling Fulton for today, he was rescheduled for August 24th with Teddy so we can have the boys on the same dosing schedule. Admittedly, both boys are not too keen on getting needles in their backs (“Mama, why do doctors have to do such brutal things???”), but I am hopeful they will soon feel, and see for themselves, the benefits of this drug. Friends of mine have reported some wonderful gains in their own children, so while I don’t want to get my hopes or the boys’ hopes up too high, I’m a bit giddy at the possibilities. I do want it understood that Spinraza is NOT a cure: Fulton will not suddenly get up and walk, and the damage already done to their muscles cannot be reversed, but could they regain lost skills and some strength? That is a very real possibility.
7. As if this news itself wasn’t wonderful enough, I also learned (on the same day!) that Fulton was approved for 12 hours a day of nursing care, seven days a week, in school and out.
Actually, when the nursing agency called to tell me I was speechless. I could not believe we got so much coverage so easily. When the nursing rep asked when I wanted to start nursing and what hours I was thinking I replied, “Uh, I’m gonna have to talk to my husband and get back to you cause I have no idea.” When I told Tony we both stared at each other in disbelief for a few moments. However, the shock quickly wore off and dancing commenced.
Long time readers will remember that we had nursing care for Fulton about five years ago for seven hours a day. Unfortunately, due to a “misunderstanding” on the part of the nursing agency we were only supposed to use those hours overnight, but we thought we could use them any time. Once Fulton’s insurance caught the error, they yanked the nurses within the week and despite investigating the possibly of nursing a few times since then, we’d learned to make do without the help. I was surprised Fulton was even approved for a nurse at school; I’d assumed he’d only get a one-on-one aide. When the nursing agency suggested we pursue nursing for the days he was off school, I thought we’d only get nursing during the school year, or on the days he was too sick to go. Even the nursing agency was happily surprised to see we’d been given all the hours we asked for, effective immediately.
So this week there’s been lots of new faces in the house as nurses meet Fulton and our family for the first time (and I pray desperately that my house isn’t too loud, weird, messy, or full of people all the time to scare them off.) We’ll probably wind up with a team of three to four regular nurses with a few others who fill in when needed. Fulton is reserved. He only has vague memories of his previous nurses, but at the time, losing nursing care was hard on him. Unable to verbalize his feelings of loss, it was a miserable few months as he tried to work out his frustrations and make sense of everything (usually by acting out towards me.) But I know he’ll warm up to everyone and we’ll settle into a new normal soon enough.
School, nursing, Spinraza: the last quarter of 2017 is shaping up to be the most exciting of all. I mean, this is how I walk around the house now, I can’t help it.
Might weird out the nurses just a bit.
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