Her lips quiver, but days before they sported a wide smile. She grabs for my hand in an attempt to gain comfort, but a day before she gave me high fives. The noises from her mouth are those of distress, but days before they were squeals of joy. The difference between now and before is a feeling of anticipation and reality as we embark on the process of the Spinraza injections.
Spinraza is an amazing medicine designed to be a treatment for SMA. According to the Biogen Press Release titled “Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular Atrophy,” it is “an antisense oligonucleotide (ASO) that is designed to alter the splicing of SMN2, a gene that is nearly identical to SMN1, in order to increase production of fully functional SMN protein.”
According to the same article the “From baseline to 15 months of treatment, patients who received SPINRAZA achieved a mean improvement of 4.0 points in the HFMSE (Hammersmith Functional Motor Scale Expanded), while patients who were not on treatment declined by a mean of 1.9 points. The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA, and a change of three points or greater in the HFMSE has previously been identified as clinically meaningful.”
“Clinically meaningful” … such elegant words for such a scientific description. We do, however, take those words to heart as our hope for Ella lies directly in the hands of this medicine. We were told that typically there are signs of improvements by the fourth injection, and here we are just finishing up the second injection.
Ella sits in her second pre-op session of Spinraza injections. She wanted to try doing it while awake, using the numbing medication rather than general anesthesia. For days she’s been looking forward to her injections, and looking forward to not having an IV, or having to stay in the hospital for hours afterward.
As she sits on the hospital bed she becomes nervous. The reality of the injection is upon her. A doctor just finished explaining the procedure to her (as they do each time) and she asks if it’ll hurt.
“You’ll be sleeping” was his reply.
The doctor didn’t know Ella wanted to try it without anesthesia.
“I will?” she responds, looking at me for confirmation.
We talked a bit more about the differences between going to sleep and staying awake and in the end Ella decided the IV wasn’t so bad. She figured having the needles in her back, even with the numbing medication, would be too unpleasant. Her fears now turn to falling asleep under the general anesthesia mask, and she wants Daddy to be with her.
The medical team convenes for a short while in the hallway while Ella fights back the tears. They return with a solution.
“How about we get Daddy dressed in a white ‘bunny suit’ and he can come into the operating room while you fall asleep?!”