Deciding to Hold Off on Spinraza, for Now

Ryan Berhar avatar

by Ryan Berhar |

Share this article:

Share article via email

Refined By Fire Ryan Berhar

In the past two years, I’ve held seemingly every possible position on Spinraza (nusinersen). I went from elation in knowing that a treatment for SMA existed to being so fed up with the process that I never wanted to hear the word Spinraza again.

The current situation is that my prior authorization has expired, so I finally decided to discontinue my pursuit of Spinraza to hold out for future treatments. I have numerous reasons for this that I would like to share.

The immense physical, emotional, and spiritual toll

Physically, an unfortunate experience at the hospital in April that I detailed in previous columns was my lowest point. Although the surgery didn’t happen, it was still a taxing ordeal. I didn’t get much sleep for a few nights and was poked repeatedly at the hospital. Worse, the entire procedure had to be called off when the nutrition plan was inadequate, causing me to be malnourished and dehydrated.

Emotionally, it’s tough to pinpoint my nadir because the entire journey has been emotional in one way or another. It’s not something I want to continue.

Spiritually, I hit bottom two summers ago. At that point, my quest for Spinraza was still in its early stage, but the setbacks already had gotten to me so much that I briefly considered the drastic measure of abandoning my faith.

Interested in SMA research? Check out our forums and join the conversation!

All of this is in the past, and I understand that. The problem is that I have no reason to believe it won’t continue. Might I be “giving up” right before the final hurdle? Sure, maybe all that’s left to do is get the assessment for the prior authorization and I’d receive Spinraza a week later. I don’t believe that’s a reasonable assumption, though. Most of the obstacles I’ve encountered were supposed to be the last ones, yet here I am.

I’ve plateaued physically

Two or three years ago, my physical strength took a nosedive. I was losing my abilities and wanted to receive treatment as soon as possible to halt the decline. However, within the last year, the drop-off has been minimal. So, I’m more willing to wait now.

Better treatments on the horizon?

I recently read that analysts expect the investigational gene therapy AVXS-101 to be approved by the U.S. Food and Drug Administration early next year. However, current clinical trials seem to focus on infants and children, and it’s not clear what the future will hold for adults. The effects of AVXS-101 treatment on adults, if any, remain to be seen, at least for now.

Nevertheless, there are positive developments with AVXS-101, a gene therapy designed to deliver a functional copy of the SMN1 gene to motor neuron cells in SMA patients. AVXS-101 is a one-time intravenous treatment, and developers hope it will soon become standard of care, should studies go well, and be safe to take with other medications.

I’m willing to wait and see. This brings me to my next point.

Spinal injections and their potential complications

This is certainly not new information, but the more time passes, the less I want to put my body through an indefinite series of spinal injections, which would be required for me with Spinraza. Even if everything went as smoothly as possible and I experienced zero side effects, that’s still a tremendous physical crucible.

Local doctors have never administered Spinraza before 

Although they’ve used the neural foramen to deliver other medications to patients and are willing to dose Spinraza this way, my local doctors have never done it before. The prospect of being the first patient to have this procedure done locally doesn’t bring me comfort, especially since I’m looking at an indefinite number of injections.

This decision is so tough because every potential outcome is unknown. I don’t think there’s even a right or wrong answer because I don’t know the future. All I can do is weigh the pros and cons and make the best decision for myself.

Of course, my decision may not be right for you and is in no way intended to influence your consideration of Spinraza or any other treatment. Consult with your medical team to make the best-informed decisions about your own care.


Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.


Chris Young avatar

Chris Young

I'm 63 years old and Type 2. I too was elated at the announcement of the approval of Spinraza. But I decided not to pursue the treatment. When I was younger absolutely nothing was known about SMA or how to deal with the side effects. The result is I never had spinal fusion which I should've had. When I needed surgery for other things, I've had anesthesiologists look up my spine and say "there's no way I would consider an epidural on that crooked thing". So the idea of trying to do a spinal injection with my severe scoliosis is pretty much out of the question. Similarly at age 63 I really doubt that the drug would do me any good. I'm also highly skeptical of its efficacy on anyone other than children and young adults perhaps. Let's face it SMA works by the death of motor neurons. So the best we can hope for is to halt the disease progression. I'm really skeptical of anecdotal reports of adults who have seen increased capability. Spinraza doesn't regrow dead motor neurons. If I was 20 years old or younger it would be a different deal. I would be beating down the doors trying to get any kind of treatment I could. I also think that this situation is much different for newborns with type 1. None of them are going to reach age 63 without some sort of treatment. I agree that the gene therapy which is a one time administration sounds like a much better choice. I will be interested in seeing what other treatments come down the pipeline in the near future.

Miriam Terrazas avatar

Miriam Terrazas

Thankfully there are other treatments coming up now, and you have the right to decide. Besides, who knows you and your body better than yourself? My baby has SMA type I and we didn't gave it a 2nd thought when we got access to Spinraza. Take care and keep on sharing your columns with us.


Leave a comment

Fill in the required fields to post. Your email address will not be published.