May 29, 2019 at 7:00 am #19079Michael MoraleKeymaster
Over the past couple of weeks, I have received numerous questions regarding the differences between Spinraza and ZOLGENSMA. On Friday, May 24, 2019, ZOLGENSMA became the second FDA-approved treatment for Spinal Muscular Atrophy (SMA). With this approval comes a lot of questions regarding these two treatments. Please understand that I’m not a doctor, but I’m going to try to describe the differences between Spinraza and ZOLGENSMA in layman’s terms.
The human body is composed of a myriad cells and genes that make us who we are. SMA is caused by a mutation in the SMN1 gene, and this mutation is the reason why we have this terrible disease. Everyone has a copy of the SMN1 gene, and it’s known as the SMN2 gene. This SMN2 gene produces the same type of protein that the SMN1 gene produces, only at lower amounts.
Spinraza works by trying to repair the SMN2 gene. SMN is an acronym that stands for Survival Motor Neuron. Without getting too technical, think of it this way. If you take a sentence that is 10 words long, and remove the 7th word, this sentence would not make sense. This missing 7th word is known as the 7th exon. When this 7th exon is removed, it produces a faulty message, which in turn, makes this an insufficient or defective protein.
Spinraza, also known as Nusinersen, fixes this SMN2 splicing error. Researchers and scientists have developed Spinraza to bind this missing or removed exon 7 sequence back in the genetic string, essentially putting this missing 7th word back in the sentence. Binding is just a process where this 7th exon will not be removed. After binding the exon 7 back in this genetic sequence, SMN2 becomes functional, giving those of us with SMA the protein that we need so that we can sustain the muscle that we already have, and possibly get stronger. While I know that my explanation may have been somewhat crude, I found an excellent video that fully explains this process.
ZOLGENSMA, while being the second FDA approved treatment for SMA, is the first gene therapy of its kind. Instead of working on the SMN2 gene, like Spinraza, ZOLGENSMA replaces the missing or defective SMN1 gene. Even though Spinraza repairs the SMN2 gene, the SMN2 gene still produces less functional protein than the SMN1 gene. Since ZOLGENSMA replaces the missing or defective SMN1 gene, this provides patients with a fully functioning SMN protein.
One thing that all of us need to remember is that Spinraza and ZOLGENSMA are the first of their kind. Four years ago, those of us with SMA had no means of stopping the progression of the disease. Now, we have two viable treatments, and many more groundbreaking medical advancements on the way.
June 17, 2019 at 1:04 pm #19413Daria LavrovskaParticipant
I don’t know, if it’s a proper topic for my question, so sorry in advance in case it is not.
I was said, there were several cases in the USA, when an insurance company covered Spinraza treatment and Zolgensma simultaneously (i.e, patient gets both: Zolgensma as one time treatment and Spinraza constantly).
It sounds a bit weird and I can’t understand the purpose of such conjunction. But the question is if such coverage of these two drags really takes place in the USA/ possible in general?
June 17, 2019 at 1:56 pm #19415Michael MoraleKeymaster
Daria – I know that there had been a few cases where some individuals have taken both Spinraza and Zolgensma, but I think these were just test cases during some of the clinical trials. I believe they were testing whether or not these 2 treatments would be able to work together, since Spinraza is a cellular-based therapy and Zolgensma is a gene-based therapy. It’s probably unlikely that there will be a combination treatment between these 2 drugs, but I guess that is still yet to be determined by researchers and medical professionals. You actually ask a very good question, and I believe this is a question that quite a few people have had. I used to teach college for 18 years, and I used to tell my students that the only stupid question is the one that’s not asked, so thank you for your question.
I know that there is a treatment coming out maybe by the end of this year or sometime in 2020, and it’s called Risdiplam. I’ve also heard quite a bit of chatter that Risdiplam will work on the SMN2 gene, similar to how Spinraza works. I believe they are looking at the possibility of a combination between Risdiplam and Spinraza, but again, this is still in the clinical trial stage. We at SMA News Today will be giving our members more information regarding Risdiplam in the coming weeks and months, so be sure to keep checking in for this information.
If you have any further questions, please feel free to let us know. Thanks again for your question have a great day.
June 17, 2019 at 2:20 pm #19418Daria LavrovskaParticipant
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