Pawel – My name is Michael Morale, and I’m the Director of Multichannel Content for BioNews Services, the parent company of SMA News Today. I wanted to take a few minutes and try to possibly answer some of your questions and concerns. First, before I began, the ultimate decision to switch from Spinraza to Zolgensma, needs to be made between you and your child’s pediatric neurologist. I’m not sure what the medical situations are in Poland, given the fact that it is a public health care type system, but my recommendation would be the same as what Kevin told you in his response regarding trying to find a doctor that has some working knowledge of both Spinraza and Zolgensma.
Zolgensma is only approved for infants and toddlers 2 years of age or younger, irregardless of the type of SMA that they have. Like Kevin, I too am on Spinraza. While I haven’t seen a great amount of muscle strength improvement in my arms, I do feel that Spinraza has halted the disease process, because I am not getting any worse. I’m now actively taking physical therapy and we are working on core strengthening exercises to improve not only my core strength, like my abdominal, back, shoulder and neck muscles, it’s also greatly improved my balance while sitting in my wheelchair. My breathing is exponentially better, but I’m not really sure if this is caused from Spinraza or my physical therapy.
Regarding your son Alex, I understand that he just received his 3rd injection of Spinraza. You need to understand that each person is going to react differently to this treatment. Some infants and toddlers will show more improvement than others, while some, may not show as much improvement in the same amount of time. You had mentioned something about a combination therapy between Spinraza and Zolgensma, but scientists and researchers are finding that this really is not showing any beneficial effects. Zolgensma works by replacing the SMN1 gene, and Spinraza works by repairing the SMN2 gene. These are 2 totally different approaches, because Spinraza is a cellular-based therapy while Zolgensma is the first FDA approved gene therapy.
Like Kevin stated in his follow-up to your question, Zolgensma is just now going into clinical trials for the intrathecal (IT) delivery for children and young adolescents, and depending how this clinical trial goes, they will start another clinical trial after that, and that will address the same issue regarding the IT delivery, but this will be for adults up to the age of 60 years old. All of this information regarding the last clinical trial is still tentative, because they will not know anything until the first IT clinical trial is completed.
Your child is still in the age range to possibly qualify for Zolgensma, but based on your healthcare system in Poland, this would be something that you would have to check with the health ministry for your child’s pediatric neurologist. I have quite a few subscribers and followers to my personal YouTube channel who live outside of the US, and I think a few of them even live in Poland. Each country bases their decisions on different criteria, so the only thing that we are allowed to tell you would be to please consult with your child’s pediatric neurologist and see if you can maybe find another doctor that has a better working knowledge of what SMA is and the treatments that are available.
Please do not quit the Spinraza treatments based on the fact that you would like to get your son on Zolgensma. Just because your son hasn’t seen too many benefits regarding Spinraza at the current time, like I said earlier, it may take a while for his body to start reacting to it. I would keep him on Spinraza as long as possible, until you are assured that he would be able to get Zolgensma. Alex, you and your family are in my prayers. Please keep us informed and let us know if we can help out in any other way.