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    • #22171
      Dagmar Munn
      Keymaster

      Tofersen is an investigational drug also known as BIIB067. It is an antisense oligoneucleotide (ASO) targeting SOD1. Mutations in the SOD1 gene appear in about 12-20 percent of people with familial ALS and 1-2 percent of people with sporadic (or singleton) ALS.

      Among the ongoing clinical trials being conducted for possible ALS treatments, Tofersen is one with promising results. You can read more about Tofersen here: https://www.als.org/research/emerging-drugs/tofersen

      If you have the SOD1 gene mutation, have you been following the news updates about Tofersen? Have you participated in one of their trials? What do you think of this potential ALS treatment?

    • #22179
      John Ceriotti
      Participant

      I participated in the closed portion of the study for six months.  Then I was allowed to participate in the open label extension and have been now for 13 doses or approx 1 year.  Open label gets you the actual medicine, Toferson, and so that is good.

      I am cautiously optimistic with the results and subjectively feel better, feel more motivated, and the Drs. read hard data from various physical tests, as well as blood tests, that indicate the medicine is doing what it was designed/created to do.  In fact Dr. Timothy Miller from Washington University here in St Louis, created the drug and recently spoke to a large group of colleagues in Scotland.

      You can read about it in one of the last few ALS Today e-newsletters.  My wife and I are positive on the results Toferson has provided, and I continue to want to push my body to do more things.  I remain very cautiously optimistic that Toferson is a very well targeted compound for the familial SOD-1 mutant gene…helping me to this point feel better and believe progression of the ALS has slowed.  I am realistic and not creating a false long-term prognosis for myself as I heavily rely upon the strong academic minds at Washington University.

      Hope this helps others!!!

    • #22192
      Amanda
      Keymaster

      My family has the SOD1 mutation which I also inherited. I do my best to keep up with the information regarding Toferson and the related trials. The Pre-fALS study that I participate in also contributes to this study and many of the participants were in the clinical trials. I’ve been told that as soon as I show significant enough symptoms that I will be a candidate. From what I’ve read and from what the researchers have told me, this treatment is very promising for ALS related to the SOD mutation. Personally, I am holding out a lot of hope for this drug to help pALS with the SOD1 mutation. I know that more studies and trials are needed in order to prove it’s effectiveness (hopefully) but it is definitely one step in the right direction.

      Amanda

      • This reply was modified 1 week, 6 days ago by Amanda.
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