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FDA approves tofersen, now Qalsody, as treatment for SOD1-ALS
FDA approves Tofersen, now Qalsody, as a treatment for SOD1-ALS. This decision marks the first conditional approval for ALS in the U.S. Qalsody is the fourth approved therapy to treat a form of ALS and the first therapy to target a genetic cause of ALS.
Read more on ALS News Today:
https://alsnewstoday.com/news/fda-approves-tofersen-now-qalsody-treatment-adults-sod1-als/
What do you think of this news?
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18 Replies
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Congratulations to all who have SOD1-ALS for finally getting access to this life-extending treatment!
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Congrats to the 10%. – How can we sporadic pALS get the research companies to trial their targeted gene drugs on us? Â I have an SOD1 mutation, along with others but not enough to allow me access to Tofersen. Â It is a crime.
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Lisa,
Don’t give up hope because the technology will be beneficial in finding treatments for ALS in general. I know they are already working on a similar treatment for the c9.Have you been working with any of the doctors affiliated with the research centers? If not, reach out to them. I wasn’t a candidate for the Tofersen trials because the SOD1 mutation that didn’t meet the criteria. Now, I’m eligible for the treatments.
We all need to continue advocating and making noise so that our entire community has access to treatments!
Amanda-
Thank you Amanda. Â I wish there were doctors let alone clinics that could help pALS in New Zealand! Â Access to drugs is made doubly difficult by our drug company laws here.
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Lisa,
Have you been in contact with University of Auckland? If you are like me you have researched everything possible! How did you find out you had the SOD1 mutation. Were you not eligible because it wasn’t associated with a rapid progressive variant? I’m not as informed about sporadic ALS, but I do recall reading that many sporadic cases also had a mutated gene. I’m not sure what separates it (sporadic vs genetic). Could it possibly be a new mutation and there aren’t other family members? I will be googling and reading tonight to learn more!
https://www.mndresearch.auckland.ac.nz/cool_timeline/early-access-program-for-tofersen-in-sod1-als/
You are right, it is a crime!
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Lisa,
Have you been in contact with University of Auckland? If you are like me you have researched everything possible! How did you find out you had the SOD1 mutation. Were you not eligible because it wasn’t associated with a rapid progressive variant? I’m not as informed about sporadic ALS, but I do recall reading that many sporadic cases also had a mutated gene. I’m not sure what separates it (sporadic vs genetic). Could it possibly be a new mutation and there aren’t other family members? I will be googling and reading tonight to learn more!
https://www.mndresearch.auckland.ac.nz/cool_timeline/early-access-program-for-tofersen-in-sod1-als/
You are right, it is a crime!
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Yes I had a gene study performed by Auckland University, however hey were only looking at a certain number of genes. Â I have small mutations in several but not enough to classify as a singular variant. Â I am currently trying to find the best place to have the full gene investigation – any ideas?
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Other than going through your doctor I’m not sure. Hopefully someone from the forum will chime in!
Amanda
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Amanda, I attempted to message you but kept getting error messages . . . did a message get through?
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Biogen is stating they are trying to get QALSODY (Tofersen) out to physicians in a week. Some places will need training on the treatment procedures and drug. The University of Miami is setting up a “Tofersen” Clinic for treatments.
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Yes i too am blessed as a slow progresser. Please dont give up hope. I look at it as i do what i can with tools God gave me. So i participate in studies to try to improve upon capabilities for earlier diagnosis.
Im hoping this gives you all a better chance to get into studies earlier.
God Bless You!
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Hello, does anybody knows, if BIOGEN is starting / planNing a study with Qalsody for people with the SOD1 mutation without having a diagnosed ALS, as a prevention therapy? Or do you know more about using Qalsody as a ALS blocker? Thanks and best regards, Peter
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Hi Peter,
I hope this is helpful.
Yes, I believe that study has started.
Contact form https://www.alsatlasstudy.com/
https://classic.clinicaltrials.gov/ct2/show/NCT04856982
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
alsatlasstudy.com
The ATLAS Study is now enrolling adults who are carriers of certain SOD1 gene variants. See if you may qualify for the ATLAS Study.
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I have sod1 mutation and have been on Toferson since June. I was diagnosed with als. Unless you have been diagnosed with als they won’t treat you. Not everyone with the mutation gets als. If you are diagnosed with als start taking Toferson. The sooner the better.
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Terry,
The research they are doing now is looking for elevated biomarkers that seem to show up prior to diagnosable symptoms. The goal is to be able to start Tofesen/QALSody at this stage to prevent the onset of symptoms. It’s amazing how far they have come.
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I’m 49 and I live in Prévost, Quebec, I started Tofersen treatments 2 months ago. I’m really looking forward to seeing if I respond well to the medication. Here we have access to 4 medications:
* Exservan (riluzole oral film)
* Radicava (edaravone)
* Relyvrio (sodium phenylbutyrate and taurursodiol) is an oral treatment
* Qalsody (tofersen)
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Dr Timothy Miller and Dr Robert Bucelli (both helped create the drug) have a two days a week Tofersen clinic at Washington University Medical Center in St. Louis.
I have been in the original trial since Dec 2020 and after 32 doses in the Extended Access portion of the trial every 4 weeks I feel so good with 7/9 maintained or improving markers with an every 6 months MUNEX nerve test!
I had to stop hard physical therapy after some emergency surgery in July this year and am planning a re-Assessment and start up In December and January.
I strongly feel the therapy was a great complement to Tofersen’s treatment and was improving my quads glutes etc to start walking within the year!
Washington University is developing a significant Physical Therapy program targeting PALs overall
I hope everyone can get any or all access to Tofersen…Dr Miller and Dr Bucelli developed a great drug with Biogen.
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The street value of what you received was a little more than a half million dollars. Once again US drug companies want their profits. At roughly 200,000 dollars a year. Who can afford it? Even the most generous insurance company will probably only pay 80%. Even that will cost the family 40,000 dollars a year. With that and other expenses replayed to this disease , what family would be solvent? Who says we need a compassionate health care system we have the money can buy!
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