Tofersen ( Qalsody) gets conditional approval

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Nikki J

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What this means is it is approved but if ATLAS the carrier prevention trial fails the fda could rescind approval. However this is a great moment even though tofersen only benefits a small number of PALS. It is the culmination of many years of research and trials of two different sod1 asos ( the first was promising but needed tweaking. )

 
How I hope this helps. I have a friend from my home town and she is taking Tofersen. She gets NfL measured periodically. She was very slow progressing, from the beginning, so her only observation was that she seems to have more energy. She did have an improvement in her NfL by nearly 20%. These are the results we're looking for and hoping for.
 
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The people I know who are doing the best got it right away when diagnosed. I have hope that the carriers will do even better. ATLAS is designed so a bump in nfl puts you into a placebo controlled study. Anyone developing ALS either in the treatment trial or in the observation portion will get tofersen open label.

I also believe this changes advice on genetic testing. SOD1 family members should test. If you wait it can delay treatment while you get tested. For aggressive variants that delay could be deadly. I also think those with variants that qualify should do atlas
 
Nikki,

Do you mean all SOD1 variants should qualify for ATLAS ?

Thanks
 
No. ATLAS only accepts the more aggressive variants. There is an unpublished list that includes a5v and some others I don’t know. If someone has a variant not on their list they can appeal. I believe their family history is reviewed and if their relatives were fast progressing they get accepted. I know others who were rejected.
if you have sod1 ALS though it doesn’t matter which variant to benefit from tofersen. so it is worth knowing your status in case symptoms start you wouldn’t want to waste time waiting for your test results
 
Ok - I've got Progressive Bulbar Palsy and am looking for appropriate trials.

Thanks Nikki
 
are you sod1? You wouldn’t qualify for atlas but I would be pushing for someone to call bulbar als so you can get tofersen. If not sod1 tofersen isn’t indicated
 
Progressive and isolated bulbar palsy are als, right? I thought those were older terms but they now just classify everyone as bulbar onset als.
 
Also, very excited about the Tofersen approval. I’m eagerly looking forward to the Atlas results. It’s such an exciting drug.
 
Buglaw,

Regarding bulbar onset vs bulbar palsy, I guess it depends on what neuro or clinic you are working with. My understanding is Neuros cannot
make a probable (level) ALS diagnosis unless there are clear indications of both upper and lower motor neuron disease. Either upper or lower motor neuron damage by themselves would indicate a PLS kind of Dx - as I understand it.
 
Pure upper motor neuron disease is called PLS(Primary Lateral Sclerosis). People with pure PLS can live a normal or near-normal lifespan. They do have varying degrees of disability. Pure lower motor neuron disease is PMA (progressive muscle atrophy). Many doctors consider these to be on opposite ends of a spectrum. People with ALS have both upper motor neuron involvement and lower motor neuron involvement. Even so, some people are upper dominant ALS or lower dominant ALS.

Progressive Bulbar Palsy originates in the lower brain stem. It's a phenotype of ALS. Isolated Bulbar Palsy (not progressive) is a fairly benign condition.

Sorry, didn't mean to take the thread off topic.
 
My husband started with isolated bulbar symptoms with a clean EMG (for years). Even with the clean EMG, every neurologist we saw was willing to diagnose him with ALS, because progressive bulbar symptoms almost always progress to ALS (I never gave up hope he would be one of the lucky ones to not progress). Apparently after 4 years, they would have changed it to PLS (with a much better prognosis) but he died before then. If you have progressive bulbar symptoms, I would push for an ALS diagnosis so you can get the available drugs and possibly get into trials.
 
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