SOD1 tofersen topline results

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Nikki J

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PALS
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Being presented Sunday at the neurology conference
 
The compassionate use program is also underway for rapid SOD1 FALS progressors.

Physicians may submit requests on behalf of eligible patients by emailing [email protected]
 
The results were trending positive but did not meet the primary endpoints. Unsurprisingly the earlier in progression people got it and the longer they were on it it looked a little better

next steps are not yet announced but for now biogen expanded their eap to all sod1 PALS if they live in a country where they are allowed. People should ask their neurologist if they want to go that route The neurologist would apply but bear in mind there are costs to administration that your clinic would have to bear
 
There was so much positive buzz from neurologists and participants as well—I follow two SOD-1 participants that have been receiving the OLE whose condition has dramatically slowed or actually improved—that I have no hope that any therapy currently in trial will meet its Phase 3 primary and/or secondary endpoints.

So many reasons in my opinion. Extreme heterogeneity, poor trial design in many cases, having to use the woefully inadequate ALSFRS questionnaire as the measuring stick absent biomarkers, FDA approvals that in my opinion don’t give enough credence to subsets of participants that responded, and on and on.

I haven’t fact checked this, but a forum member on the ALSTDI site said there have been more than 500 failed trials for MND and 2500+ failed trials for Alzheimer’s. Not a good batting average. I do not believe the discovery of truly revolutionary treatments for neurodegenerative diseases will occur within the next 25-50 years.

Having zero hope for the near term development—within five years—of an effective therapy (ie. halting or improving function for all subsets of pALS) is beyond what any former or current caregiver, family member, or close friend can truly comprehend. We can talk here all we want about living one day at time, getting our affairs in order, planning for DME, etc., all of which are logical and correct. But the loss of all hope is devastating to a pALS psyche. I take a Riluzole and number of supplements that my rational mind knows are of minimal to no value, but it’s all I’ve got.

And how in the hell did Radicava get approved?
 
Totally agree about frs which is why I constantly advocate for observational study participation. We only have nfl that is being used in these studies because of participants. Ditto the emerging PET biomarker. ATLAS - which I hope and think will fare better- is only happening because of the SOD1 gene carriers who participated in the Miami study. I knew some of the converters - none of the ones I knew are still here though a couple were in the phase 1 tofersen trial

it is my opinion that early intervention is key and we need better early diagnostic tools

radicava got approved because their subgroup trial met clinical endpoints for frs
 
Secondary endpoints faired better luckily. The FRS is horrible for clinical trials as you have mentioned. It varies wildly which neurons are effected and when, and the FRS does not provide a good measure of disease progression. It is also fairly subjective.
 
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