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?