Healey D topline results

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The trial actually seemed reasonably encouraging for fast progressing patients. They "only" declined 7.5 points vs the 12 points for the placebo and had pretty substantial NFL decrease. That's a pretty marked change for those people. The voice biomarker also seems encouraging. It seems hopeful to me that we are seeing more trials "fail" like this where it does seek to be working for at least some people.
 
I think the science is better so they have clearer targets. I also suspect that things that show effect in the fast progressing probably re doing something for others that we can’t see in a short trial. For example the early trial results of tofersen were all about slowed progression in the aggressive onset group but there are people with less aggressive forms in ole who have been stable for several years.

I continue to believe that we need better biomarkers so we can dispense with frs as a primary endpoint and so that we can have earlier diagnosis. It is still going to be tricky for SALS to get to treatment early enough but we could certainly shave off several months if there were a simple test.
 
I do wonder about NFL serum tests. I’ve read a paper suggesting that they are close to as accurate as emgs at distinguishing between ALS and mimics. I know when I was going through the diagnostic process I had like 14 different blood panels done. One more wouldn’t be a big deal, and the results can come back relatively fast. One could imagine it being added to the standard initial blood tests when you go to your pcp with slurred speech or unexplained weakness. There is a very small list of other diseases which cause the sort of elevates serum nfl levels that ALS has.
 
I think nfl has some value. If elevated ( and no recent head trauma) it says something neurodegenerative is going on which would presumably help stop the ortho etc detours. It isn’t specific though and not everyone shows an elevation so I am hoping for something even better. But nfl is not that expensive ( the list price of mine was about 240 ) so maybe it will be more common
 
Nikki have you had an NFL test? How does yours compare with ranges found in research papers? I wonder if that correlated with your slow progression?
 
. It was elevated but not super high so maybe? I haven’t seen my neurologist yet to discuss
 
Nikki, I generally agree. Something conclusive would certainly be better, but it does feel like nfl tests could have more utility than just research. People with als generally have numbers that are seen in only a small number of diseases. So, it seems like it would be useful until something better comes along.
 
I agree. I justwant them to keep pushing for more ( which I think the genetic studies are)
 
Just had the results from my second Neurofilament test (had my second clinic visit last Thursday). Result was still elevated about 3 points higher than the first one but not super high. Time will tell if I am 2-5 year fatality or slower like some. I have sensory loss as well as motor so it is unlikely that my neural death will be minimal. Am still fighting with Accredo specialty pharmacy to get my Radicava ors and Relyvrio Rx-s filled. My insurance approved formulary exceptions last November but the pharmacy seems not interested in even finding what my copay might be. Will be interesting to see if Neurofilament stays high if I ever get on the other two R meds since I have been on Riluzole since September.
 
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