Dizzying number of therapies

KevinM

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Much as I enjoy reading ALS News Today with its almost daily announcement of new trials, drugs, etc., (Ultomiris, AstroRX, Reldesemtiv, AMX0035, Ibudilast, to name just a few), which thrills me at first glance, I think I end up more discouraged and angry than excited.

I know this is a purely egocentric attitude and I should stand and cheer the volume of new ideas, but as Nikki pointed out previously, those News Today articles tend to be worded a bit more optimistically than they sometimes deserve. Just read today about Ultomiris entering Phase 3, which will start sometime in the next few months and then run for a freaking year! Just for the trials. For a drug that is already FDA approved, and is looking to be repurposed!

The researcher part of me gets it, but the PALS part of me throws my hands up, and I’m sure everyone here feels the same.

Right now, are there any therapies out there (assuming they work) that could somehow be approved in 2020? NurOwn might be most advanced in trials, but that’s 2021 under the current timeframe. Hope is so important, but it’s hard to find that hope knowing we are banking on somehow “hanging on” until something shakes out of the tree. More hope than 10 years ago, but still bad timing.

Okay, my mini-rant is over, and I know I’m covering familiar ground. But man, is it frustrating. Kevin
 

Nikki J

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I don’t see anything coming out imminently. I think Nurown and SOD1 aso are among the closest also Levosimendan and Arimoclomol are also in phase 3.

as someone who is newly diagnosed if you meet El Escorial criteria I would pursue a clinical trial of something. I believe the platform trial will have over 50 sites in the US (5 in Florida!)and is supposed to start in the next few months. There are more opportunities than there used to be for trial participation. Participating in trials and observational studies helps to advance the science if not for us for others
 

lgelb

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Maybe a little off topic, but you might be interested in this post and similar threads where we talk about things available to do today, with evidence behind them.

Best,
Laurie
 
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