FDA Panel Advised Against ALS Drug, Cites Moral Injury

This story is near and dear as I have ALS and it invokes moral injury. In advising against US approval one panelist cited moral injury saying:

Creating false hope can be considered a moral injury and the use of statistical magic or manipulation to provide false hope is problematic.


Let’s unpack that. For one, all trials involve statistics, there’s nothing magical about them. Can they be manipulated? Of course, if you eliminate outliers for instance, you can move the numbers significantly. That though would be fraud, which is not alleged here. Can results be not statistically significant? Yes. Or results not be repeatable? Again, yes. For instance, psychological studies are notorious for not being repeatable. Why? Because they are based on subjective observation. Which leads to my next point.

All ALS progression is measured by subjective observation and aided by questionairre. Many questions are ambiguous. For instance, one of the questions is can you pull covers up yourself? This is problematic, what type of cover: sheet, blanket, duvet? It doesn’t specify so different people can refer to different things.

Another example is the arguably more objective lung function test. They sound objective but really aren’t. The tests are subject to which type of mouthpiece is used and how much coaching the technician administering gives. My own experience is illustrative. I had two tests three weeks apart. In the first test they used a round mouthpiece and the technician wasn’t clear about where I should place in mouth. In addition, because my ALS was bulbar onset, I already had issues with sealing my lips. That resulted in a reading of 50 percent lung capacity. The one that followed was administered with a football shaped mouthpiece. This result was 67 percent. Quite a variation! And with a progressive disease I didn’t gain 17 points.

The bottom line is that there are no real internal chemistry markers for diagnosis of ALS. So unlike other diseases where FDA will grant marketing go ahead for statistically insignificant results because there’s a biomarker change that is improved, with ALS that doesn’t apply.

All that aside, the glaring hole that our intrepid bioethicist quoted above is what gives anyone the right to restrict a 100 percent fatal disease patient from trying anything,? That’s the real moral injury that she conveniently fails to ponder.
I leave you with this quote, Even if it’s just placebo effect:

When Matt is on Nurown it helps him, when he’s off of it he gets worse,” said Mitze Klingenberg, speaking on behalf of her son, Matt Klingenberg, who was diagnosed with ALS in 2018.


Food for thought, average life expectancy is 3-5 years from first symptoms, Matt on this drug is at 5 and alive, I’m at 3.5. You decide.

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