
Testing drugs on stem cells from ALS patients - dazosan
https://massivesci.com/articles/als-stem-cells-modeling-in-the-lab-treatment-breakthrough-new-drugs/
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JPLeRouzic
As someone who lost two people to ALS (and who risk loosing more) I strongly
hope a cure would be found soon.

Alas the proposal of this article is misleading: Models describe what we think
that we know about some phenomena. But as they say, all models are wrong.

Scientists spent many years (from 1993 to 2008) on ALS animal models based on
SOD1, before switching to TDP-43. SOD1, not only is a gene when 95% of cases
are sporadic, but it is a quite rare gene in ALS (2% of all cases). TDP-43
aggregations was shown, as early a 2009, to be a consequence of stressed cells
[3], not a cause of ALS, so why making two dozens animal models after
artificial diseases which we know are _NOT_ what is killing people?

Astonishingly scientists were and still are using mice to model ALS. There are
around 50 ALS mice models today. But _the prefrontal cortex (PFC), is much
less developed in rodents than in primates_. Rodents lack the direct
cortico–motor neuron projections[0] that support fine control of forelimb
movements. Many of the drugs used to treat CNS disorders exert their effects
via neurotransmitter and neuromodulatory systems, and many of these systems
differ between primates and rodents.

You may find surprizing that there are thousand patents about how "to cure
ALS" when no drugs are efficacious. Some biotechs are manipulating patients in
order to bypass FDA [1]. Invest your money carefully in ALS research! The
snake oil industry is working at full speed.

Prize4Life spent lot of money during a decade and then they stopped [2], they
understood the problem is not that more money is needed, the problem is that
ALS research is not focused, on contrary it is fragmented.

What we need is large pharmaceutical companies working to deliver an imperfect
drug in the short term. ALS organizations received a lot of money after the
"Ice bucket challenge", but it was mismanaged. _We do not need more research,
we need commitment to help people_. An imperfect solution is much better than
some elusive quest after "solving ALS".

Here is a concrete proposal, inspired by the recent, acclaimed and cynically
priced Zolgesma by Novartis (and it has a wide market, as it would help in 1/3
of Alzheimer cases as well):

[http://padiracinnovation.org/News/2019/05/a-plea-for-a-
gene-...](http://padiracinnovation.org/News/2019/05/a-plea-for-a-gene-therapy-
for-als)

[0] Lemon, R.N. Descending pathways in motor control. Annu. Rev. Neurosci.
195–218 (2008).

[1] [https://www.statnews.com/2019/06/07/als-advocates-protest-
fd...](https://www.statnews.com/2019/06/07/als-advocates-protest-
fda/?utm_campaign=rss)

[2] [http://prize4life.org/](http://prize4life.org/)

[3] Eran Perlson and Erika Holzbaur, 2009

