3 ms·
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:
by JPLeRouzic 7y ago
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-therapy-for-als http://padiracinnovation.org/News/2019/05/a-plea-for-a-gene-...
[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-fda/?utm_campaign=rss https://www.statnews.com/2019/06/07/als-advocates-protest-fd...
[2] http://prize4life.org/ http://prize4life.org/
[3] Eran Perlson and Erika Holzbaur, 2009