ALS Neuron Damage Reversed With New Compound

[jean-pierre-le-rouzic]

It looks like a therapy to degrade TDP-43. Since 2019 there were some experimental therapies. Initially the vehicle for the drug was a virus (i.e. a genetic therapy) but more recently the bearer was a peptide (for example having a TAT sequence) or similar mechanisms like PROTAC.

It’s interesting times, different teams are converging toward the same kind of therapy. But this does not mean a drug will appear tomorrow, several years and a few $M are necessary to make it a drug accessible on the market.

I think this kind of therapy will be able to stop the progression, but not reverse the disease, because many motor neurons have already died when one is diagnosed, and motor neurons do not regenerate. (but there are teams working on that last problem)

[jennifer-obrien]

Hi,  Richard,

I’m glad you flagged this. As the news release states, it’s early days (mouse/cell studies), requiring toxicology and pharmacokinetic studies prior to initiating a Phase 1 clinical trial, butI will certainly be following. A top university, a peer-reviewed journal.

Thank you for sharing!

Jennifer

[richard-maloon]

ALS Therapy Development Institute | ALS Therapy Development Institute

The above link may be worthwhile. Hard to sort out the “snake oil” from accepted treatment. I am doing a “snake oil” treatment using Melatonin.   J. Pineal Res. 2002; 33:186–187    ( also see updates)

Regarding TDP-43:

Maybe you can pretend you are a mouse and have them send you the compound. I didn’t like the term “administered” .

RM

[christine-moretti]

As someone with PLS, I was so excited reading this press release. Thanks for sharing this, Richard. Research on UMN will help all of us — ALS, PLS and HSP!

[Dagmar]

Finally, researchers are getting to the cause of ALS – – the misfolding of proteins – – and NU-9 helps ‘unfold them and refold them’ properly.

But Richard is correct: NU-9 isn’t able to recreate the motor neurons that have already died. That, is the second-half of the solution.

Let’s hurry up those mouse studies and have trials with human patients!

[alesia-johnson]

I am thinking the Scottish breakthrough in Edinburgh was reversing the damage.  Maybe these 2 groups should get together and compare notes.

[jean-pierre-le-rouzic]

You can find here my detailed analysis of this paper, you could jump to (my) conclusions.

I think that there is some potential here, but what strikes me the most is that they should have studied astrocytes because when a drug seems to affect only the UMN and not LMN, it is much more probable that it is the astrocytes that they affect.

[Deleted User]

I don’t think they can spark dead motor neurons back to life. They can stimulate nerve growth though. Nerve Growth Factors (NGF) can be stimulated with different substances (I have been using lions mane mushrooms). Nootropics stimulate nerve growth and that helps reinnervate denervated muscles (my interpretation).

[Dagmar]

You are correct John – – the research is at the level of identifying how to save motor neurons from dying and/or repair them: NU-9. But they haven’t figured out how to create news ones yet – – that would qualify for the Nobel Prize!

There are supplements that “support nerves” but, I haven’t read anything yet about their effect protecting motor neurons.

[Deleted User]

Developers site/Northwestern

 

ALS neuron damage reversed with new compound | Chemistry of Life Processes Institute (northwestern.edu)