Jean-Pierre Le Rouzic

> If I turn my palm up I can do it. This makes no sense from the nerves in arm/hand perspective.
Intriguing! I guess that it makes no sense if we think of nerves as electrical conductor between the brain and the muscle. Here is a random speculation:

However it’s a bit more complicated. When we make a movement, the primary motor area in the brain…[Read more]

Thanks for the link, Richard, but it’s a work done in-vitro and for a mutation (VCP) that represents ~0.3% of all cases.
The odds that it could be useful for the average pALS are near zero.

> There are 4 or 5 clinics offering Stem Cell therapy also in India. They do not divulge any positive outcomes to the therapy.

In India as elsewhere, there have been official warnings about “stem cell clinics”.

Stem cells aroused a huge craze a few decades ago, it was seen as a solution to all problems. But in the end it didn’t solve any…[Read more]

Dr. Rothstein works in ALS research since a long time (~20 years?). But what is presented here is not very impressive. Working with iPSC-derived is something that could be done at little cost now, and (fortunately) many teams are working on ways to mitigate TDP-43 aggregates.
And it only pushes the goal post further away: What makes CHMP7…[Read more]

I think that a very important step has been achieved in understanding the mechanism of how misfolded TDP-43 aggregates in cells.

This is important as misfolded TDP-43 aggregates has been identified since 2006 by Virginia Lee has one of the most common pathological feature in all forms of ALS (> 97%), half of FLD dementia as well as a quarter of…[Read more]

Thanks for the link you gave, but it is a temporary file.
It can be downloaded again here

There is no reason that this drug is not moved up and approved now by the FDA for ALS?

I do not understand how data is impressive?

If you look at the figure 2 in the scientific publication you will see that there is a 3% difference between placebo group and groups that received Masitinib. each group contained ~30 patients, so I guess this is…[Read more]

There is no reason that this drug is not moved up and approved now by the FDA for ALS?

John, I do not understand what data is impressive here.

If you look at the figure 2 in the scientific publication you will see that there is a 3% difference between placebo group and groups that received Masitinib. each group contained ~30 patients, so I…[Read more]

Thanks John,

Nutrition is indeed of the utmost importance for pALS.

There are several calorie calculators for pALS, including on my website padiracinnovation.org
They are based on the work of Dr. Edward Kasarkis, Medical Director at the University of Kentucky.

There are bizarre and interesting stories on this topic, for example the mice animal model of C9orf72 that dies quickly at Stanford but where perfectly healthy at MIT.
After investigating the matter, the scientists (including Dr. Eggan) found that the mice didn’t have the same gut…[Read more]

Thanks Dagmar for researching some simple and easily comprehensible text on what is ALS, but I find this specific text extremely imprecise and confusing about what scientists know about ALS. And it lacks to tell about some areas, for example the genetic background.

> ALS is a heterogeneous disease,
There is no real diagnosis of ALS. It’s only a…[Read more]

Hi,

I wrote a book on ALS research in 2019 and I tried to update it every few months.
My last update was in March, after the Arimoclomol fiasco.
I waited to have more results, wrote to Amylyx, but did not receive any other information than what the press had.

The title of my book is “Status of Amyotrophic Lateral Sclerosis Research: From…[Read more]

I agree with you Steve, it’s not the same thing.

Do you have more information about what to understand with this text:

FDA Orphan Approval Status: Not FDA Approved for Orphan Indication

Thanks!

Marion,

A Orphan Drug Designation only means someone has asked FDA to examine the commercial preparedness of a drug.
Anybody can ask this to the FDA (try it)

It was indeed examined, but it was rejected. It is written in the link you posted.

Now, there is a lot of misunderstanding here. The FDA will not give an authorization market to a…[Read more]

Thanks for the link.

In the previous message you wrote:

as I believe they have just been granted Orphan Drug status

Do you agree that it is NOT approved for Orphan status?

FDA Orphan Approval Status: Not FDA Approved for Orphan Indication

In 2021 the European drug agency granted Orphan status to Ganglioside GM1 for treatment of ALS. It was presented by 3R Pharma Consulting GmbH, a consultancy organization with represent probably another organization.

As far I know there was no clinical trial about Ganglioside GM1.

> as I believe they have just been granted Orphan Drug status
@Marlon+Ness, I just checked the 2020 and 2021 pages of FDA Orphan drug status, and I can’t find any drug which was granted Orphan status for ALS for those two years. But that does not mean anything, for example Spinogenix claims it had been given this status for ALS this year, yet it…[Read more]