A new version of my book on ALS research

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The previous revision of this book was done in February 2021. The new version has fewer pages because it focuses on research and less on clinical trials.

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At that time, we were still awaiting the results of clinical trials with Arimoclomol. What a disappointment after that of Nurown! There may be a few lessons in this debacle, that a small molecule cannot help stop the progression of ALS (the corollary being that ALS is not due to single molecular dysfunction like a failed cell receptor) and that a single drug cannot be effective in the face of the diversity of cases encountered in a clinical trial.

There has been little news on the research front in 2021, especially compared to 2020.

On a regulatory front, what is a bit surprising is the turnaround of the FDA decision on AMX0035 under. the pressure of public opinion. AMX0035 is not a cure, during a clinical trial it can extend life expectancy by 6 months, which is both a lot and too little.

The only therapy that would be effective in stopping the progression of most cases of ALS is a TDP-43 therapy. A number of these therapies have been designed in laboratories for a few years, but no company has taken the risk of doing a clinical trial. Instead, companies prefer to bet on new compounds made from existing drugs, which helps speed up the passage of regulatory hurdles. A godsend for any investor including, curiously, some NGOs.

This once again illustrates how disorganized our pharmaceutical industry is. The biotechs care little about academic research, which is of poor quality anyway. Biotechs just want a quick payoff as they are essentially a gamble for investors, but they have a high death rate anyway. Large companies wait for successful biotechs, but generally do not research rare diseases because they are deemed unprofitable.

The new version of the book has also been updated in the last part which deals with the generation of new motor neurons in-vivo. This is the only way to restore health. Yet we have learned that new motor neurons derived from the patient's astrocytes are also carriers of the disease, so there is a phenomenon here that is not well understood.

Perhaps ALS and other neurodegenerative diseases belong to a spectrum of diseases where cells are not functional, for example because they are in a perpetual UPR / ISR state. An Italian clinical trial hints at something like this, let's hope Sephin1 / IFB-088 will be tried soon and give good news.

The French book is still not updated.

Otherwise the best advice ALS patients could be given in 2021 is still to keep their BMI at 27.

Jean-Pierre Le Rouzic Please do not hesitate to send me any feed-back

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