Arimoclomol may reduce the levels of protein aggregates in the motor nerves, a possible cause of ALS, by boosting expression of chaperonins Hsp70 and Hsp90 which help newly synthesized proteins properly fold.
We do not know if arimoclomol is of benefit to people with ALS. A phase II/III trial is ongoing. However, based on existing pharmacokinetic analyses, we feel more targeted chaperonin inhibitors may prove to be better candidates for ALS therapies.
Arimoclomol appears safe and well-tolerated according to a phase I clinical trial of 84 ALS PALS.
ALS mice treated late in the disease course experienced a boost in muscle function but no change in lifespan.
Arimclomol slowed progression and extended the lifespan of ALS mice treated early in the disease course nearly 25%.
The purpose of this study will be to demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with SOD1 positive familial ALS (fALS).
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