Studies have previously shown that TUDCA has neuroprotective properties. A phase 2b clinical trial showed that patients who received TUDCA in addition to riluzole for 54 weeks had a prolonged median survival of 4-5 months. This is a much larger improvement than a few other ALS phase II clinical trials. It would have been on par with Riluzol and phase II of AMX00035 (Relyvrio).
Given that in online forums, people have praised TUDCA for many years I had great hope that a phase III clinical trial would succeed in demonstrating this prolonged survival time. In the meantime, phase III of AMX0035 failed to reach its objectives, which was a bit frightening, as AMX0035 was a compound of TUDCA and another common drug.
This TUDCA-ALS trial was developed to determine the clinical efficacy of TUDCA in ALS. It was funded by the European Union’s Horizon 2020 Research and Innovation program, a huge program to fund research in Europe. As a previous EU project leader (not in medicine) I had a lot of hopes for this project. It was also interesting as this project tested the drug for 18 months, three times the amount of usual clinical trials. Some scientists have expressed that six months may be too few to see improvements in ALS disease.
There was not a clear mechanism of action, but it's clear there is a relationship between neurodegenerative diseases and metabolism. As TUDCA is a bile acid synthesized from cholesterol in the liver, and as cholesterol is indispensable in the central nervous system (CNS), at least there are some hints that patients' interest in TUDCA is found. Las, and similarly to Arimoclomol where there were also hints at a mechanism of action to mitigate misfolded proteins, the project has announced today that it didn't meet its primary and secondary objectives.
We still have to wait to know more on this topic, but as was published a few days ago by a VC, researchers may have to reconsider the way they do research.