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Tauro-Urso-Deoxycholic Acid Trials in Amyotrophic Lateral Sclerosis: What is Achieved and What to Expect

Phase II studies on tauro-urso-deoxycholic acid (TUDCA) raised the promise of safety and efficacy in patients with amyotrophic lateral sclerosis, a currently incurable and devastating disease. We review the available evidence on the efficacy and safety of TUDCA, administered alone or in combination, by analyzing and comparing published and ongoing studies on amyotrophic lateral sclerosis. Two independent phase II studies (using TUDCA solo or combined with sodium phenylbutyrate) showed similar efficacy in slowing disease progression measured by functional scales. One open-label follow-up TUDCA+sodium phenylbutyrate study suggested a benefit on survival. Two subsequent phase III studies with TUDCA (solo or combined with sodium phenylbutyrate) have been initiated and are currently ongoing. Their completion is expected by the end of 2023 and beginning of 2024. Evidence collected by phase II studies indicates that there are no safety concerns in patients with amyotrophic lateral sclerosis. The efficacy shown in phase II studies was considered sufficient to grant approval in some countries but not in others, owing to discrepant views on the strength of evidence. It will be necessary to wait for the results of ongoing phase III studies to attain a full appreciation of these data.

 

Comments:

It sounds like the research on TUDCA for amyotrophic lateral sclerosis (ALS) is showing promising results, particularly from phase II studies. These studies demonstrated safety and suggested efficacy in slowing disease progression when TUDCA was used alone or in combination with sodium phenylbutyrate.

The similarity in efficacy between the two independent phase II studies is encouraging, and an open-label follow-up study even hinted at a potential benefit on survival. However, the discrepancy in approval across countries might be due to differing opinions on the strength of evidence presented in these studies.

The ongoing phase III studies are crucial for confirming and expanding upon the findings from phase II trials. These studies, expected to conclude by the end of 2023 and beginning of 2024, will provide more comprehensive data on both the safety and efficacy of TUDCA in ALS patients.

Given that the evidence from phase II studies showed no safety concerns and indicated sufficient efficacy to warrant approval in some countries, the completion of phase III trials will likely provide a more comprehensive understanding of TUDCA's potential in treating ALS. This fuller picture may influence regulatory decisions in countries where approval is pending.

It's essential to await the results of these ongoing trials to have a clearer understanding of TUDCA's role in managing ALS and its potential for wider approval and adoption as a treatment option.

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