Article:

A recent study conducted in Italy has indicated that TUDCA, a bile acid salt traditionally used to treat liver disease, may have a beneficial effect in amyotrophic lateral sclerosis (ALS) patients by potentially prolonging their survival. While ongoing Phase 3 clinical trials are still underway to confirm these findings, the study provides encouraging insights into the potential efficacy and safety of TUDCA as an oral therapy for ALS.

ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord, leading to muscle weakness and eventual paralysis. Currently, there is no cure for ALS, and available treatments are limited in their ability to slow disease progression.

The Phase 3 TUDCA-ALS clinical trial, sponsored by the European Commission, is evaluating the efficacy of TUDCA as an adjunct treatment to standard therapy with riluzole in individuals with recent ALS. The trial is expected to conclude later this year, shedding more light on TUDCA's potential benefits in ALS management.

TUDCA is also a key component of Relyvrio, a medication approved for ALS treatment in the United States and Canada, marketed as Albrioza in Canada. Amylyx Pharmaceuticals, the developer of Relyvrio, is conducting the PHOENIX Phase 3 study (NCT05021536) to further confirm the treatment's effectiveness.

In the Italian study, researchers analyzed data from 86 ALS patients who received TUDCA and compared it with data from 172 patients who did not use the therapy. The two groups were matched based on various factors including sex, age at onset, diagnostic delay, and disease severity.

The results showed that patients who received TUDCA at daily doses of 1,000 mg or higher had longer median survival times of 56.5 months compared to those who received lower doses (29.7 months) or were not treated with TUDCA (36.2 months). Statistical models indicated that high-dose TUDCA treatment reduced the risk of death or tracheostomy by 55% compared to patients who did not receive TUDCA.

"While these findings are promising, it's important to note that this analysis is based on real-world data, which may have certain limitations compared to clinical trials," cautioned the researchers. Therefore, further confirmation of TUDCA's efficacy and safety in ALS will be obtained from ongoing clinical trials.

It is worth mentioning that approximately 20% of patients treated with TUDCA in the study experienced side effects, such as diarrhea, abdominal pain, or rash. However, most of these side effects were manageable with drug reduction. Only a small percentage of patients discontinued treatment due to intolerable side effects.

Overall, these findings provide preliminary evidence supporting the potential of TUDCA as a therapeutic option for ALS patients, particularly when used at higher doses. However, further research is needed to establish the definitive impact of TUDCA on ALS outcomes and to better understand its safety profile.