Oral tauroursodeoxycholic acid Effective in Prolonging Survival for ALS Patients: Lancet

A recent retrospective study conducted in the Emilia Romagna Region of Italy suggests that oral tauroursodeoxycholic acid (TUDCA) might extend the survival of those suffering from the devastating neurodegenerative disease, Amyotrophic Lateral Sclerosis (ALS) patients. The findings were published in the Lancet eClinical Medicine Journal.

ALS, often referred to as Lou Gehrig's disease, is a progressive condition with limited treatment options. However, this study investigated the real-world impact of TUDCA on ALS patients' overall survival compared to those who received standard care only.

The study analyzed data from 627 ALS patients diagnosed between January 2015 and June 2021, matching 86 patients who received TUDCA with 172 patients who did not, based on various factors such as age, sex, disease severity, and more.

The findings revealed that patients treated with TUDCA exhibited a median overall survival of 49.6 months, compared to 36.2 months in the control group. Notably, patients exposed to a higher dosage of TUDCA (defined as ≥ 1000 mg/day) experienced a significantly reduced risk of death, with a hazard ratio of 0.56, indicating a 44% lower risk compared to the control group. The drug was generally well-tolerated, with only a minority of patients discontinuing treatment due to mild gastrointestinal side effects.

These results provide a glimmer of hope for ALS patients and suggest that TUDCA may have the potential to prolong survival in this patient population. However, it's important to note that this was a retrospective study, and further prospective randomized trials are needed to confirm the drug's efficacy and safety conclusively.

ALS is a devastating disease characterized by the progressive loss of motor neurons, leading to muscle weakness and paralysis. Currently, there is no cure for ALS, and available treatments aim to manage symptoms and improve the patient's quality of life.

The potential benefits of TUDCA are significant, offering a potential lifeline to those affected by this debilitating condition. Continued research and clinical trials will be crucial in determining the drug's true effectiveness and safety profile in ALS patients.

Source:

Zucchi, E., Musazzi, U. M., Fedele, G., Martinelli, I., Gianferrari, G., Simonini, C., Fini, N., Ghezzi, A., Vacchiano, V., Zinno, L., Anceschi, P., Gessani, A., … Ganino, C. (2023). Effect of tauroursodeoxycholic acid on survival and safety in amyotrophic lateral sclerosis: a retrospective population-based cohort study. In eClinicalMedicine (Vol. 65, p. 102256). Elsevier BV. https://doi.org/10.1016/j.eclinm.2023.102256