High-dose GM1 shows promise in treating spinocerebellar ataxia type 3

High-dose ganglioside GM1 (ganglioside-monosialic acid) significantly improves ataxic symptoms in patients with spinocerebellar ataxia type 3 (SCA3) and is well-tolerated, according to results from a randomized controlled trial.

Researchers evaluated the safety and efficacy of GM1 in 48 patients with SCA3, who were randomly assigned to receive high-dose GM1, low-dose GM1, or a placebo for 4 weeks.

After 12 weeks, the high-dose GM1 group showed significant improvement, with a mean reduction of 3.80 points on the Scale for the Assessment and Rating of Ataxia compared to minimal changes in the low-dose and placebo groups. Secondary outcomes, including improvements in the International Cooperative Ataxia Rating Scale and activities of daily living, further supported the benefits of high-dose GM1. The treatment was well-tolerated, with no major safety concerns.

These findings suggest that high-dose GM1 could be a promising therapeutic option for SCA3.

Reference
Chen YK, Tian HY, Zhu QY, et al. Potential Disease-Modifying Effects of Ganglioside GM1 Pulse Treatment on Spinocerebellar Ataxia Type 3, a Parallel-Group, Double-Blind, Randomized, Controlled Trial. Mov Disord. 2025;40(1):57-66. doi: 10.1002/mds.30050. Epub 2024 Nov 7. PMID: 39508583.