ALS, a fatal neurodegenerative disease, leads to rapid decline in patients, with a mean survival time of only two to five years. The search for treatments that can slow this progression is crucial for improving the prognosis and quality of life for those affected.
BRAVYL’s Role in Transforming ALS Treatment Preclinical evidence has long suggested that BRAVYL (oral fasudil) positively affects ALS in animal models. Recent human data from the REAL Phase 2a study supports the potential of BRAVYL to slow disease progression, offering hope for better clinical outcomes.
REAL Phase 2a Study Findings The study, involving 31 participants at a 180 mg/day dose, found BRAVYL to be safe and well-tolerated, with no drug-related adverse events leading to withdrawal. A significant 15% decrease in NfL (a marker of neuronal damage) from baseline to 6 months was observed, suggesting BRAVYL could reduce NfL levels up to 30% compared to a placebo over the same period.
Impact on TDP-43 Pathology Beyond NfL reduction, BRAVYL treatment significantly decreased TDP-43 aggregation and cytoplasmic redistribution by 60% and 61%, respectively. TDP-43 pathology, characterized by its translocation from the nucleus to the cytoplasm and the formation of aggregates, is a known contributor to ALS progression.
Future Directions Encouraged by these findings, the REAL study has expanded to evaluate higher doses of BRAVYL, with results from the 300 mg/day cohort expected in June 2025. Woolsey Pharmaceuticals also holds extensive intellectual property rights for BRAVYL, including patents for its use in ALS and formulations designed to overcome dysphagia, a common challenge in ALS patients.
Conclusion The REAL Phase 2a study's results underscore BRAVYL's potential as a disease-modifying treatment for ALS, with significant implications for slowing disease progression and improving patient outcomes. However, it's important to note that BRAVYL is not yet approved for commercial use.