AI Insight
Systemic amyloidoses, which were previously fatal diseases without approved treatments, have become highly treatable conditions. Since 2018, six novel therapies have been approved for transthyretin (ATTR) amyloidosis, developed through advances in understanding the molecular mechanisms of protein misfolding and aggregation. Additionally, a combination therapy of daratumumab, cyclophosphamide, bortezomib, and dexamethasone was approved in 2021 for light chain (AL) amyloidosis.
Why it matters
This represents a significant transformation in the treatment landscape for rare, previously fatal diseases affecting multiple organ systems. The rapid development of multiple therapeutic options provides hope for patients with systemic amyloidoses and demonstrates how basic science research into protein misfolding can translate into clinical treatments.
Understand the Science
Once rapidly fatal, neglected, and orphan diseases without approved therapeutic options, systemic amyloidoses are now highly treatable. Basic scientific discoveries regarding the molecular mechanisms of transthyretin misfolding and aggregation have driven the development of drugs for the treatment of transthyretin (ATTR) amyloidosis, with six novel therapies approved since 2018. The combination of daratumumab, cyclophosphamide, bortezomib, and dexamethasone was approved for the treatment of light chain (AL) amyloidosis in 2021.