Amyloidosis: Novel Treatments Could Improve Poor Outcomes

Systemic light chain amyloidosis is a clonal plasma cell disorder that can lead to end-organ dysfunction, with no approved treatment in the relapsed/refractory setting. Given this challenging clinical situation, two studies of novel therapies for this disease reported at the 2026 ASCO Annual Meeting were met with particular enthusiasm.

Although the phase III CARES study of anselamimab, a first-in-class kappa light chain–directed anti-fibril antibody, did not meet its primary endpoint in the overall population, the drug reduced the risk of death by 62% (hazard ratio [HR] = 0.38, 95% confidence interval [CI] = 0.17, nominal P = .012) in patients with kappa free light chain (FLC) involvement.1In a broader population, the bispecific antibody linvoseltamab-gcpt demonstrated meaningful clinical activity as a single agent in a phase I/II trial, producing a hematologic very good partial response or better in 100% of patients.2