We’re studying BCMA-directed ... unprecedented, but regulators are mandating randomized clinical trials to prove the benefit of these transformative therapies. I find it a big issue when you have a rare disease to have to do a large, randomized trial, which can only be ... We’re studying BCMA-directed therapies in this space, and there are excellent response rates and safety profiles that are unprecedented, but regulators are mandating randomized clinical trials to prove the benefit of these transformative therapies. I find it a big issue when you have a rare disease to have to do a large, randomized trial, which can only be ethically done in the upfront setting, because there’s nothing approved in the relapsed setting. Heather J. Landau, MD, discussed promising results of CAR T-cell therapy in light chain amyloidosis and the challenge of developing trials for this rare plasma cell disorder. ... Amyloid light-chain (AL) amyloidosis is a disease with a more challenging prognosis than other plasma cell disorders like multiple myeloma due to cardiac and other organ involvement and lack of survival benefit from many established myeloma therapies. However, using B-cell maturation antigen (BCMA) as a target has shown promise for chimeric antigen receptor (CAR) T-cell therapy, opening up a new single-infusion treatment approach in this rare disease setting. In the phase 1/2 NEXICART-2 trial (NCT06097832), the investigational CAR T-cell therapy NXC-201 What was really impressive and different was what we saw was the patients who were sickest coming into the trial, who had high light chains or obvious organ progression due to their relapsing disease, experienced significant clinical benefit very rapidly—as soon as the light chains were controlled. I can’t say I’ve witnessed that with any other therapy that I’ve given.

Original title: “Deep Responses Support Push for CAR T-Cell Therapy in AL Amyloidosis | Targeted Oncology - Immunotherapy, Biomarkers, and Cancer Pathways”

concurrent light chain proximal tubulopathy and crystal storing histiocytosis in monoclonal gammopathy of renal significance a rare case report with 4 year follow up and literature review

Original title: “Concurrent κ light chain proximal tubulopathy and crystal-storing histiocytosis in monoclonal gammopathy of renal significance: a rare case report with 4-year follow-up and literature review.”

prevalence and prognostic significance of restriction versus systolic dysfunction in patients with transthyretin and light chain cardiac amyloidosis

Original title: “Prevalence and Prognostic Significance of Restriction Versus Systolic Dysfunction in Patients With Transthyretin and Light Chain Cardiac Amyloidosis.”