Amyloidosis happens when misfolded proteins build up in organs, damaging the heart, kidneys, liver, or nerves. Light-chain (AL) amyloidosis stems from abnormal plasma cells, while hereditary forms trace to genetic mutations and wild-type affects mostly older adults. Treatment depends on the type — chemotherapy for AL, gene-silencing drugs for hereditary forms, and supportive care for wild-type cardiac amyloidosis.
What's actually going on in research
Trials are testing new proteasome inhibitors and antibodies for AL amyloidosis, CRISPR gene editing for hereditary forms, and monoclonal antibodies that aim to dissolve existing amyloid deposits. Researchers are also studying better ways to detect organ damage early and combination regimens that deepen responses in people whose disease returns.
Amyloid-clearing antibodies
Drugs like birtamimab and CAEL-101 target amyloid deposits directly, aiming to break them down. If successful, they could reverse organ damage rather than just stopping new deposits from forming.
CRISPR for hereditary forms
Gene editing aims to permanently silence the mutated gene that produces misfolded transthyretin. Early results show the approach can nearly eliminate production of the problem protein.
Deeper remissions in AL
Trials are testing whether adding daratumumab or other antibodies to chemotherapy improves outcomes. The goal is to drive minimal residual disease negative, which may lead to longer remissions and better organ recovery.
What to know before you search
Eligibility depends on amyloidosis type, organ involvement (especially heart and kidney function), prior treatments, and whether genetic testing confirms a hereditary form.
What types of trials are currently open
- Treatment trials for AL — Testing new chemotherapy combinations or antibodies like daratumumab to eliminate the plasma cells producing abnormal light chains.
- Amyloid-clearing trials — Testing antibodies that target and dissolve amyloid deposits already present in organs like the heart or kidneys.
- Gene therapy trials — Testing CRISPR or RNA-silencing drugs for hereditary transthyretin amyloidosis, aiming to stop production of the misfolded protein.
- Cardiac support trials — Testing treatments for heart failure symptoms in people with cardiac amyloidosis, including devices and medications to improve heart function.
- Biomarker studies — Tracking protein levels and imaging to understand how well treatments are working and how quickly organs recover.
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