New Treatments & Clinical Trials for Amyloidosis

Amyloidosis occurs when misfolded proteins clump in organs, disrupting normal function. AL amyloidosis, the most common form, often affects the heart and kidneys. Treatment depends on the type: AL amyloidosis typically requires chemotherapy to stop the bone marrow cells making the abnormal protein, while ATTR amyloidosis now has RNA-targeting drugs that reduce production of the misfolded protein.

What's actually going on in research

Trials are testing new ways to silence or degrade the proteins that form amyloid, drugs to remove existing amyloid deposits from organs, and combinations that address both production and clearance. Research focuses heavily on preserving heart and kidney function, improving quality of life, and extending survival. Several RNA-targeting therapies approved since 2018 have changed the outlook for ATTR amyloidosis, and similar approaches are being tested for AL forms.

What to know before you search

Eligibility typically depends on amyloidosis type (AL, ATTR, or other), which organs are affected, severity of heart or kidney involvement, and prior treatments received.

What types of trials are currently open

• Amyloid-targeting trials — Testing drugs that directly remove or prevent formation of amyloid deposits in organs like the heart and nerves.
• Gene therapy trials — For hereditary ATTR amyloidosis, testing one-time treatments that turn off the gene making the misfolded protein.
• Combination trials — Testing whether combining a drug that stops amyloid production with one that removes existing deposits works better than either alone.
• Cardiac function trials — Studying treatments specifically for people whose amyloidosis has affected the heart, measuring survival and heart function improvements.
• Quality of life studies — Following people with amyloidosis to understand symptom burden, treatment side effects, and how new therapies affect daily function.