Are there depleter drug trials available for wild-type amyloidosis? Great question — and there is some promising research happening in this space!
While most currently approved treatments for wild-type ATTR amyloidosis are stabilizers (which prevent the transthyretin protein from misfolding), researchers are actively Show Full Answer
Are there depleter drug trials available for wild-type amyloidosis? Great question — and there is some promising research happening in this space!
While most currently approved treatments for wild-type ATTR amyloidosis are stabilizers (which prevent the transthyretin protein from misfolding), researchers are actively studying "depleter" type drugs that work differently — by reducing how much transthyretin protein the liver produces in the first place. The following gene-silencing drugs work as depletors and are being studied or used in this area:
- Eplontersen — currently in clinical trials for wild-type ATTR amyloidosis
- Inotersen — also being studied in clinical trials for wild-type ATTR amyloidosis
- Patisiran — another gene silencer under investigation for wild-type ATTR amyloidosis
- Vutrisiran (Amvuttra) — already FDA-approved for cardiomyopathy caused by wild-type amyloidosis
These drugs work by silencing the gene responsible for producing the transthyretin protein, which helps prevent amyloid buildup and organ damage.
Speaking with a specialist about eligibility for any of these trials would be a worthwhile next step.
June 28
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