Cardiac amyloidosis future or investigational therapies: Difference between revisions
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Potential future treatment options targeting cardiac TTR amyloidosis include: | Potential future treatment options targeting cardiac TTR amyloidosis include: | ||
* AG10: AG10 is a selective, oral TTR stabilizer which | * AG10: AG10 is a selective, oral TTR stabilizer which mimics a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and achieve near-complete stabilization of TTR.<ref>{{Cite web|url=https://www.sciencedirect.com/science/article/pii/S0735109719339208?via%3Dihub|title=Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy|last=Judge|first=Daniel P.|date=10/29/2019|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref> A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130). | ||
* Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.<ref>{{Cite web|url=https://www.nejm.org/doi/10.1056/NEJMoa1716153?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dwww.ncbi.nlm.nih.gov|title=Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis|last=Adams|first=David|date=10/29/19|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref> | * Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.<ref>{{Cite web|url=https://www.nejm.org/doi/10.1056/NEJMoa1716153?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dwww.ncbi.nlm.nih.gov|title=Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis|last=Adams|first=David|date=10/29/19|website=|archive-url=|archive-date=|dead-url=|access-date=}}</ref> | ||
Revision as of 18:10, 30 October 2019
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Cardiac amyloidosis Microchapters |
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Raviteja Guddeti, M.B.B.S. [2]; Aarti Narayan, M.B.B.S [3]
Overview
Several investigational products targeting ATTR amyloid are being studied in clinical trials. These include small interfering RNA (siRNA) molecules which reduce the production of the amyloid precursor misfolded protein and ATTR stabilization molecules.
Future or Investigational Therapies
Potential future treatment options targeting cardiac TTR amyloidosis include:
- AG10: AG10 is a selective, oral TTR stabilizer which mimics a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and achieve near-complete stabilization of TTR.[1] A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130).
- Patisiran: this is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.[2]
References
- ↑ Judge, Daniel P. (10/29/2019). "Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy". Check date values in:
|date=(help) - ↑ Adams, David (10/29/19). "Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis". Check date values in:
|date=(help)