Molecules that curb errant proteins of AL amyloidosis point to new type of therapy

(Scripps Research Institute) Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease. The drug mechanism that the team identified is analogous to a different drug that also originated at Scripps Research: Tafamidis, which stabilizes the protein transthyretin to treat the most common systemic amyloid disease that affects the heart or other organs.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news

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Blood Cancer Journal, Published online: 14 April 2020; doi:10.1038/s41408-020-0306-5A validated composite organ and hematologic response model for early assessment of treatment outcomes in light chain amyloidosis
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Conditions:   Cancer;   Cardiovascular Diseases;   Hereditary Cancer;   Hereditary Cardiac Amyloidosis Interventions:   Behavioral: e-Health (web-based) disclosure portal;   Behavioral: Provider mediated disclosure Sponsors:   University of Pennsylvania;   Fox Chase Cancer Center Enrolling by invitation
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
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