Amyloid formation plays a role in over 20 human diseases including Alzheimers disease, type II diabetes and the systemic amyloidoses. Dialysis-related amyloidosis (DRA) occurs in dialysis patients when -2 microglobulin builds up in the blood and deposits in the joints as amyloid. DRA eventually affects all long-term kidney dialysis patients and is only effectively treated by kidney transplant; if left untreated, the systemic amyloidosis can be deadly. Over 300,000 people in the United States and over 1 million worldwide are on kidney or peritoneal dialysis. Finding a treatment to dissolve the -2 microglobulin fibrils would provide the only alternative to a kidney transplant for these patients.
Researchers at UCLA have identified 2 compounds that disrupt preformed human -2 microglobulin fibrils in a dose-dependent manner at concentrations that are clinically relevant. Both of these compounds have been proven safe in clinical trials for other diseases and could be readily administered during dialysis. These compounds were identified using a high throughput screen developed by the researchers for the identification of drugs that dissolve fibrils. The developed assay can be used to find drugs that dissolve fibrils associated with other human diseases.
|United States Of America||Issued Patent||9,463,195||10/11/2016||2006-584|