Drug for Dialysis-related Amyloidosis

Tech ID: 20142 / UC Case 2006-584-0

Background

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.

Innovation

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.

Applications

  • Treatment of dialysis-related amyloidosis
  • It is anticipated the drug could be added to the dialysis solutions given to dialysis patients to treat or prevent DRA
  • The assay may be used to identify drugs that dissolve fibrils associated with other human diseases

Advantages

  • Compounds already have a proven safety profile
  • Treatment for DRA would occur during normal dialysis treatment directly into patient blood where fibrils are found

Patent Status

Country Type Number Dated Case
United States Of America Issued Patent 9,463,195 10/11/2016 2006-584
 

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Inventors

  • Eisenberg, David S.

Other Information

Keywords

therapeutic

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