Iron Chelation Therapy

Case Number CH-016 and CH-028

Inventor: Elizabeth Theil, PhD

Patent No. Title Issue Date Country
5,834,199 Methods of Identifying Transition Metal Complexes that Selectively Cleave Regulatory Elements of mRNA and Uses 11/10/98 United States
  Enhancement of Iron Chelation Therapy   United States
  Enhancement of Iron Chelation Therapy   PCR

Abstract
Iron overload is a complication of the treatment, by chronic transfusion, of a number of genetic diseases that lead to excessive iron absorption from the diet.  Two relatively common anemic conditions, sickle cell disease and thalassemia, can result in iron overload from hypertransfusion.  Since the genes that lead to iron overload (either congenitally or indirectly from transfusion treatment) are very common in the population, the combined frequency of carriers for the diseases that can involve iron overload approaches 20% in North American and European populations alone.  Frequencies as high as 10% are observed in Africa, Southeast Asia and the Mediterranean.

This invention provides methods of enhancing the rate of iron release from native ferritin, such that chelation of released iron is facilitated, thereby treating conditions associated with iron overload.  This invention also provides methods and reagents that are useful for identification and characterization of compounds that enhance iron release from ferritin.

Advantage
Current therapies involve long periods of intravenous or subcutaneous administration (up to 24 hours/day for at least 5 days/week) of an iron chelator.  Such long-term treatment is expensive and impractical for most of the world's population.  The drug predominantly used for treatment oral administration, desferrioxamine, is also expensive and has presented some undesirable side effects, which encourages lack of compliance.  Further, oral administration greatly reduces treatment efficacy as compared to intravenous or subcutaneous administration.

By increasing the amount of iron available for chelation, the invention provides methods and reagents for treating conditions associated with iron overload.   Another utility of the invention is its potential application for screening different types of agents for their ability to enhance iron release from ferritin.

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