Oral Drug for Stimulation of Erythropoietin Levels in Anemic Patients

Erythropoietin (EPO) is a naturally occurring hormone that stimulates the production of red blood cells.  Recombinant EPO, marketed as Epogen® or Procrit®, have been used as a treatment for anemia and neutropenia, especially in those patients suffering from the effects of cancer chemotherapy, chronic renal failure or treatment for HIV.  Recombinant EPO is administered intravenously, with patients traveling to a clinic or hospital periodically to receive it.

UCSD investigators have designed artificial transcription factors that can activate specifically the endogenous gene for EPO in a patient’s own cells.  These factors, administered to those prone to anemia such as cancer patients, have the potential to combat anemia more effectively than Epogen® or Procrit®.

COMMERCIAL POTENTIAL: Compared to injecting manufactured recombinant EPO into the bloodstream of patients, this invention would allow the patient’s natural EPO to be produced.  It has the potential to be safer, less likely to trigger an immune response, less toxic, more convenient and less painful than injection.  The 2003 revenues for recombinant EPO from Amgen and Johnson & Johnson were approximately $7 billion.  Revenues worldwide may reach $15.4 billion by the end of the decade.  Epogen® or Procrit® are the most financially successful biopharmaceuticals in the world. 

The technology also has the potential to up-regulate many other gene targets, not just EPO.

Stage of development: in vitro data.

Publications:
Tachikawa K, Schroder O, Frey G, Briggs SP, Sera T.  Regulation of the endogenous VEGF-A gene by exogenous designed regulatory proteins.  Proc Natl Acad Sci U S A. 2004 Oct 19;101(42):15225-30