Investigators at the National Eye Institute and the National Institute of Child Health and Human Development are working with Sigma Tau Pharmaceuticals to prepare cysteamine eyedrops for testing in cystinosis patients. This is the first step in an attempt to bring an eyedrop preparation to New Drug Approval by the Food and Drug Administration, which would mean that the eyedrops could be prescribed by any licensed physician. The sponsorship of this venture represents an act of good will, since the small patient population makes this effort financially unprofitable. Current hopes are to begin a study of the new eyedrops in the summer of 1997. Cystinosis patients with severe pain due to corneal crystals may contact the NIH through the Cystinosis Foundation.

- William Gahl, M.D., Ph.D.
  Head, Section on Human Biochemical Genetics, NICHD

Current update 2/1/98

A protocol for the eyedrop study has been developed and is under review. Studies will probably start within the next six months. Continue to check this web page for updates.