Enteric-Coated Cystagon Study – Interim Report

Enteric-Coated Cystagon Study – Interim Report

I am pleased to report that so far we have been able to study enteric-coated Cystagon in 6 control patients.  Our results were somewhat variable, but overall did suggest that there would be a delay in the release of the cysteamine from the capsules.  Although this is beneficial as the stomach would potentially be spared direct contact with the drug, it is also possible (from the blood cysteamine profiles) that in two of the control subjects, the capsules actually dispersed in the colon.  We know that from previous studies colonic absorption of cysteamine is not high.  Overall, however, the results obtained from the control patients have been useful and we feel would warrant a publication in their own right.  They may be particularly helpful to those studying cysteamine for the treatment of neuro-degenerative disorders.

We have planned to study 4-5 cystinosis patients within the next 2 months.  Organizing these patients is particularly challenging as other institutions (and their Human Subjects Committees) have to be involved.  In addition, we are also waiting for our new TMS to be operational.  Hopefully we will be able to generate some useful results by the end of the year, and this will certainly give us enough information regarding the future direction of our study.

Ranjan Dohil, M.D.
Associate clinical Professor
Division of Pediatric Gastroenterology and Hepatology
University of California at San Diego