A Sidetracked, $1 Million, One-Person Clinical Trial to Receive Fresh Hearing Next Week at California Stem Cell Agency

It's a case of "n of one" and an extremely rare disease

Directors of the California stem cell agency will vote next week on whether to spend $1 million for a clinical trial involving only one person suffering from an extremely rare disorder known to affect only about nine persons worldwide.

The clinical trial was proposed by Joseph Gleeson of UC San Diego, who studies pediatric brain diseases and is director of neuroscience research at the Rady Children's Institute for Genomic Medicine in San Diego.

The single, unidentified patient involved in the trial is “a child with a severe neurodevelopmental disorder presenting with intractable epilepsy and severe neurodevelopmental delay,” according to a stem cell agency summary of the application.  (This link takes you to several summaries. Gleeson’s can be found by using its number, CLIN2-15085 .)