Unintended Consequences: The Story of PERF.

In the days before widespread internet use, iPhones, and iPads, the American Board of Psychiatry and Neurology part II oral board examination provided a unique opportunity for examiners to meet, mingle, and network. Sometimes interesting collaborations developed. Such was the case in 1994 when we had the same hour off on Dr. Alan Percy’s child neurology team. We were discussing infantile spasms and the reports out of Europe that vigabatrin was showing efficacy in treating infantile spasms.1,2 We thought, “Wouldn’t it be neat if we could get access to vigabatrin for our patients?” We knew Hoechst Marion Roussel had submitted a new drug application for vigabatrin for adult patients with complex partial seizures. We expected approval in the United States within 3-4 years and thought it would be great for our infantile spasms patients if we could get access to it while awaiting Food and Drug Administration (FDA) approval. Thus was born the journey that eventually would lead to the Pediatric Epilepsy Research Foundation (PERF). Over the summer of 1994, we developed a compassionate use protocol for treating infantile spasms with vigabatrin. This was presented to a preliminary group of investigators at the 1994 San Francisco Child Neurology Society meeting, most of whom would eventually form the Vigabatrin Infantile Spasms Study Group. Over the next 6 months or so, the details were hammered out and the Vigabatrin Infantile Spasms Study Group was formalized (see Table). Hoechst Marion Roussel agreed to provide an unrestricted grant and three large barrels (55-gallon drums with 165,000 pills) of 500-mg vigabatrin tablets to support the effort.