When I was a stroke fellow, UCSD was the coordinating center for a multicenter trial of a promising device called NeuroThera, made by a local company called PhotoThera. NeuroThera was kind of like an ultrasound machine, except that instead of insonating tissue with ultrasonic waves, it illuminated tissue with near-infrared light. There is pre-clinical evidence that cytochrome C oxidase (the last complex in the electron transport chain) responds to stimulation with near-infrared light by increasing ATP production. The theory was that driving ATP production in the ischemic penumbra may salvage some brain tissue and improve outcomes after stroke. A phase II study called NEST-1 was positive, and we were coordinating and participating in what was hoped to be a definitive, phase 3 study, NEST-2.
So, my co-fellows and I ran around San Diego shaving the heads of stroke patients who consented to study participation and illuminating their brains with the NeuroThera device, as pictured above. If I remember correctly, it was 2 minutes per site times 20 sites–something like that. The study was negative, but there were some encouraging signals in patients with mild-moderate stroke severity and superficially-located infarcts. NEST-3 was designed to focus on these patients.
Turns out that NEST-3 was stopped early by its data safety and monitoring board for futility, and what happened next is tragically fascinating. The study was funded through venture capital, and the investors pulled out of the venture upon learning of the futility analysis. This left no money to properly close out the study and publish the negative results. For example, all of the company’s employees were abruptly terminated, and so everyone who hung on to ensure that the study was wrapped up in a responsible manner worked for free. Wow.
The study manuscript was published online in Stroke yesterday, and there’s an accompanying editorial that goes into more detail about the way this played out. The editorialists make the sensible suggestion that money be escrowed during such studies to ensure that they can be closed out properly should investment be abruptly withdrawn.