My inaugural post! Here goes.
The current issue of Movement Disorders has two papers on a late Phase II study of the GST-3β inhibitor tideglusib in PSP. The first, by Eduardo Tolosa et al, reports the failure of the drug to slow progression as measured by the PSP Rating Scale and a number of other clinical scales. But the second, by Günter Höglinger et al, analyzes a subgroup of patients from the same study who had MRIs before and after treatment. They found 58% less progression of cerebral atrophy in the active drug group relative to placebo. Most of the effect occurred in the parietal and occipital lobes, the neocortical areas least affected in PSP.
Full (really full) disclosure: I was a one of the co-I’s in the study, though not one of the sites doing the MRIs (they were the European sites); the primary outcome measure was a scale that I devised and published; I consulted for the industry sponsor in the study design; Günter Höglinger is my very good friend (as is Eduardo, but his paper isn’t the controversial one); and I have an editorial in the same issue of Movement Disorders trying to interpret the findings. Is my bias reduced by my inability to figure out in which direction it points?
Anyhow, I’ve heard various reasons why this couldn’t possibly be a real neuroprotective effect, and none of them are all that convincing. But I don’t want to bias you. Discuss.