I don't understand why they need to run another trial with a comparison group. Isn't median survival well established to compare it to?
http://www.dukechronicle.com/article/2017/05/duke-researchers-develop-treatment-that-triples-survival-time-of-brain-cancer-patients
Small phase 1 trials like this often show much better results than larger trials with a larger, broader patient population. They (Duke University) are currently running a larger trial (45 patient estimated enrollment) for the CMV-targeted dendritic cell vaccine + tetanus shot + TMZ, with patients randomized to receive either 5 day or 21 day schedule of TMZ.
ReplyDeletehttps://clinicaltrials.gov/ct2/show/NCT02864368
Unfortunately even if this larger trial shows positive results it still won't likely be enough to get the vaccine FDA approved, they'll likely have to run a large multi-center phase 3 trial for that. My own position is that placebo controls are unethical for a disease like GBM, and that large, multi-center trials with no placebo arm could establish whether a treatment is superior to standard treatments alone, using some form of matched, contemporary, standard-treatment cohort for comparison. However drug development companies have to work with current regulations in order to obtain FDA approval.