This study is companion research to CEP-1347 restores BDNF expression in the R6/2 mice . This second study suggests that levels of BDNF's messenger RNA in the blood might serve as biomarkers. While levels of the BDNF protein itself were not detectable in the blood, levels of mRNA were detectable in rodent models. These levels declined with age but were restored to normal levels by CEP-1347 treatment.
If BDNF mRNA is also detectable in the blood of those who are gene positive, then it would be a good biomarker to determine when treatment is necessary and whether it has been effective in restoring BDNF.