Translation of regenerative neurological therapies

Common themes of inflammation, oxidative stress, mitochondrial dysfunction and protein mishandling recur across multiple neurological diseases. To date, therapeutic strategies targeting unique features of individual diseases studied in animal models have proved unsuccessful in clinical trials.

To identify recurrent themes across human neurological diseases, Dr. Burns and colleagues took an alternate approach, performing a rigorous transcriptional meta-analysis of available human data for Alzheimer's disease, Huntington's disease, Parkinson's disease and amyotrophic lateral sclerosis, thereby defining the conserved transcriptional signature of human neurodegeneration.

Comparison of this human signature to that of neurodegenerative animal models revealed striking differences. These data help explain why therapies that appear promising in animal models of neurodegeneration have rarely succeeded in human clinical trials. This work helps provide a path forward in developing innovative translational pipelines that yield therapies that work in humans rather than mice.

Related publications: