Cell Reprogramming Could Treat Parkinson’s Disease

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Converting astrocytes to dopamine neurons shows promise in a mouse model of Parkinson’s

Parkinson’s is a complex disease, but one of the major aspects is a loss of dopamine neurons in the substantia nigra region of the brain. There are several ongoing studies attempting to treat the disease through gene therapy, primarily by injecting the genes for dopamine production into surrounding neurons, or by replacing lost cells with new ones. Of this latter group a team has published a new paper in which they reprogrammed other brain cell types into dopamine neurons – suggesting that a reprogramming technique may be able to create more dopamine neurons to replace those that have been lost.  What did it involve? In the new study researchers used 3 transcription factors NEUROD1, ASCL1 and LMX1A, and the microRNA miR218 to coax astrocyte cells to become dopamine ones. This process was tested both in vivo (in the mice) and in vitro (on cells in a laboratory). They were successfully able to transform these cells at an efficiency rate of 16%; an encouraging rate compared to many other studies. These induced dopamine neurons (iDANs) were even able to correct some movement in the treated mice, suggesting the therapy could be a viable new option for Parkinson’s sufferers. 

“Moving from this study to doing the same in humans will be a huge challenge. Further development of this technique is now needed”

While this study is certainly interesting, it will take a great deal more finessing and work to develop an approach that is scalable and applicable to human patients. It should also be noted that we still don’t know the real cause of Parkinson’s disease, so it could be that any newly reprogrammed cells in the brain would eventually be lost as the disease progressed. This approach is definitely exciting, but it would have to be combined with preventative drugs to delay the disease onset until we have a curative treatment for the disease.  Read more at BBC News and Nature Biotechnology