Neuroscientists at the Georgetown University Medical Center discovered that two deadly neurodegenerative diseases: amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD), can be controlled by removing protein garbage in nerve cells, reports Medical Xpress.
These two diseases are caused due to the cells' inability to remove or destroy TDP-43, a mutated gene that produces excess amount of proteins in the nucleus of the cell, according to Medical Xpress.
"This finding suggests that if we're able to 'rev up' that clearance machinery and help the cell get rid of the bad actors, it could possibly reduce or slow the development of ALS and FTD," said the study's lead investigator, neuroscientist Charbel E-H Moussa, MB, PhD, reports Medical Xpress. "The potential of such an advance is very exciting."
ALS occurs in the motor neurons, responsible in controlling muscles and located in the central nervous system. FTD occurs in frontal lobe of the brain, which contains mostly the dopamine sensitive neurons.
Moussa further alerts that taking this step further and treating humans will take years of hard work and research. Addition of parkin, a natural disposal unit of the cell, will automatically dispose the protein garbage in the nerve cells.
The study was conducted on two animals, by injecting the parkin genes and was found that it "slowed down ALS pathologies connected to TDP-43," said a report in Medical Xpress.
"In both diseases, TDP-43 is over-expressed or mutated, and the scientific debate has been whether loss of TDP-43 in the nucleus or gain of TDP-43 in the cytoplasm is the problem," Moussa said, according to Medical Xpress.
"Our study suggests TDP-43 in the cell cytoplasm is deposited there in order to eventually be destroyed-without contributing to disease-and that TDP-43 in the nucleus is causing the damage," he said. "Because so much protein is being produced, the cell can't keep up with removing these toxic particles in the nucleus and the dumping of them in the cytoplasm. There may be a way to fix this problem."
After detailed research and study on parkin, molecule structure and functions in different states and also studied the Alzheimer's disease and also the other forms of Dementia, Moussa concluded that parkin could remove the toxic fragments in the brain found in Alzhermer's patients.
"This discovery challenges the dogma that accumulation of TDP-43 in the cytoplasm is," he said. "We think parkin is tagging proteins in the nucleus for destruction, but there just isn't enough parkin around-compared with over-production of TDP-43-to do the job."
The study is published online in the journal of Biological Chemistry.
