According to a story from Science & Technology Research News, at team of researchers led by Kenneth S. Kosik, who is affiliated with UC Santa Barbara, have made a discovery that could allow for the development of new treatments for neurodegenerative diseases associated with the build up of misfolded proteins in the brain tissue called tau. Such diseases include Alzheimer’s disease, progressive supranuclear palsy, and many others. Kosik says that he and the team are “super excited” about the findings.
About Tau Protein and The Study
Tau protein normally plays a role in stabilizing microtubules, a portion of the nerve cell that is critical to transmitting signals to other nerve cells. In tauopathy diseases, tau becomes misfolded and aggregates into tangles that prevent normal neuron function and ultimately are the driving force behind neurogeneration and dementia. The team identified mutations of the RASD2 gene as their beginning point. They began to investigation a protein called Rhes which works on the cell surface. The process in which this protein becomes attaches to the cell surface is called farnesylation. It became the focus of a potential cancer drug that ultimately failed during trials.
However, the use of one of these drugs, called lonafarnib, was able to reduce symptoms in a mouse model of frontotemporal dementia, a disease called by tau build up. Further analysis allowed the scientists to confirm that the drug was functioning by inhibiting the farnesylation process of the Rhes protein. Another promising aspect of these findings was that the drug didn’t appear to have any harmful or toxic effect on the mice, which is a requirement for any therapy to treat neurodegenerative disease that is happening in the brain.
A New Hope
The field of treatment for tauopathies has been pretty dry for a while so this study heralds a potentially significant step forwards. While the stage is set for early tests with human subjects, it may very well be some time before they get off the ground. The makers of lonafarnib are currently testing the drug as a treatment for progeria, another rare disorder characterized by early aging. It is doubtful that they will want to involve the drug in another test before the earn an approval.
Check out the original study here.