According to a public release from University of Texas Health Center at San Antonio on eurekalert.org, research using fruit fly tauopathy disease models has revealed a genetic “copy-paste” activity that may play a major role in tauopathies, which are neurodegenerative illnesses that are linked to the build up of tau protein in the brain. The scientists also discovered a potential treatment for these illnesses during the study as well.
Tauopathies comprise a variety of both common and rare neurodegenerative diseases. Tau most famously plays a role in Alzheimer’s disease, one of the leading causes of dementia that affects millions of people around the world. This is also the disease in which the role of tau in disease was first identified. Several rare diseases, such as progressive supranuclear palsy, panthotenate kinase-associated neurodegeneration, and tuberous sclerosis are all affected by changes to the activity of the tau protein. The protein builds up into harmful deposits that are called neurofibrillary tangles.
Portions of genetic material essentially create clones of themselves and then insert these cloned sections into new areas on the DNA strand. This activity is thought to be the result of tau protein buildup over time, and ultimately causes neurons to die.
The team also found that a certain medication called Iamivudine could substantially reduce the cloning and insertion activity by limiting the expression of the genes that play a role. Iamivudine is normally used to treat hepatitis B and HIV, but it be a potential treatment option for this class of devastating and debilitating diseases. Many of them have no effective treatments that can slow down or halt disease progression.
New Hope for Treatment
The treatment was successfully able decrease the death of neurons in the fruit fly model. The team was also able to assess other data that suggested that the drug would also be effective in treating humans. The next step will be the design and implementation of clinical trials testing the capability of this drug in humans who have tauopathies. While it may be some time before this process begins, it could be a potential breakthrough for patients suffering from progressive supranuclear palsy and diseases linked to tau activity.
Check out the original study here.