Scientists at the University of Colorado Boulder have identified a virus-like protein, PEG10, as a significant factor in the progression of amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to the loss of motor function. Currently, there are only a few drugs available that can moderately slow the progression of the disease, and there is no cure.
The researchers discovered that PEG10, which is usually associated with placental development, changes cell behavior in a harmful way when present in high amounts in nerve tissue. This finding opens up new possibilities for the diagnosis and treatment of ALS.
According to senior author Alexandra Whiteley, assistant professor in the Department of Biochemistry, “Our work suggests that when this strange protein known as PEG10 is present at high levels in nerve tissue, it changes cell behavior in ways that contribute to ALS. We may have a new target for treating ALS. For a terrible disease in which there are no effective therapeutics that lengthen lifespan more than a couple of months, that could be huge.”
The researchers are now working to understand the molecular pathways involved in PEG10’s harmful effects and to find a way to inhibit the protein. With funding from the ALS Association, the National Institutes of Health, and Venture Partners at CU Boulder, they hope to develop new therapeutics that target the root cause of the disease.
PEG10 is an ancient, virus-like protein that likely played a key role in the development of placentas in mammals. However, when it is overly abundant in the wrong places, it can fuel disease, including certain cancers and a rare neurological disorder called Angelman’s syndrome.
Whiteley’s research is the first to link PEG10 to ALS. The protein was found to be present in high levels in the spinal cord tissue of ALS patients, where it likely interferes with the communication between brain and nerve cells.
The discovery of PEG10’s role in ALS could also lead to a better understanding of other diseases that result from protein accumulation and provide insights into how ancient viruses influence health.
In conclusion, the identification of PEG10 as a significant factor in ALS progression offers new possibilities for the diagnosis and treatment of this devastating disease. The research could potentially lead to the development of new therapeutics that target the underlying cause of ALS, providing hope for patients and their families.
How does PEG10 contribute to the damage and loss of function in nerve cells in ALS patients?
The development of targeted therapies for ALS.
The study, recently published in the journal Nature Cell Biology, involved experiments using nerve cells derived from both mouse models and human patients with ALS. The researchers found that high levels of PEG10 led to significant damage to the nerve cells, causing them to lose their ability to function properly.
Furthermore, the scientists discovered that PEG10 interacts with a protein called TDP-43, which is already known to play a role in ALS. This interaction further exacerbates the negative effects of PEG10, leading to accelerated disease progression.
By identifying the role of PEG10 in ALS, the researchers have provided a new target for potential therapies. Currently, the few drugs available for ALS only provide limited relief and do not halt the progression of the disease. However, with the discovery of PEG10’s harmful effects, scientists can now focus on developing drugs that specifically target this protein to slow down disease progression.
The researchers are particularly excited about the potential of gene therapy to address the issue. By introducing a targeted gene that can inhibit the production of PEG10, it may be possible to reduce its harmful effects and provide relief for ALS patients.
This research opens up new avenues for understanding the mechanisms behind ALS and offers hope for the development of more effective treatments. With further study, scientists may be able to develop therapies that can slow down or even halt the progression of this devastating disease.
This article sheds light on the potential of PEG10 as a hopeful target for treating ALS. Exciting times in ALS research!