Erectile Dysfunction Drugs can Help Fight Rare Diseases, says study


According to medical knowledge, the abnormal buildup of misfolded proteins is a sign of host of diseases, including neurodegenerative diseases such as Parkinson’s and Alzheimer’s. If such protein aggregate they create havoc in the function of tissues, cells, and organs. Despite several years of intensive research, there are still no effective means to prevent or remove their accumulation in humans.

The remedy may someday come from an unexpected source – a common class of drugs to treat erectile dysfunction.

To address this, scientists at Harvard Medical School have identified a new mechanism to activate the protein quality-control system of cells. The mechanism is to improve the ability to dispose misfolded proteins, including the ones that cause neurodegenerative diseases.

As reported in the Proceedings of the National Academy of Sciences, the researchers describe the function of erectile dysfunction drugs, including tadalafil and sildenafil. The report describes how these drugs lower the accumulation of mutant proteins, reduce cell death, and anatomical abnormalities in zebrafish models of neurodegeneration.

Expansion of misfolded Protein related to Rare Diseases

“Meanwhile, the study indicates a new approach to fight the basic root of many neurodegenerative diseases, as well as rare muscle and cardiac diseases. These diseases are related to the buildup of misfolded intracellular proteins,” said the senior author of the study

The new approach is attractive as it utilizes a previously unknown mechanism. The mechanism leverages that cells have to remodel their protein composition naturally through increased degradation. These findings, hopefully, will lead to newer therapies in the coming years, added the lead author of the study.

The results of the mechanism are an early step towards potentially addressing the reasons of failure of protein quality control, and build-up of toxic proteins that underlie many neurodegenerative disorders.

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