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Apabetalone Shows Promise as Epigenetic Therapy for Chronic Diseases

• Qatar University researchers highlight Apabetalone's potential as an epigenetic therapy for treating various chronic diseases by targeting gene expression. • Apabetalone, a unique inhibitor with FDA breakthrough therapy designation (excluding oncology), impacts multiple biological functions involved in disease development. • Preclinical studies suggest Apabetalone's efficacy in cardiovascular, renal, neurological, viral, and cancer disorders, warranting further investigation in clinical trials. • The research underscores the importance of epigenetic treatments in regulating gene transcription and improving cell communication for enhanced health outcomes.

Qatar University (QU) scientists have made a significant advancement in precision medicine, revealing the potential of Apabetalone (RVX-208) as a novel epigenetic therapy for chronic diseases. The study, published in Pharmacology and Translational Science by the American Chemical Society, suggests that Apabetalone could revolutionize the treatment of cardiovascular, renal, neurological, viral, and cancer disorders by addressing their underlying causes through gene expression modulation.

Epigenetic Mechanism of Action

Apabetalone functions by blocking specific harmful proteins, thereby reducing their production. This epigenetic approach regulates gene transcription without altering the DNA sequence itself. Notably, Apabetalone is the only inhibitor of its kind to receive breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) outside of oncology, highlighting its unique therapeutic potential.

A New Era in Medicine

Dr. Zaid al-Ma'ayah, assistant professor of Pharmacology and Toxicology at QU and lead researcher, emphasized the significance of this work, stating, "This work represents a new era in medicine, focusing on using epigenetics to regulate the transcription of genes involved in cardiovascular, infectious, and cancer disorders." He also mentioned ongoing experimental discoveries regarding new applications of this epigenetic therapy, which will be shared with the scientific community soon.

Broad Impact on Biological Functions

Unlike traditional treatments that target a single aspect of a disease, Apabetalone takes a comprehensive approach by acting earlier in the disease process and influencing multiple biological functions. By blocking proteins involved in disease development, Apabetalone offers a novel way to treat chronic illnesses, improving both local and systemic cell communication and promoting overall health.

Preclinical and Clinical Prospects

Early animal studies have demonstrated the impact of Apabetalone on gene function, particularly concerning protein modifications. Researchers plan to explore the combined use of Apabetalone with other treatments to enhance its protective effects. As Apabetalone advances through clinical trials, continued preclinical research is crucial to fully understand its potential across various diseases.

QU's Commitment to Research

The study, supported by an internal grant from QU, highlights the university's dedication to advancing scientific research. The state-of-the-art research facilities and supportive environment at QU have been instrumental in exploring new frontiers in epigenetic therapies, offering hope for the future of precision medicine.
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Reference News

[1]
QU scientists in precision medicine breakthrough - Gulf Times
gulf-times.com · Sep 16, 2024

Qatar University scientists developed Apabetalone, an epigenetic therapy targeting gene regulation for treating chronic ...

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