Guifen Qiang, PhD | Institute of Materia Medica, Chinese Academy of Medical Sciences & Peking Union Medical College; Tengfei Ji, PhD; Haiguang Yang; Peng Ma; Zijing Wang; Tianshu Xu; Tingting Zhu; Siyuan Zhang
Competition Sponsor: Chinese Academy of Medical Sciences
Awardee Year: 2023
The increasing prevalence of obesity in children is a growing concern due to the potential health problems they may face in middle age and beyond. Obesity is a significant risk factor for metabolic diseases, including diabetes, cardiovascular and cerebrovascular diseases, and tumors. The development of safe and effective weight-loss drugs has been a challenge, with many drugs being subject to FDA black box warnings or withdrawal. This project aims to explore a new therapy by targeting the thermogenic gene uncoupling protein 1 (UCP1) in adipose tissue to reduce adipose and combat obesity. The researchers used CRISPR/Cas9 gene editing technology to establish a screening model for UCP1 inducers and identified a lead compound called MD02. Preclinical evaluations in obese mice and rats demonstrated that MD02 had significant anti-obesity activity, reducing adiposity, improving glucose metabolism, through promoting white adipose tissue browning and brown adipose tissue thermogenesis. The next steps involve investigating the mechanism of action of MD02 and conducting comprehensive pharmaceutical studies, including formulation, stability, and safety evaluations. The researchers have completed preliminary evaluations and applied for patents. They aim to submit an application for clinical trials and publish scientific articles to support the development of MD02 as a novel anti-obesity drug. The goal is to provide an innovative drug that can improve the health of obese patients, reduce the incidence and death of metabolic diseases.