New AI-Driven Discovery: Malaria Drug Reverses Bone Loss in Osteoporosis

Using artificial intelligence, researchers from Peking University have identified an antimalarial drug, dihydroartemisinin, as a potential osteoporosis treatment. The study, published in ACS Central Science, revealed that dihydroartemisinin reversed bone loss in osteoporosis-afflicted mice. The integration of AI in drug discovery offers a beacon of hope for innovative therapeutic solutions.

In a groundbreaking study facilitated by artificial intelligence (AI), scientists from China's Peking University International Cancer Institute have identified dihydroartemisinin (DHA), an antimalarial drug derived from the sweet wormwood plant, as a potential treatment for osteoporosis. This innovative approach focuses on promoting bone-forming cells, osteoblasts, rather than inhibiting bone-breaking osteoclasts, the traditional treatment method.

Osteoporosis, a disease predominantly affecting older adults, occurs when bone-breaking osteoclast cells become overactive, leading to bone loss. Traditionally, treatments have centred on inhibiting these osteoclasts. However, this new study shifts the focus to osteoblasts, the cells responsible for bone formation. 

Specifically, the researchers examined bone marrow mesenchymal stem cells (BMMSCs), which, during osteoporosis, are inclined to transform into fat-creating cells. The idea is to redirect these cells to combat the disease.

By employing a deep learning algorithm previously developed by the team, the researchers analyzed the influence of various small-molecule drugs on gene expression associated with osteoporosis. DHA emerged as a top contender. When administered to mice with osteoporosis for six weeks, DHA not only reduced bone loss in the femur but also preserved their bone structure, showing promising results.

To further optimize the drug's delivery, DHA was encapsulated in specialized nanoparticles called MSN-ALN, tailored specifically for bone targeting. This method ensured that the drug effectively reached the BMMSCs. Mice receiving this treatment displayed bone quality nearly equivalent to that of healthy controls, without exhibiting any signs of toxicity.

Delving into the mechanics, the researchers established that DHA preserves the stemness of BMMSCs by promoting histone 3 lysine 9 acetylation through GCN5 activation. This finding echoes the central theme of the original research abstract, which emphasizes the significance of maintaining BMMSC stemness for bone health and restoration.

DHA's roots trace back to traditional Chinese medicine. The antimalarial properties of DHA and its parent compound, artemisinin, were first identified in the 1970s by Tu Youyou, who later received a Nobel Prize in medicine in 2015 for this discovery. This breakthrough provides a promising new direction in osteoporosis treatment.

Supported by numerous funding agencies, including the National Natural Science Foundations of China and the Beijing International Science and Technology Cooperation, this research points to a potential paradigm shift in osteoporosis treatment.

Abstract of the research