KAIST develops GFRAL ASO therapy for cancer cachexia

Researchers at KAIST, led by Professors Minho Shong and Jinkuk Kim, in collaboration with THOR Therapeutics, developed an RNA-based antisense oligonucleotide (ASO) therapy targeting the GFRAL receptor in the brainstem to treat cancer-associated cachexia. The therapy blocks the GDF15-GFRAL signaling pathway, which drives muscle and fat wasting in cancer patients. In tumor-bearing mice, the treatment preserved body weight, muscle mass, and metabolic function, and improved survival to 90% at day 50 compared to 20% in untreated controls. The findings were published in Cell Reports on July 27, 2026. The team plans further preclinical studies and aims to begin clinical development by 2030.