A three-year collaborative project entitled “Research and development of aptamer-triptolide conjugate for treatment of rheumatoid arthritis” conducted by the Institute for Advancing Translational Medicine in Bone and Joint Diseases of HKBU and the Institute of Basic Research in Clinical Medicine of the China Academy of Chinese Medical Sciences was recently selected as the Major New Drugs Innovation and Development Scheme in 2013 by the National Science and Technology Major Projects and awarded a start-up fund of 1.5 million renminbi.
“Tripterygium wilfordii Hook F” is a Chinese medicinal herb used in the treatment of arthritis despite its toxicity. One of its major medicinal compounds, triptolide, is effective in treating rheumatoid arthritis but its toxicity could cause serious damage to the liver, which makes it challenging to translate the herb into a medicine for the treatment of rheumatoid arthritis.
The principal investigator of the research project, Professor Lu Aiping, Dean of the School of Chinese Medicine, likened this innovative research to the design of a “self-targeting missile”. The research team will conjugate triptolide with nucleic acid aptamer through a pH-sensitive linker – the specific aptamer targeting inflammatory cells serves as a missile warhead with the triptolide inside serving as ammunition. The “missile”, which is capable of automatically “targeting” and “attacking”, will enable triptolide to avoid liver cells and therefore reduce damage to the liver. The nucleic acid aptamer will guide thetriptolide to the inflammatory cells affected by rheumatoid arthritis before automatically rupturing in the acidic environment of the rheumatoid arthritis cells, leaving the “warhead” to play its therapeutic role.
Professor Lu said that the study paves way for the development of a new drug which is effective and safe for treating rheumatoid arthritis and serves as a reference for future related research, thereby contributing to the development of new drugs derived from Chinese medicines and other natural products.
Professor Lu Aiping is granted funding from the National Science and Technology Major Projects to conduct his collaborative research project
Tripterygium wilfordii Hook F
The process of how the aptamer and triptolide work together in the treatment of rheumatoid arthritis: 1. The aptamer guides triptolide to the inflammatory cells affected by rheumatoid arthritis. 2. The linker ruptures in the acidic environment of the rheumatoid arthritis cells. 3. Triptolide is released to play its therapeutic role.