DRUGS that could one day regenerate tissue in the liver and other organs may be possible, researchers in China revealed in a study published in Science Translational Medicine.
The research is significant because, while tissue regeneration is possible, it usually relies on complex therapies involving targeted delivery of regenerative cells (such as stem cells) and other biomaterials to damaged areas.
“Most regenerative medicine strategies have focused on delivering biomaterials and cells, yet there is the untapped potential for drug-induced regeneration with good specificity and safety profiles,” the researchers led by Dr. Fan Fuqin of Xiamen University said in their paper.
The research team explained that their work focused on an important molecular signaling pathway called the Hippo pathway, which regulates organ size and growth. The researchers looked for a small molecule that could block the action of two enzymes in this pathway, MST1 and MST2, that normally prevent cells from proliferating. In the process, they discovered the small molecular inhibitor XMU-MP-1, which blocked MST1/2 activity and allowed cells to proliferate and avoid programmed cell death.
The molecular inhibitor helped to regenerate liver tissue in several mouse models of liver damage, including damage by acetaminophen overdose, the leading cause of acute liver failure worldwide.
The researchers also reported that, unexpectedly, their tests found that the drug protected mice against a model of ulcerative colitis as well.
While the research is very preliminary, the researchers expressed confidence that continued work could pave the way for medicines that help to rebuild organs.