UM171 is a synthetic molecule developed by researchers at Université de Montréal. In stem cell research, it is used to increase the number of blood stem cells outside the body. The molecule helps cells retain their stem cell properties rather than mature into blood cells and stimulates their multiplication. The aim is to produce enough stem cells for transplantation in patients with serious blood diseases. In recent years, researchers, including those at Lund University, have shown that the molecule works by exploiting a protein called KBTBD4. This causes an important protein structure in the cell, called CoREST, to break down.

“The same structure in the cell is also affected by mutations in the KBTBD4 gene, genetic changes that occur in a subgroup of aggressive brain tumors in children. Although the causes differ, the result is the same—the structure breaks down,” says Agatheeswaran Subramaniam, a researcher at Lund University.