Scientists identify small-molecule cocktail to improve stem cell use in research and disease treatments

Monday, May 3, 2021

Researchers at the National Institutes of Health have devised a four-part small-molecule cocktail that can protect stem cells called induced pluripotent stem cells (iPSCs) from stress and maintain normal stem cell structure and function. The researchers suggest that the cocktail could enhance the potential therapeutic uses of stem cells, ranging from treating diseases and conditions — such as diabetes, Parkinson’s disease and spinal cord injury — to genome editing.

Human pluripotent stem cells are cells that, in theory, can grow forever and serve as an inexhaustible source for specialized cells, such as brain, kidney and heart cells. But stem cells are sensitive, and their potential uses in medicine are hampered by the stress of growing in a cell culture dish, which can damage their DNA and lead to cell death.

In a series of experiments, scientists led by Ilyas Singeç, M.D., Ph.D., director of the Stem Cell Translation Laboratory at NIH’s National Center for Advancing Translational Sciences (NCATS), used high-throughput screening to systematically test thousands of compounds and drugs to identify a unique combination that greatly improved stem cell survival and reduced cell culture stress. Singeç and his co-investigators described how they developed the cocktail, called CEPT, and its potential applications May 3 in Nature Methods.

“The small-molecule cocktail is safeguarding cells and making stem cell use more predictable and efficient. In preventing cellular stress and DNA damage that typically occur, we’re avoiding cell death and improving the quality of surviving cells,” said Singeç. “The cocktail will become a broadly used staple of the stem cell field and boost stem cell applications in both research and the clinic.”