The Powerful Potential of Stem Cells in Pre-Clinical Research
Over 15 years ago, stem cells burst into the general news, and while over time they may have slid from the consciousness of the general public, stem cells remain vitally important in the laboratory for researchers around the world, including those working in pre-clinical drug research, like the team at LQT Therapeutics.
Strictly speaking, a stem cell is an undifferentiated cell of a multicellular organism which is capable of giving rise to indefinitely more undifferentiated cells (i.e., more stem cells), and from which other kinds of cells arise by differentiation. Speaking more simply, stem cells are a type of cell that can develop into many different types of cells, or can simply regenerate themselves by dividing.
The human body has a variety of different types of cells - skin cells, blood cells, muscle cells (including heart cells), and the like. As these cells mature, they lose the power to divide or replicate. Therefore, stem cells, which have the capability to differentiate into any cell type, have garnered a lot of interest from the global scientific community.
In some laboratories, scientists are exploring stem cells’ regenerative properties and how they replicate, with an eye toward possible treatments of conditions including diabetes and heart disease. Another way researchers use stem cells is in the development and testing of new chemical compounds and drugs to treat various health conditions. In this type of work, researchers work primarily with a type of stem cell called induced pluripotent stem cells (iPSCs), which are generated from adult cells.
With appropriate nutrients and inducers, iPSCs can be programmed to differentiate into any cell type of the body, including cardiomyocytes that make up heart muscle/cardiac muscle. These cells then serve as a great modelling platform for therapeutic drug screening or assay development. Another notable application of iPSCs in cardiac research is in optical mapping technology, where high-speed cameras and fluorescence microscopy are deployed to examine the etiology and therapy of cardiac arrhythmias in patient-like environments.
At LQT Therapeutics, our researchers use iPSC-derived cardiomyocytes from patients of Long QT Syndrome (LQTS), and these cells are then programmed to acquire properties of cardiomyocytes of LQTS patients. Using these cells, the LQT Therapeutics team tries to understand the effect of their compounds on LQTS. It also allows them to determine not only what works and what doesn’t, but also what works without any undesired toxicity to other cells.
While stem cells might be the (mostly) unsung heroes of the human body, they certainly are appreciated and valued on the bench in the lab. They are indispensable assets in the race for cures for so many problematic health challenges.
Lab work is a rigorous cycle of formulation, testing and modification of hypotheses through systemic observation, measurement, experimentation and analysis. LQT Therapeutics’ dedicated research team continues their stem cell work, relentlessly pursuing more precision therapies to treat Long QT Syndrome, allowing patients to live their best lives.