The Magic, Power, and Impact of iPSCs

Written by Jürgen Eckhardt, Head of Leaps by Bayer

Rarely have I been so impressed with a foundational technology’s ability to sound like magic as I am with iPSCs, or induced pluripotent stem cells. Before talking about what it means to ‘induce’ them, consider that stem cells are undifferentiated cell types that have not yet been told exactly what distinct types of cells in the body they will turn into, like skin cells, or those of an eye, or a liver. Their functions in life lie ahead of them still, which is why they’re plentiful in embryos that haven’t yet taken on much form. But thanks to technology pioneered by Shinya Yamanaka’s lab in the mid 2000s, researchers can induce this pluripotent state from any adult cell that has already become a specific cell type.

By forcing adult cells to express genes and other factors that define the properties of embryonic stem cells, scientists are able to bring them back to a moment when more was possible for their fate. Then once they’re pluripotent again, or in other words — “rewound” — scientists can decide what type of tissue they want them to become, and coax them to grow in that direction. So in other words, this astounding technology enables any adult cell to be deprogrammed and then reprogrammed into any other cell type in the body. iPSCs combine time travel and “choose your own adventure” mechanisms of biology, bringing whimsy, wonder, and life-saving potential to the world.

When Yamanaka discovered iPSCs in 2006, it caused a lot of excitement. iPSCs seemed promising not only for their therapeutic potential, but also because they allow researchers to sidestep some of the ethical challenges associated with stem cell research. But it was never guaranteed that the technology would take off. If you speak candidly with biotech industry leaders, many will say they’re hesitant to call any technological advancement, even one as exciting as iPSCs, a “true breakthrough” until it has had 10 to 15 years to prove its widespread application. Fortunately, we’ve seen that time pass with iPSCs and can now see their growing impact on biotech as a whole. Over the last decade and a half, iPSCs have changed what’s possible in drug screening, fertility, longevity research, transplants, regenerative medicine, and become integral to several quizzical missions, like the “resurrection” of extinct species in a field known as de-extinction. In short, they’re incredibly flexible and can be found making their way in most of biotech’s nooks and crannies.

Now with the rise of CRISPR gene-editing technology and the ability to manufacture iPSCs at scale, Yamanaka’s discovery is advancing into even more sophisticated applications. It’s no surprise then that several iPSC companies, including Century Therapeutics and BlueRock, have garnered our attention at Leaps by Bayer. We invested in Century Therapeutics, who are leveraging iPSCs and genetic engineering to make cancer-fighting therapies that are designed to target a variety of blood and solid cancers that affect millions every year. By aiming to improve so many lives, they have the potential to provide a huge return on humanity. We also invested in BlueRock Therapeutics, which combines genetic engineering and iPSCs to target neurological, cardiological and immunological diseases by growing new cells for tissues that can’t replace themselves. This can then be used to treat diseases like Parkinson’s, which causes the progressive loss of brain cells that can’t be naturally regrown, or a heart attack, after which some heart muscle cells completely lose their function.

For someone who has never heard of iPSCs before, it can be hard to believe that adult cells can be reprogrammed to do all of that. What a privilege it is then to be almost 15 years on from the technology’s discovery and be able to say, resoundingly, yes — they can do all of that. I believe that in these rare cases, when breakthrough science sounds like magic for human health, we have a duty to enchant the world with it.