A pioneering collaboration is unlocking the secrets of disease by zooming in on individual cells. Using single-cell and spatial genomics, scientists are discovering new cell types, revealing new drug targets, and harnessing artificial intelligence to transform how we understand and treat complex conditions – from COPD to liver and kidney disease.
In the quest to solve the mysteries of human disease, a new technology is handing researchers unprecedented detail.
While decoding the human genome has advanced our understanding, DNA only tells part of the story. Researchers may know that a gene variant is linked to an illness but often do not know how and when it affects the disease process.
Now, new cutting-edge techniques are enabling scientists to zoom in on single cells – the fundamental units of biology – and gain a far clearer picture of the molecular intricacies of disease.
That promise – and its power for drug discovery and development – is at the heart of a new collaboration between GSK and the Teichmann Laboratory at the Cambridge Stem Cell Institute, a world-leading centre for these new techniques.
“We have been able to isolate the cell type that’s responsible for the organ dysfunction and the timing of its involvement. That’s incredibly powerful for drug discovery and what we need to know to prevent and change the course of disease,” says Kaivan Khavandi, SVP & Global Head, Respiratory, Immunology & Inflammation R&D at GSK.
Advances in single-cell and spatial genomics, along with computational power, are allowing researchers to untangle the differences between individual cells, making it possible to selectively sequence the genes that are “switched on” in a given cell and build a detailed picture of the cell from that data.
“In the past, metaphorically speaking, we’ve basically been trying to work with a smoothie and then trying to work out what fruit went into the smoothie,” says David Michalovich, VP of Translational Sciences at GSK’s Respiratory, Immunology and Inflammation Research Unit. “But now we’re able to understand precisely what fruit is in the smoothie, and can work out how many bananas, apples, blueberries, blackberries, and so on, are in there. Translating that back to our work at GSK, by using these cutting-edge scientific approaches, we’re now able to get to that very fine review of what cells are in a tissue.”
Studying tissues at different stages of disease, cell by cell, makes it possible to identify the processes involved in unprecedented detail. In turn, that work generates new ideas and stronger evidence for drug-mediated mechanisms that might work to stop or delay disease processes at the molecular level. Confirming a drug’s mechanism using single-cell and spatial genomics could double the chances of its eventual success in clinical trials on top of the already-significant confidence boost from establishing a genetic link.