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By Get Science Staff
Proteins are the stuff of life. They comprise the hormones, enzymes and many other molecules that are essential to our body’s functioning. But sometimes these loyal workhorses can go rogue, leading to cancer, autoimmune diseases and a variety of neurological conditions.
Scientists who develop drugs are always looking for new ways to target these disease-related proteins — either to shut down their activities or to turn them up — depending on the circumstances. But a novel new approach to drug discovery called targeted protein degradation seeks to outright destroy these troublesome proteins. By co-opting the body's own natural "garbage disposal system," called the proteasome, this class of medicines marks these rogue players for destruction and sends them off for recycling within the cell.
This approach shows great promise for a class of proteins related to cancer, known as transcription factors, which have historically been deemed extremely difficult to target with a medicine.
“We can really explore an “undruggable” target, one that we couldn’t touch before,” says Andrea Weston, an Associate Research Fellow in the Primary Pharmacology Group at Pfizer’s Groton, Connecticut research site. “It’s a totally different drug paradigm because it opens up so many possibilities.”