A new kind of drug points to where medicine is headed (Priscilla Duggan, July 30, 2026, Larchmont Chronicle)
Just this past May, the Food and Drug Administration approved a new drug named vepdegestrant (Veppanu) for a subset of patients suffering from metastatic breast cancer. First and foremost, this is a promising development for individuals with oestrogen receptor-positive, HER2-negative breast cancer. However, there is an extra layer of excitement to this news: Veppanu is the first drug of its kind (known as a PROTAC) to ever be approved by the FDA. For the remainder of the article, I will go over exactly what I mean by this and unpack all these science terms.Let’s break it down into simple concepts: proteins are everywhere in the body. Receptors, enzymes, structural elements—they’re all made up of proteins. Many diseases stem from proteins that aren’t behaving as expected. For example, an overactive protein could lead to cancer, or a misfolded protein could lead to a neurodegenerative disease. Therefore, many existing drugs work by blocking a protein’s function.
PROTACs (which is short for proteolysis-targeting chimera) work in a completely different way. In simple terms, they stick onto a specific protein and bring it to our cell’s natural garbage-disposal system.
What is this “garbage disposal system”? In biology, when a protein needs to be destroyed by the body, this is referred to as “ubiquitination.” A faulty or misfolded protein that needs to be destroyed is first “tagged” with a little flag called an E3 ligase. This flag calls over another protein called ubiquitin, the cell’s garbage man. Basically, ubiquitin’s job is to destroy whatever the E3 is connected to. […]
Medicine is learning not only how to inhibit problematic proteins, but how to remove them. Consider the plethora of diseases caused by protein malfunction: Alzheimer’s, Parkinson’s, Huntington’s, cystic fibrosis—the list goes on. With this in mind, this step forward has promising implications for many diseases that have long been considered non-treatable.