Can Targeting Brachyury Unlock New Treatments for Rare and Metastatic Cancers?

A recent breakthrough from the Institute of Cancer Research in London could mark a turning point in how scientists approach some of the most difficult cancers. Led by professor Paul Workman, researchers have discovered a way to potentially drug a protein called brachyury, a target long considered out of reach.

Brachyury plays a critical role in a rare type of cancer known as chordoma, which affects the spine and base of the skull. These tumors are notoriously hard to treat, often recurring after surgery and showing poor response to traditional therapies. For years, researchers believed that brachyury could not be targeted by drugs because of its structure and location inside the cell.

That view is now changing. Using the UK’s Diamond Light Source synchrotron, Workman’s team was able to map precise pockets on the surface of brachyury. These pockets can potentially bind with small molecules, opening the possibility of developing drugs that interfere with the protein’s activity and disrupt the survival of cancer cells.

This is more than just a promising lead for chordoma. Brachyury is also active in several types of metastatic cancer, where it is linked to invasion, resistance, and poor outcomes. If the new findings lead to a working drug, the impact could extend far beyond one disease.

You can read the full article here:

https://www.theguardian.com/science/2025/feb/15/british-professor-paul-workman-cancer-breakthrough-chordoma

The work is still at an early stage but it reflects a renewed focus in biotech on tackling tough, overlooked targets with the right tools and deep structural insights. This is where real progress happens — slowly, precisely, and with lasting consequences.