Professor Paul Workman, a renowned medical researcher, knows the heartbreak of losing a loved one to cancer all too well. At 37, after already establishing himself as a prominent researcher, he lost his mother, Ena, to a rare and aggressive form of bone cancer known as chordoma. Affecting just one in a million people, chordoma is notoriously difficult to treat, leaving Workman and many others frustrated by the lack of available treatment options. However, a groundbreaking discovery by Workman and his team may soon offer new hope to those affected by this devastating disease.

A New Target for Treatment: Brachyury Protein

After years of research, Workman and his colleagues have pinpointed a key protein, known as brachyury, that plays a crucial role in the survival of chordoma tumor cells. This discovery has sparked excitement within the scientific community, as it presents an opportunity to develop targeted treatments that could block the activity of this protein and prevent the growth of cancerous cells.

Brachyury, which is essential for the survival and proliferation of chordoma cells, has long been considered drug-resistant due to its complex structure. However, Workman’s team, in collaboration with researchers from Oxford and North Carolina, used cutting-edge technology, including the Diamond Light Source synchrotron in Oxfordshire, to study brachyury in unprecedented detail. This allowed them to identify several sites on the protein’s surface that could serve as potential targets for specially designed drugs.

Targeting Brachyury: A Game-Changer for Cancer Treatment

The team’s findings, published in Nature Communications, are a major step forward in the fight against chordoma. By isolating several promising compounds, Workman’s team is now working to create drugs capable of attacking and degrading the brachyury protein. This could eventually lead to a treatment for chordoma, a condition that has so far resisted conventional cancer therapies.

Credits: Pancreatic cancer UK

For Workman, the discovery is especially personal. “It is thrilling to realize that I am now helping to do something about a disease that killed my mother,” he shared. “It has taken considerable effort by a lot of scientists from centers on both sides of the Atlantic, but it has been worth it.”

Wider Implications for Cancer Research

What makes this breakthrough even more exciting is its broader potential to treat other cancers. Brachyury plays a role in the metastatic spread of several types of cancer, meaning that drugs targeting this protein could also help prevent the spread of more common cancers, such as breast, lung, and colon cancer. The ability to block brachyury’s activity could, therefore, pave the way for more effective treatments for a range of malignancies.

Credits: ISSUU

Workman’s passion for cancer research stems not only from his professional expertise but from his own personal experiences. After losing both parents to cancer—his father to bowel cancer and his mother to chordoma—Workman is determined to make a difference in the lives of others. Despite being diagnosed with prostate cancer himself in 2022, he remains optimistic about the future of cancer research.

Targeted Protein Degradation: A Revolutionary Approach

One of the promising approaches in developing drugs to target brachyury involves a technique called targeted protein degradation (TPD). This method takes advantage of the body’s natural waste disposal system, coaxing cells to break down and eliminate harmful proteins. By binding to the brachyury protein, TPD drugs could facilitate its removal from cancer cells, offering a novel and potentially effective way to treat chordoma.

Workman remains cautious, acknowledging that more research is needed before these new treatments can reach patients. “We need to begin trials in chordoma cell lines first and then in animal models before we can proceed with human trials,” he explained. This process could take several years, but Workman is optimistic that the day will come when chordoma can finally be conquered.

The Long Road Ahead

While this research is a monumental step forward in the fight against chordoma, Workman emphasizes that there is still much work to be done. With continued collaboration, advanced technology, and relentless determination, Workman and his team are hopeful that their efforts will lead to new treatments that could save countless lives.

As research progresses and trials begin, the dream of developing an effective treatment for chordoma moves closer to reality. Workman’s personal journey, from losing his mother to advancing cancer research, is a testament to the power of perseverance and the potential for scientific discovery to change lives.