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Friday, Feb 21, 2025

Breakthrough in Chordoma Research by British Scientist Offers Hope for Treatment

Professor Paul Workman and his team identify key protein linked to a rare form of bone cancer, paving the way for potential therapies.
In a significant advancement for cancer research, Professor Paul Workman, head of the Centre for Cancer Drug Discovery and CEO of the Institute of Cancer Research in London, has led an international team in identifying a crucial protein associated with chordoma, a rare type of bone cancer.

Chordoma affects about one in a million people and is often referred to as untreatable, leaving many patients with very few options.

Workman's mother, Ena, succumbed to this condition, which motivated his research into effective treatments.

The key to this breakthrough lies in the protein called brachyury, which recent studies indicate is essential for the survival of chordoma cancer cells.

Blocking this protein could potentially impair cancer cell growth.

In a paper published in _Nature Communications_, Workman and his colleagues in Oxford and North Carolina detailed their analysis that revealed several targetable sites on the surface of the brachyury protein.

Crucial to this discovery was the use of the Diamond Light Source synchrotron in Didcot, Oxfordshire, one of the world's leading facilities for generating X-rays.

This technology has allowed Workman's team to isolate promising compounds that could lead to the development of drugs specifically targeting brachyury.

Current research aims to formulate treatments that can effectively eliminate this protein, which plays a pivotal role in chordoma's resistance to existing therapies.

The implication of these findings extends beyond chordoma itself; Workman pointed out that brachyury may contribute to the metastatic spread of other tumors.

Thus, therapies developed to block brachyury could enhance treatment strategies for more prevalent cancers.

Workman's personal connection to the disease has underscored his dedication to this research.

Following the deaths of both his parents from cancer, he has made strides in the medical field, now facing his own diagnosis of prostate cancer.

Although treated successfully with radiotherapy, he remains acutely aware of the challenges many patients face.

Research methodologies employed in this study include targeted protein degradation (TPD), a cutting-edge approach that utilizes a cell’s own mechanisms to remove harmful proteins.

Workman describes this process as one where a drug binds to the target protein while also engaging with the cellular waste disposal systems, ultimately leading to protein degradation.

Despite these advancements, Workman emphasized that the journey toward an effective treatment for chordoma is ongoing.

Further research will involve trials beginning with chordoma cell lines, progressing to animal models, and potentially leading to human trials.

This process is expected to take several years before a viable drug can be introduced to the market.

In summary, this breakthrough in targeting brachyury represents a hopeful turning point for chordoma patients, offering possibilities not only for better treatment outcomes but potentially extending to broader applications across various forms of cancer.
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