Chordoma is a rare cancer of the spine and skull base, diagnosed roughly one person per million each year. There is no approved drug therapy for it anywhere in the world. The standard treatments are surgery and radiation, but more than half of patients eventually experience a recurrence, and many reach a point where surgery and radiation are no longer safe, effective, or possible. For those patients, drug therapy is the only remaining option, yet today only three drugs have been shown to provide meaningful benefit, and each benefit only some patients. The toolbox is far too small.
The reason it remains small is structural. Because chordoma is rare, the commercial market is too limited to attract the sustained investment that ordinarily drives drug development, and no single hospital sees enough patients to study new therapies efficiently. As a result, promising treatment ideas have historically gone untested.
This project seeks to develop better drug therapies for chordoma by (1) testing promising therapeutic concepts to determine which are most likely to benefit chordoma patients; (2) generating the preclinical data needed to advance the strongest candidates into clinical evaluation; and (3) building and applying the specialized laboratory capabilities required to drug brachyury, chordoma’s defining molecular target.
Activities of this project may include:
Myriad Canada is working with the Chordoma Foundation on this project, a nonprofit dedicated to improving the lives of those affected by Chordoma and advancing the search for a cure. Through its 2026–2030 Strategic Plan, the Foundation aims to double the number of effective drug therapies available to patients.
To achieve this goal, the Foundation established Chordoma Foundation Labs (CF Labs) in Durham, North Carolina. The laboratory enables the Foundation to evaluate new treatment ideas, generate the preclinical evidence needed to justify clinical testing, and move the most promising drugs toward patients far faster and at lower cost than would otherwise be possible. It is the first and only laboratory in the world dedicated entirely to chordoma therapy development, and one of the first of its kind for any disease.
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