New Drug Hope for Patients with Rare Bone Cancer

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Patients with a rare bone cancer of the skull and spine – chordoma – could be helped by existing drugs, suggest scientists from the Wellcome Trust Sanger Institute, University College London Cancer Institute and the Royal National Orthopaedic Hospital NHS Trust. In the largest genomics study of chordoma to date, published today (12 October) in Nature Communications, scientists show that a group of chordoma patients have mutations in genes that are the target of existing drugs, known as PI3K inhibitors.

Patients with a rare bone cancer of the skull and spine – chordoma – could be helped by existing drugs, suggest scientists from the Wellcome Trust Sanger Institute, University College London Cancer Institute and the Royal National Orthopaedic Hospital NHS Trust. In the largest genomics study of chordoma to date, published today (12 October) in Nature Communications, scientists show that a group of chordoma patients have mutations in genes that are the target of existing drugs, known as PI3K inhibitors.

Researchers suggest a clinical trial of PI3K inhibitors for this particular group of chordoma patients, who could benefit from existing treatments.

Chordoma is a rare form of bone cancer that strikes individuals of all ages and affects 1 in every 800,000 people in the UK*. Chordomas are slow-growing, yet aggressive and life-threatening tumours that form in the vertebral bodies of the spine, the sacrum and base of the skull. The cancerous tumour is considered to develop from persistent embryonic tissue, known as the notochord.

As they involve critical structures such as the brainstem, spinal cord, and important nerves and arteries, chordomas are difficult to treat. Surgery and radiation are currently the only effective forms of treatment.

Read more at Wellcome Trust Sanger Institute

Image: Sacral bone chordoma. (Credit: Dr Harry Gouvas)