Neurosurgeon Sean Barber, MD and colleagues offer a timely analysis of the most forward-looking treatments of chordoma in the Journal of Clinical Medicine. The current prognosis of patients with chordoma is poor, yet our growing understanding of the genetic and molecular basis for its pathophysiology shows promise in improving care. Ongoing research has led to the discovery of several pathways that may serve as potential targets for molecular therapy.

Chordoma, a rare tumor of the skull base, spine and sacrum, is challenging to treat because it presents late in the disease course, has a high recurrence after resection, invades adjacent neuro anatomy, and resists conventional radiotherapy and chemotherapy.

While wide surgical margins are currently the most important prognostic factor in treating chordoma, molecular sequencing, radiotherapy and clinical trials for molecular targeted agents should be part the overall treatment decision-making process. Advances in targeted medical therapy and radiotherapy, although controversial now, may drive these modalities to a more prominent role, with aggressive surgery being reserved only for the most advanced cases.

For more detailed discussion on advancements in treatment for chordoma, with an emphasis on promising ongoing molecular and genetic research, see the study in the Journal of Clinical Medicine, January 14, 2021.