Potential treatment for rare spine cancer discovered by California researchers

Researchers at Santa Monica, Calif.-based Providence Saint John’s Health Center published new findings from a clinical trial that shed light on a possible treatment for a rare spine cancer.

The clinical trial was small in scale, enrolling only 15 patients who had previously been treated for progressive chordoma — a rare solid tumor that forms at the base of the skull and spine. Patients were given a high-dose of intravenous pemetrexed once every three weeks. 

The results, which were published Dec. 4 in Clinical Cancer Research, show that 71% of patients in the trial had a favorable response to the high-doses of pemetrexed. The drug was chosen for the research specifically "based on the observation that approximately 80% of chordoma tumors have low levels of the enzyme thymidylate synthase — a characteristic associated with response to pemetrexed in some other tumor types," according to a news release shared with Becker's

"While no drugs have been approved by the Food and Drug Administration to treat chordoma, pemetrexed appears to be an effective drug in our study. A subset of patients had tremendous clinical benefit," Santosh Kesari, MD, PhD, lead study author and director of neuro-oncology at Pacific Neuroscience Institute and Saint John's Cancer Institute, stated in the Dec. 4 release. "We plan to build on this framework of pemetrexed and combine it with other drugs, including immunotherapy, in future clinical trials."

Adverse side effects were minimal and rare and the trial yielded no unexpected toxicities, or dose-limiting toxicities. The work will next move into a larger scale, phase 2 trial.

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