Gene therapy increases survival for end-stage head and neck cancer

Drug that restores tumor-suppressor gene expression originated at M. D. Anderson

Jack Roth, M.D., professor in M. D. Anderson's Department of Thoracic & Cardiovascular Surgery. Click here for more information.

HOUSTON - A gene therapy invented at The University of Texas M. D. Anderson Cancer Center is the first to succeed in a U.S. phase III clinical trial for cancer, as announced today at the American Society of Gene Therapy annual meeting in Boston.

Introgen Therapeutics, Inc., reported results of its phase III trial of Advexin(r), a modified adenovirus that expresses the tumor-suppressing gene p53, for end-stage head and neck cancer.

"Cells become cancerous because p53 no longer functions. Restoring p53 works unlike any current cancer treatment because it treats the cancer genome," said Jack Roth, M.D., professor in M. D. Anderson's Department of Thoracic & Cardiovascular Surgery, who invented the drug and co-founded Introgen. He remains a shareholder and paid consultant to Introgen, and the University of Texas System is also a shareholder in Introgen.

The p53 gene is inactivated in many types of cancer. Its normal role is to halt the division of a defective cell and then force the cell to kill itself.

The trial showed that p53 expression in the patient's tumor before treatment is a reliable biomarker for how to treat head and neck cancer. Patients with a favorable p53 profile who received Advexin(r) had a median survival of 7.2 months, compared with 2.7 months for those whose tumor expressed high levels of mutant p53 before treatment. Patients with this unfavorable profile were better off taking the chemotherapy drug methotrexate, resulting in median survival of 5.9 months.

"The important finding is that patients who benefit from treatment can be identified with the p53 biomarker. The biomarker will enable physicians to personalize treatment," said Roth, who also directs M. D. Anderson's W.M. Keck Center for Innovative Cancer Therapies.