Proton Therapy Demonstrates Advantages in Phase 3 Head & Neck Cancer Trial

According to preliminary data from a multi-institution Phase 3 trial led by researchers at The University of Texas MD Anderson Cancer Center, intensity modulated proton therapy (IMPT) achieved similar clinical outcomes and offered significant patient benefits when compared to traditional intensity modulated radiation therapy (IMRT) as part of chemoradiation treatment for patients with oropharyngeal (head and neck) cancer.

The results were presented today at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting by Steven Frank, MD, professor of Radiation Oncology and Executive Director of the Particle Therapy Institute at The University of Texas MD Anderson Cancer Center.

With a median follow-up of 3 years, the progression-free survival (PFS) rate was 83% and 83.5% for IMPT and IMRT, respectively, and IMPT was statistically non-inferior to IMRT. There was a significant reduction of malnutrition with IMPT, with 24% of patients sustaining their nutrition with less than 5% weight loss during treatment compared with 14% of those receiving IMRT. Additionally, there was a significant reduction of feeding-tube dependence with IMPT at 28%, compared to 42% with IMRT.

“The results of this multi-center Phase 3 randomized trial provide evidence for IMPT as a new standard-of-care treatment approach for the management of head and neck tumors,” Frank said. “This is significant for patients as it represents a curative, de-intensified option compared to traditional radiation therapy.”

Proton therapy has both biological and physical advantages over traditional radiation therapy using photons. Unlike photons, protons have mass and can be stopped by the human body. This allows proton radiation to be delivered specifically to the targeted area, limiting the amount that reaches nearby normal tissues. This trial represents the largest randomized Phase III trial to date to investigate proton therapy in comparison to traditional radiation.

The trial enrolled 440 patients at 21 sites in the US, with 219 receiving IMRT and 221 receiving IMPT. Patients were stratified based on human papillomavirus (HPV) status, smoking status and whether they had received induction chemotherapy. The primary endpoint of the study was the PFS rate at 3 years.

“Historically, this kind of large-scale trials to confirm the benefits of proton therapy have been challenging, due in part to relatively few patients having access to proton therapy centers,” Frank said.” Encouraging results like these demonstrate the benefits of proton therapy and hopefully help pave the way for increased access for patients in need.”

This study was funded by grants from the National Institutes of Health (NIH)/National Cancer Institute (NCI) (U19CA021239, R03CA188162, R56DE025248) and Hitachi. Frank reports proton-related grant funding by Hitachi and honoraria fees from Ion Beam Applications S.A. (IBA). He also has non-proton related health care relationships with Boston Scientific (consulting fees), Affirmed Pharma (NIH grant), and C4 Imaging (founder, scientific advisory committee, patents/royalties, ownership interest). A full list of collaborating authors and their disclosures can be found with the abstract here.

The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world’s most respected centers focused on cancer patient care, research, education and prevention. The institution’s sole mission is to end cancer for patients and their families around the world, and, in 1971, it became one of the nation’s first National Cancer Institute (NCI)-designated comprehensive cancer centers. MD Anderson is No. 1 for cancer in U.S. News & World Report’s “Best Hospitals” rankings and has been named one of the nation’s top two hospitals for cancer since the rankings began in 1990. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).