Some patients who were “supposed” to die years ago based on their cancer prognosis are not only alive today, they’re cancer-free and living well. That’s all thanks to a novel immunotherapy that primes the patients’ immune system to hunt down cancer cells and eradicate tumors.

Researchers at the Institute of Cancer Research (ICR), London, and the Royal Marsden NHS foundation trust may have struck medical gold by combining nivolumab and ipilimumab medications to significantly reduce the size, and in some odd cases completely eradicate, the tumors in patients diagnosed with terminal head and neck cancer.

Head and neck cancer is distinct from brain cancer, affecting tissues in the lip and oral cavity (mouth), larynx (throat), salivary glands, nose, sinuses or the skin of the face. Globally, head and neck cancer accounts for around 900,000 cases and over 400,000 deaths annually

Previous studies that tried the same combination and various amounts found similar benefits in terminally ill kidney, skin, and bowel cancer patients, suggesting the immunotherapy cocktail is active for a broad range of cancers.

Nivolumab is an anti-PD-1 drug, containing an antibody that promotes the tumor-killing effects of T-cells, a type of white blood cell that helps the body ward off disease. Ipilimumab, sold under the brand name Yervoy, is a monoclonal antibody medication used for adjuvant therapy in patients who have had surgery to remove melanomas in the skin and lymph nodes.

Almost 1,000 terminally ill head and neck cancer patients were given this combination as part of a phase 3 clinical trial. Early results suggest the therapy is extremely promising and, in some cases, life-saving, despite many aspects of the trials still ongoing.

“These are promising results,” Prof Kristian Helin, the ICR chief executive, told the Guardian. “Immunotherapies are kinder, smarter treatments that can bring significant benefits to patients.”

Guardian journalists talked to a number of patients who agreed to participate in the trial with a ‘nothing to lose’ attitude. Barry Ambrose, a 77-year-old man from Bury St Edmunds, was among them.

“Although I had to make biweekly trips from Suffolk to the hospital for the treatment, I had virtually no side-effects and was able to carry on as normal doing the things I love: sailing, cycling, and spending time with my family.”

Eight weeks later, the tumor in his throat had disappeared.

“When the research nurses called to tell me that, after two months, the tumour in my throat had completely disappeared, it was an amazing moment,” said Ambrose. “While there was still disease in my lungs at that point, the effect was staggering.”

The early results from the trial suggest that the immunotherapy combination was most successful in patients whose tumors had high levels of an immune marker called PD-L1. Compared to those receiving conventional chemotherapy, those who were given the cocktail lived three months longer, on average.

In 2020, the FDA approved the combination of nivolumab and ipilimumab as first-line treatment for patients with metastatic non-small cell lung cancer whose tumors express PD-L1. In a clinical trial involving 793 cancer patients from the United States, researchers found the therapy improved overall survival from 14.9 months to 17.1 months.

Researchers want to continue this work by performing follow-up studies. It is still rather early and they would like to determine with greater confidence whether the therapy improves survival rates across all patients in the trial.