A phase I clinical trial exploring a personalised cancer vaccine (PCV) has shown promising results for patients with high-risk, fully resected clear cell renal cell carcinoma (RCC), a form of kidney cancer. The study, which enrolled nine participants, tested a neoantigen-targeting vaccine designed to generate immune responses against cancer driver mutations. The trial was conducted with or without the immune checkpoint inhibitor ipilimumab and aimed to determine whether such vaccines could prevent recurrence of RCC in patients with low mutational burden.
At a median follow-up of 40.2 months post-surgery, all nine patients remained free of recurrence, with no dose-limiting toxicities observed. The vaccine successfully induced T cell immune responses against key RCC driver mutations, including VHL, PBRM1, BAP1, KDM5C, and PIK3CA. Notably, T cell reactivity was detected against the patients’ own tumours in seven out of the nine participants.
These findings demonstrate that neoantigen-targeting PCVs can trigger robust immune responses and target critical mutations driving cancer. The durable expansion of T cell clones and the absence of recurrence in all vaccinated patients provide strong evidence for the potential of PCVs as an effective adjuvant therapy for high-risk RCC. As the research progresses, these results suggest a promising future for personalised vaccines in cancer immunotherapy.
Helena Bradbury, EMJ
Reference
Braun DA et al. A neoantigen vaccine generates antitumour immunity in renal cell carcinoma. Nature. 2025; doi: 10.1038/s41586-024-08507-5.
