EXPERT REACTION: New class of cancer drug targeting the most commonly mutated gene shows early promise

Publicly released:
Australia; International; QLD
Photo by Jon Tyson on Unsplash
Photo by Jon Tyson on Unsplash

A new class of cancer drug, which targets the most commonly mutated gene in human cancers, has shown promise in an early-stage trial with 20% response rate in patients with advanced solid cancers. The gene, called p53, is what's known as a tumour suppressor gene, and it is mutated in at least half of all people diagnosed with cancer. The trial drug, rezatapopt, reactivates a form of p53 that has one specific mutation. The small trial of 77 patients with advanced cancer that had been previously treated with a range of other drugs found that around 20% of patients had a response to rezatapopt and their tumours shrank in size. The most common side effects were nausea and vomiting. A linked editorial says that the trial represents a proof of concept for tumour-suppressor reactivation, which has enormous potential in cancer therapy. The next phase trials of this drug are currently recruiting patients in Australia

Expert Reaction

These comments have been collated by the Science Media Centre to provide a variety of expert perspectives on this issue. Feel free to use these quotes in your stories. Views expressed are the personal opinions of the experts named. They do not represent the views of the SMC or any other organisation unless specifically stated.

Professor Nigel McMillan is a Professor in the School of Pharmacy and Medical Sciences at Griffith University

"P53 is mutated in around 70% of all cancers and is therefore an attractive target for therapy, yet despite many years of research, it has proven difficult for researchers to develop drugs to.  Part of the problem is there are many different mutations that affect this master regulator of cell survival.  This paper presents promising results from an early phase clinical trial using a new drug, Rezatapopt, which targets one particular mutation.  The trial showed 20% of patients had a response (at least 30% tumour shrinkage), and in a wide range of cancer types.

These results are highly impressive for such an early stage of trial in patients who had all undergone many different therapies before entering this trial. These are the most resistant and difficult cases, as their cancers were highly advanced.

This approach is attractive as Rezatapopt would be able to treat many different cancer types, and it can be taken orally, so it could be taken at home.

It offers a hope that we might finally have at least a partial solution to targeting the most important protein in cancer.

A larger trial is underway that will test if treatment will cure patients of their cancer."

Last updated:  25 Feb 2026 11:58am
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Declared conflicts of interest I am the receipt of grants form the NHMRC, MRFF and Nation Breast Cancer Foundation. I have no other financial interests to declare.

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Research Massachusetts Medical Society, Web page
Editorial / Opinion Massachusetts Medical Society, Web page Please link to the article in online versions of your report (the URL will go live after the embargo ends).
Journal/
conference:
New England Journal of Medicine
Research:Paper
Organisation/s: University of Texas, Memorial Sloan Kettering Cancer Center, USA
Funder: Supported by PMV Pharmaceuticals.
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