Glaucoma Drugs Show Promise for Treating Aggressive Cancers

Rather than a one-size-fits-all approach, the ICR team identified distinct biological markers that could predict which tumours are most likely to respond to ROCK inhibitor treatment .

• Breast cancer (epithelial solid tumours): Cancer cells responded to ROCK inhibitors when the E-Cadherin gene was not functioning properly .
• Melanoma (non-epithelial solid tumours): Responsive cells had a more rounded shape and high activity of the NFKB signalling pathway, a driver of tumour growth, spread, and inflammation .
• AML (blood cancer): Cells that responded well carried a specific set of gene alterations .

Across all tumour types, cells more sensitive to ROCK inhibitors also showed higher activity in genes that regulate cell identity and division, providing a broader signature for patient selection .

From Database to Lab to Mouse Models

The research followed a rigorous multi-step validation process. The team first analysed a large drug-sensitivity database to uncover patterns linking ROCK inhibitor response to specific genetic and cellular traits. They then confirmed these patterns through direct lab experiments on patient tumour samples and in studies with mouse models .

A Faster Route to Patients

Because ROCK inhibitors such as fasudil, netarsudil, and ripasudil are already approved for glaucoma treatment and have well-documented safety profiles , repurposing them for cancer could dramatically accelerate the timeline to patient access. The drugs could bypass much of the early toxicity and safety testing required for entirely new compounds .

In the future, the team envisions a biopsy-based test: analysing a patient's tumour for markers like E-Cadherin malfunction, rounded cell shape paired with high NFKB activity, or specific gene alterations could quickly indicate whether they are likely to benefit from ROCK inhibitor therapy .

Next Steps: Combination Therapies and Clinical Trials

The study, which the ICR describes as 'laying the foundations' for precision repurposing, has a clear roadmap ahead. First author Dr. Jaume Barcelo (now at Barts Cancer Institute) stated that the immediate next stage involves testing ROCK inhibitors in combination with other existing treatments to maximise patient benefit. The team aims to progress into clinical trials in the near future .