Groundbreaking Trial Reveals New Drug Combination Delays Prostate Cancer Progression

A major international trial, led by researchers from the University of College London (UCL), has found that a new combination of drugs significantly delays the progression of a life-threatening form of prostate cancer in men with specific genetic mutations. The Phase III AMPLITUDE trial tested the addition of niraparib, a type of targeted cancer drug, to the standard treatment of abiraterone acetate and prednisone (AAP). The study focused on patients with advanced prostate cancer who had alterations in genes involved in DNA defect repair, known as homologous recombination repair (HRR).

Key Takeaways:

• The trial enrolled 696 men across 32 countries, with a median age of 68.
• Approximately one in four people with advanced prostate cancer at this stage have alterations in HRR genes, such as BRCA1, BRCA2, CHEK2, and PALB2.
• The study found that patients with HRR gene alterations showed a significant delay in disease progression when treated with niraparib plus AAP compared to standard treatment.
• Side effects were more common in the niraparib group, including anaemia, high blood pressure, and treatment-related deaths were higher, although overall discontinuation rates remained low.
• The results suggest that genomic testing at diagnosis can identify patients who may benefit from targeted treatment with niraparib.
• Further research is needed to confirm long-term survival benefits and to explore the impact of newer imaging techniques and broader genetic testing.

Statistics:

• 55.6% of patients had alterations in the BRCA1 or BRCA2 genes.
• 696 men were enrolled in the trial.
• The median follow-up was 30.8 months.
• Treatment-related deaths were 14 in the niraparib group and 7 in the AAP group.
• 25% of patients in the niraparib group required blood transfusions due to anaemia.

Sources:

• Gerhardt Attard et al. 'Niraparib and abiraterone acetate plus prednisone for HRR-deficient metastatic castration-sensitive prostate cancer: a randomized phase 3 trial' (Nature Medicine, October 2025).
• UCL Cancer Institute Press Release (October 2025).
• National Institute for Clinical Excellence (TA1032, October 2025).