
In a large global trial led by University College London (UCL), scientists discovered that adding the drug niraparib to standard prostate cancer therapy significantly delayed disease progression in men whose tumors carry DNA repair gene mutations.
The results, published in Nature Medicine, could change how doctors treat advanced prostate cancer and pave the way for a new era of precision oncology.
Prostate cancer is one of the most common cancers in men, but it can behave very differently depending on genetics. About one in four men with advanced prostate cancer carry mutations in homologous recombination repair (HRR) genes — including BRCA1, BRCA2, CHEK2, and PALB2 — which make the cancer more aggressive and harder to treat.
The AMPLITUDE Phase III trial, led by Professor Gerhardt Attard at UCL’s Cancer Institute, tested whether adding niraparib — a PARP inhibitor that targets tumors with faulty DNA repair — could improve outcomes when combined with abiraterone acetate and prednisone (AAP), the current standard treatment.
The trial enrolled 696 men with newly diagnosed, metastatic prostate cancer across 32 countries, with a median age of 68. Half received the niraparib + AAP combination, while the other half were given AAP + placebo. More than half had mutations in the BRCA1 or BRCA2 genes.
After about two and a half years of follow-up, the results were clear:
Symptoms took twice as long to worsen, compared to those on standard therapy
There was also an encouraging trend toward improved overall survival, though researchers say longer follow-up is needed to confirm that benefit.
Professor Attard explained that this approach represents a vital shift:
“We now know that prostate cancers with HRR mutations behave more aggressively. By combining niraparib with standard therapy, we can delay the cancer returning and hopefully extend life expectancy. These findings also support routine genetic testing so that the right patients receive the right treatment.”
While the combination therapy was largely safe, anemia and high blood pressure occurred more frequently in patients receiving niraparib. Around 25% required blood transfusions. Treatment-related deaths were slightly higher (14 vs. 7), but overall dropout rates were low — a sign that most patients tolerated the therapy well.
The study reinforces the growing importance of genomic testing in prostate cancer care. By identifying patients with DNA repair gene mutations, doctors can tailor treatments to exploit the cancer’s genetic vulnerabilities — improving survival and quality of life.
Globally, 1.5 million men are diagnosed with prostate cancer each year. In the UK alone, more than 56,000 men face the disease annually, and around 12,000 die from it. Targeted advances like this could help change that trajectory.
While more research is needed to confirm long-term survival benefits, the AMPLITUDE trial marks a major milestone. It not only highlights the potential of niraparib in prostate cancer but also showcases how precision medicine — tailoring therapy to a patient’s genetic makeup — can transform outcomes for even the most aggressive forms of the disease.