A new international study has found that combining two cancer medicines may significantly slow the growth of a dangerous form of prostate cancer in some men. The research was led by scientists at University College London (UCL) and involved hundreds of patients from many countries.
The findings were published in the medical journal Nature Medicine and offer new hope for people whose cancer carries certain genetic changes that make the disease harder to treat.
Prostate cancer is one of the most common cancers in men. The prostate is a small gland that sits below the bladder and helps produce semen. As men age, cells in the prostate can sometimes grow out of control and form a tumor. In many cases, prostate cancer grows slowly and can be managed with treatment.
However, some forms of the disease are aggressive and can spread to other parts of the body, such as the bones or lymph nodes. When the cancer spreads in this way, it becomes much more difficult to treat and can threaten a person’s life.
Scientists have been working for many years to understand why some prostate cancers become aggressive while others do not. One important clue lies in a group of genes that normally help repair damaged DNA inside cells. This system is known as homologous recombination repair, or HRR.
These genes act like a repair team that fixes mistakes in the DNA of cells. When the system works well, it helps prevent cells from turning into cancer. But when these genes are damaged or mutated, the repair process breaks down. As a result, cancer cells can grow faster and spread more easily.
About one quarter of men with advanced prostate cancer have mutations in HRR-related genes. Some of the best-known genes in this group include BRCA1 and BRCA2, which are also linked to breast and ovarian cancer.
Other genes such as CHEK2 and PALB2 can also play a role. When these genes stop working properly, the cancer becomes more aggressive and often responds less well to standard treatments.
To explore new ways to treat this type of prostate cancer, researchers carried out a large clinical study known as the AMPLITUDE trial. This was a Phase III trial, meaning the treatment had already shown promise in earlier studies and was now being tested in a large group of patients to confirm its effectiveness and safety.
The study included 696 men from 32 countries around the world. All of the participants had prostate cancer that had already spread to other parts of the body and had not yet started treatment for this stage of the disease. The average age of the participants was 68. Every patient also had a mutation in one of the HRR genes.
Half of the patients were given the current standard treatment, which includes the medicines abiraterone acetate and prednisone.
These drugs are commonly used to slow the growth of prostate cancer by reducing the effect of male hormones that help cancer cells grow. The other half of the patients received the same standard treatment plus an additional medicine called niraparib.
Niraparib belongs to a class of medicines called PARP inhibitors. These drugs target a weakness in cancer cells that already have trouble repairing DNA. By blocking another repair pathway, PARP inhibitors make it even harder for the cancer cells to fix damage.
Eventually the cancer cells accumulate too much damage and die. Healthy cells are usually less affected because their repair systems still work.
The AMPLITUDE trial was designed as a double‑blind study. This means that neither the patients nor the doctors knew who was receiving the new drug and who was receiving a placebo. This method helps prevent bias and ensures the results are as reliable as possible.
After following the patients for a median period of about 30.8 months, the researchers saw clear differences between the two groups. Men who received niraparib together with the standard treatment had a significantly lower risk of their cancer getting worse.
Across all patients in the trial, the risk of cancer progression was reduced by 37 percent compared with those who received standard therapy alone.
The benefit was even stronger in patients whose cancers carried BRCA1 or BRCA2 mutations. In this group, the risk of disease progression was reduced by nearly half, about 48 percent. This suggests that the treatment may be particularly powerful for patients whose cancer has these specific genetic weaknesses.
The study also found that symptoms of the disease took longer to worsen in patients receiving the new combination therapy.
Only about 16 percent of patients in the niraparib group experienced serious symptom progression, compared with 34 percent in the group that did not receive the drug. In simple terms, the treatment helped many patients maintain a better quality of life for a longer time.
Researchers also observed signs that the new treatment might improve overall survival. However, the follow‑up period is still ongoing, and scientists need more time to confirm whether the drug combination helps patients live longer.
As with many cancer treatments, side effects were more common in patients who received niraparib. Some patients developed anemia, which is a condition where the body does not have enough healthy red blood cells. High blood pressure was also reported more often in the niraparib group.
Around one quarter of the patients needed blood transfusions during treatment. There were also slightly more treatment‑related deaths in the niraparib group compared with the placebo group, although the number was still relatively small. Overall, most patients were able to continue treatment.
The researchers say the findings highlight the growing importance of genetic testing in cancer care. By testing tumors for HRR gene mutations at the time of diagnosis, doctors may be able to identify patients who are most likely to benefit from targeted therapies such as niraparib.
Globally, about 1.5 million men are diagnosed with prostate cancer each year. In the United Kingdom alone, more than 56,000 men are diagnosed annually, and around 12,000 die from the disease. Because prostate cancer is so common, even modest improvements in treatment can have a major impact on public health.
The AMPLITUDE trial was sponsored by Janssen Research and Development, a division of Johnson & Johnson. The results suggest that combining targeted genetic therapies with existing hormone treatments could represent an important step forward in personalized cancer care.
Overall, the study provides strong evidence that using niraparib alongside standard treatment can slow disease progression in patients with certain genetic forms of advanced prostate cancer. However, the therapy also carries risks and side effects that must be carefully weighed.
More long‑term research is needed to determine whether the treatment improves survival and to better understand which patients will benefit the most. Even so, the findings mark an important advance in the effort to tailor cancer treatments based on the unique genetic features of each patient’s disease.
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