Breakthroughs in Bladder Cancer Treatment Revolutionize Survival Rates, Unveiling Unprecedented Treatment Options

A new combination of cancer medications has shown promising results in extending the overall survival of patients with advanced bladder cancer compared to the current standard of care chemotherapy treatments. The research, presented at this year's European Society for Medical Oncology Congress in Madrid, reveals that the combination of enfortumab vedotin and pembrolizumab, delivered through IV infusions, reduces the risk of disease progression or death by 55% in previously untreated patients with advanced bladder cancer that has spread to other parts of the body. This combination treatment extended the median overall survival to 31.5 months, whereas standard chemotherapy only provided 16.1 months.

This study is part of a series of new research studies that aim to enhance treatment methods for bladder cancer.

According to Dr. Thomas Powles, a renowned expert in urology cancer and the director of the Barts Cancer Centre at the University of London in the United Kingdom, chemotherapy has shown promising results in the beginning but resistance develops rapidly. The median survival rate for metastatic bladder cancer has only been approximately a year. Dr. Powles presented these findings during the recent research conference.

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The less is more approach is revolutionizing cancer treatment for certain patients. In a significant Phase 3 study, two novel drugs were compared to standard chemotherapy. The results revealed that when these two drugs were administered together, the risk of death for this type of cancer decreased by more than 50%. This translated to a doubling of survival rates, with patients now living twice as long, and achieving long-lasting remission.

Bladder cancer is a prevalent form of cancer in men worldwide, ranking sixth in frequency. Annually, approximately 200,000 individuals lose their lives to this disease, with a significant number succumbing to advanced stages that have spread from the bladder. Treatment options are typically determined based on the stage of the disease, with options ranging from surgery to commonly used chemotherapy drugs like carboplatin and cisplatin. Pertinent to note, the latest research findings have been funded by leading US drug manufacturers Seagen, Inc., Astellas Pharma, and Merck & Co. These findings are particularly significant in light of the current scarcity of platinum-based chemotherapy drugs, such as carboplatin and cisplatin, in the United States.

In the Phase 3 trial, 886 patients were randomly assigned to receive either three-week cycles of the enfortumab vedotin and pembrolizumab combination, administered through IV infusions, or a chemotherapy treatment called gemcitabine with cisplatin or carboplatin.

The trial results showed that the new drug combination had a comparable safety profile to the chemotherapies. Adverse reactions like skin rashes or hyperglycemia were observed in 55.9% of patients receiving the combination, whereas 69.5% of those receiving platinum chemotherapy experienced such reactions.

Just three weeks ago, we received the data, and since then, things have been moving at an incredibly rapid pace," Powles stated. The research team's next move is to submit their findings to various agencies worldwide, including the US Food and Drug Administration. However, it is important to note that the data has not been published in a peer-reviewed journal at this point.

A cancer patient holds the IV tubes during chemotherapy.

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US cancer centers continue to experience significant shortages of crucial chemotherapy medications, according to a recent survey. The findings are so conclusive that conducting a larger study is unnecessary. Instead, the focus now lies on promptly delivering this vital information to relevant agencies such as the FDA in the United States, the EMA in Europe, and other global regulatory bodies. Urgent approval of this research is imperative.

According to Dr. Toni Choueiri, director of the Lank Center for Genitourinary Oncology at the Dana-Farber Cancer Institute, the research findings by Powles and his colleagues have the potential to revolutionize the care for advanced bladder cancer. Dr. Choueiri, who is currently attending the ESMO Congress in Madrid, stated that historically, gemcitabine and platinum have been the standard treatment for metastatic bladder cancer. However, the combination of pembrolizumab, an immunotherapy checkpoint blocker, and enfortumab vedotin, an antibody-drug conjugate, has proven to be far superior to the old regimen that has been used since the late 1990s. Dr. Choueiri believes that this new combination will likely become the new standard of care.

Another approach with longer survival

Additional research has assessed alternative approaches for managing a distinct group of individuals diagnosed with advanced bladder cancer, specifically those whose condition exhibited ongoing advancement despite undergoing either one or two prior treatment modalities.

Another Phase 3 trial demonstrated the efficacy of a targeted therapy in treating patients with advanced or metastatic urothelial cancer with specific gene mutations. Urothelial cancer, a form of bladder cancer, originates from the urothelial cells that line the interior of the bladder. Urothelial carcinomas account for the majority of bladder cancers.

On October 1, 2023, a woman is seen working at the Ose Immunotherapeutics laboratory in Nantes, diligently working on developing a vaccine for lung cancer. The photograph captures her dedication and commitment. (Photo credit: LOIC VENANCE / AFP) (Photo by LOIC VENANCE/AFP via Getty Images)

Researchers are puzzled by the increased incidence of lung cancer in women. A recent study, published in the New England Journal of Medicine on Saturday, examined the survival rates of 136 patients who received the anti-cancer drug erdafitinib orally, compared to 130 patients who received traditional chemotherapy. These patients had previously undergone various forms of treatment for their cancer and were tracked for an average of 15.9 months.

The researchers from the University of Texas MD Anderson Cancer Center and other US institutions conducted a Phase 3 study funded by drugmaker Janssen. They found that erdafitinib therapy is more effective than chemotherapy for patients with newly diagnosed advanced and metastatic urothelial cancer. In the study, it was noted that over 50% of patients with metastatic urothelial carcinoma cannot receive cisplatin treatment and experience progression within a few months after chemotherapy. The researchers reported that the median overall survival for patients on erdafitinib therapy was 12.1 months, whereas for patients on chemotherapy, it was 7.8 months.

In 2019, erdafitinib received accelerated approval from the US Food and Drug Administration. This approval was specifically for patients with locally advanced or metastatic bladder cancer who have certain gene mutations and have experienced disease progression during or after chemotherapy. Remarkably, erdafitinib became the first FDA-approved therapy for metastatic urothelial cancer that targets a genetic alteration.

Erdafitinib is a kinase inhibitor medication that works by blocking the protein responsible for signaling cancer cells to multiply. By doing so, it effectively slows down or stops the spread of cancer cells, as stated by the National Library of Medicine. During the Phase 3 trial, the incidence of treatment-related adverse events was comparable between erdafitinib and chemotherapy recipients. However, fatalities were less frequent among erdafitinib patients (0.7%) compared to those receiving chemotherapy (5.4%).

A new standard approach

A recent article, which appeared in the New England Journal of Medicine and is also being presented at the ESMO Congress, revealed that the utilization of a combination of chemotherapy and immunotherapy drugs has led to enhanced survival rates in individuals who have advanced bladder cancer and have not received any previous treatment, as opposed to those who solely underwent chemotherapy.

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The Phase 3 trial included 608 patients with advanced bladder cancer that had spread or was unable to be surgically removed and had not been previously treated. Among the participants, half received intravenous infusions of the immunotherapy drug nivolumab along with the chemotherapies gemcitabine and cisplatin every three weeks for up to six cycles, followed by nivolumab every four weeks for a maximum of two years. The other half received gemcitabine-cisplatin alone every three weeks for up to six cycles. The researchers observed that 61.8% of patients receiving the nivolumab combination experienced grade 3 or higher adverse events, such as anemia or urinary tract infections, compared to 51.7% of those in the chemotherapy group, which was consistent with findings from a Phase 2 trial.

The research team, which consisted of scientists from Mount Sinai and other international institutions, discovered that the median overall survival with the nivolumab combination therapy was 21.7 months, whereas with gemcitabine-cisplatin alone, it was 18.9 months. The trial was funded by Bristol Myers Squibb, in collaboration with Ono Pharmaceutical Company.

Study author Dr. Matthew Galsky, co-director of the Center of Excellence for Bladder Cancer at The Tisch Cancer Institute at Mount Sinai, stated that, until now, no new agent added to first-line standard-of-care cisplatin-based chemotherapy has shown improved overall survival in metastatic urothelial carcinoma. However, he expressed that the results of the study support the use of nivolumab plus cisplatin-based chemotherapy as a new standard approach for treating metastatic urothelial cancer.