In patients with high-grade cancer in the lining of the bladder, cancer often comes back within a year following standard treatment.
Although there are several approved treatments, none have significantly improved survival with few side effects. However, a new study evaluating a novel immunotherapy called nadofaragene firadenovec (NF) gene therapy may offer new hope.
According to the study, 53.4% of participants receiving NF were free of cancer at three months and 45.5% of patients continued to be cancer-free for at least 12 months. The Phase 3 study included 153 patients from 33 U.S. institutions including the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center.
“We were very pleased with the results of this study and feel that nadofaragene firadenovec has the potential to be a breakthrough treatment. We are continuing to follow these patients for four years to learn more about how they respond to this treatment long term,” said Robert Svatek, MD, principal investigator of the Mays Cancer Center study site. He also is professor and chair of the Department of Urology at UT Health San Antonio.
“This product is remarkable as one of the first virus-delivered gene therapies to treat cancer. NF is a virus that has been altered to transport a genetically modified protein called interferon alfa-2b. Once in the bladder, interferon alfa-2b prompts the cells in the lining of the bladder to make more of this protein to fight cancer, strengthening the body’s immune response,” he added.
In previous studies, interferon alfa-2b showed some effectiveness, but the medication was not active in the bladder long enough to produce good results. By piggy-backing the protein and delivering it with a virus in a liquid placed in the bladder through a catheter, the protein is contained in the bladder for an hour, long enough to provide a significant immune response. The NF liquid is expelled through urination.
Patients that responded well to the treatment received three more doses of NF at three, six and nine months.
“NF has fewer and less serious side effects than other treatments approved to treat this type of cancer. NF also allows patients to avoid surgery to remove the bladder, which is the next treatment to consider if the current standard treatment fails,” Dr. Svatek explained.
According to the American Cancer Society’s key statistics, about 83,730 new cases of bladder cancer and approximately 17,200 new deaths from this disease are expected in 2021. It is the fourth-most-common type of cancer in men and is much less common in women.
This study was published Nov. 27 in Lancet Oncology, DOI: 10.1016/S1470-2045(20)30540-4. Funding was provided by FKD Therapies Oy.
Other treatments for bladder cancer
Bacillus Calmette-Guérin (BCC) is the current standard treatment for cancer in the lining of the bladder, also called non-muscle invasive bladder cancer. BCG is initially effective in about 80% of patients. Some bladder tumors, however, do not respond to BCG.
If this occurs, the next recommended treatment is radical cystectomy – complete removal of the bladder. “This is a complicated surgery, especially for patients who have other health conditions,” Dr. Svatek said. In addition to removing the bladder, the surgeon makes a new vessel from intestinal tissue to hold the urine. This surgery permanently changes the way people urinate and can cause serious side effects. Another concern is that there is a worldwide shortage of BCC due to manufacturing issues.
To learn more about other treatments and quality-of-life issues in bladder cancer, see this Jan. 28 article in the ASCO Daily News written by UT Health San Antonio faculty members Kakarla Sushanth, MD, Deepak K. Pruthi, MD, FRCSC, and Chethan Ramamurthy, MD.