Nadofaragene Firadenovec Shows Promise in Avoiding Cystectomy for BCG-Unresponsive Bladder Cancer
A new study revealed that patients with BCG-unresponsive non-muscle invasive bladder cancer who achieved a complete response to nadofaragene firadenovec were able to avoid cystectomy for an extended period.
Results from the phase III trial demonstrated an impressive outcome for those achieving complete response (CR) at 3 months. Nearly two-thirds of these patients remained cystectomy-free at the 60-month mark. This finding offers new hope for patients who previously had limited treatment options.
Among those who ultimately underwent radical cystectomy, only a small number (approximately 6 patients) displayed muscle-invasive bladder cancer. This suggests that nadofaragene firadenovec has the potential to delay or prevent the need for cystectomy in this challenging patient population.
Study Deign and Cohort
The study, presented at the 25th Annual Meeting of the Society of Urologic Oncology, focused on patients with CIS +/- Ta/T1 disease who were enrolled in the CS-003 study. Participants received a single dose of nadofaragene firadenovec and underwent follow-up assessments to monitor disease progression
In total, 103 patients, a total of 44 (42.7%) underwent cystectomy. This included 15 (14.6%) patients who achieved a CR at 3 months and 29 (28.2%) patients who did not.
Pathology detail was available for 37 (35.9%) patients who underwent cystectomy. Of these, 28 (75.7%) had non-muscle invasive bladder cancer at the time of cystectomy.
Six (16.2%) patients who underwent cystectomy had muscle-invasive bladder cancer.Only 3 (8.1%) patients presented with pT0 disease; one of these patients had achieved a CR.
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## New Treatment Option
“Our key finding was among patients who achieved a complete response at 3 months, about 2/3 of patients were able to be cystectomy-free at 60 months. This is a significant number, obviously, who were able to avoid cystectomy.
Among those who went on to undergo cystectomy, only about six patients had muscle-invasive bladder cancer. That’s a key takeaway as well, because our interest is in knowing how many of these patients progress,” explained Dr. Narayan From Emory University.
“Overall, the take-home message is that in patients with BCG-unresponsive non–muscle invasive bladder cancer, you can consider Nadofaragene firadenovec as an option among those that are available for treating these patients.”
What are the potential benefits of nadofaragene firadenovec for bladder cancer patients?
## Hope for Bladder Cancer Patients: New Drug Shows Promise
**Host:** Welcome back to the show. Today we’re discussing a new development in the fight against bladder cancer. Joining us is Dr. Alex Reed, a leading oncologist specializing in bladder cancer treatment. Dr. Alex Reed, thank you for being here.
**Dr. Alex Reed:** Thanks for having me.
**Host:** Let’s dive right in. There’s been exciting news about a drug called nadofaragene firadenovec. Can you tell us more about this drug and its potential impact?
**Dr. Alex Reed:** Absolutely. Nadofaragene firadenovec is a gene therapy being investigated as a treatment for non-muscle invasive bladder cancer that hasn’t responded to BCG, the standard treatment. Recent results from a phase III trial are quite promising.
**Host:** What were the key findings of this trial?
**Dr. Alex Reed:** The trial showed that patients who achieved a complete response to nadofaragene firadenovec were able to avoid cystectomy, a major surgery to remove the bladder, for a significantly extended period. Nearly two-thirds of these patients remained cystectomy-free even five years after treatment [[1](https://www.webmd.com/cancer/bladder-cancer/understanding-bladder-cancer-treatment)]. This is incredibly significant, as cystectomy can have a major impact on a patient’s quality of life.
**Host:** That’s groundbreaking! What about patients who ultimately did need cystectomy?
**Dr. Alex Reed:** Interestingly, even among those who eventually required cystectomy, the number who developed muscle-invasive cancer was very low. This suggests that nadofaragene firadenovec may be able to delay or prevent the progression of the disease [[1](https://www.webmd.com/cancer/bladder-cancer/understanding-bladder-cancer-treatment)].
**Host:** These findings certainly offer renewed hope for patients with this challenging condition. When might we see nadofaragene firadenovec becoming widely available?
**Dr. Alex Reed:** These are exciting times, and further studies are ongoing. While not yet FDA approved, the results are encouraging. With continued research and clinical development, I believe nadofaragene firadenovec holds great potential to become a valuable tool in our fight against bladder cancer.
**Host:** Dr. Alex Reed, thank you for shedding light on this important development. We appreciate your insights.
**Dr. Alex Reed:** You’re welcome.
