Nadofaragene firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is a novel gene therapy used in the treatment of non-muscle invasive bladder cancer (NMIBC) that is unresponsive to Bacillus Calmette-Guérin (BCG). This therapy represents a significant advancement in the management of NMIBC, offering a new option for patients who have limited treatment choices. Nadofaragene firadenovec works by delivering a recombinant adenovirus vector carrying the gene for interferon alpha-2b directly into the bladder, where it is expressed and exerts anti-tumor effects.

Mechanism of Action

Nadofaragene firadenovec utilizes a non-replicating adenoviral vector to introduce the interferon alpha-2b gene into the bladder wall cells. Once inside the cells, the gene is expressed, and interferon alpha-2b is produced. Interferon alpha-2b is a cytokine with potent antiviral, antiproliferative, and immune-modulating effects. In the context of bladder cancer, these effects contribute to the reduction of tumor growth and the enhancement of the body's immune response against cancer cells.

Clinical Trials

Several phase I and II clinical trials have demonstrated the safety and efficacy of nadofaragene firadenovec in patients with NMIBC. The most notable phase III trial showed a significant response rate in patients with high-grade NMIBC who were unresponsive to BCG therapy. These patients experienced a durable response, with many achieving a complete response at the 12-month follow-up.

Administration

Nadofaragene firadenovec is administered directly into the bladder through a catheter in an outpatient setting. The treatment regimen typically involves an initial induction phase followed by a maintenance phase, depending on the patient's response to therapy.

Side Effects

The most common side effects associated with nadofaragene firadenovec include mild to moderate flu-like symptoms, such as fever, fatigue, and muscle aches. These symptoms are generally transient and can be managed with standard supportive care. Other reported side effects include bladder irritation and urinary symptoms, which are also typically transient.

Future Directions

Research is ongoing to further understand the potential of nadofaragene firadenovec in NMIBC and to explore its use in other types of cancer. Additionally, studies are investigating the combination of nadofaragene firadenovec with other therapies to enhance its efficacy and broaden its applicability in oncology.

See Also

• Bladder cancer
• Gene therapy
• Interferon alpha-2b
• Non-muscle invasive bladder cancer
• Bacillus Calmette-Guérin