FDA Approves First Gene Therapy for Bladder Cancer

The US Food and Drug Administration (FDA) approved Adstiladrin, the first gene therapy for high-risk, non-muscle-invasive bladder cancer.

Adstiladrin (nadofaragene firadenovec-vncg) is non-replicating adenoviral vector-based gene therapy. It is approved for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

NMIBC is a type of cancer that has grown through the lining of the bladder but hasn't yet invaded the muscle layer. This type of cancer is more likely to recur and progress to invasive and metastatic cancer. About 75% to 80% of newly diagnosed bladder cancers are classified as NMIBC.

CIS are abnormal cancer cells found where they first formed and that have not spread to nearby tissue.

Treatment and care of patients with high-risk NMIBC often involve removing the tumor and using BCG to reduce the risk that cancer will recur.

BCG is the most common intravesical immunotherapy for treating early-stage bladder cancer that prevents cancer from growing and coming back.

When BCG, a germ that usually does not cause severe disease, is put into the bladder through a catheter, it reaches the cancer cells and activates the immune system, which then attacks the bladder cancer cells.

Developing BCG-unresponsive disease is associated with an increased risk of death or a disease-worsening event.

How effective is the therapy?

The clinical study evaluating Adstiladrin's safety and effectiveness included 157 patients who received therapy once every three months for up to 12 months or until the side effects of treatment became unacceptable, or cancer recurred.

In more than half (51%) of patients, all signs of cancer disappeared, and 46% of patients remained cancer-free for at least one year.

What causes bladder cancer?

According to the Centers for Disease Control and Prevention (CDC), about 57,000 men and 18,000 women receive bladder cancer diagnoses every year in the US. The disease costs some 16,700 lives annually.

Smoking is one of the main causes of bladder cancer. The other factors for the disease are having a family history of bladder cancer, chronic urinary tract infections, certain gene mutations, and being exposed to too much of workplace chemicals.

The most common symptom of bladder cancer is blood in the urine. The other signs of the disease may include having to urinate often, feeling pain while urinating, and experiencing back and pelvic pain.


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