In the middle of December, the Food and Drug Administration approved Adstiladrin (Nadofaragene firadenovec) for patients with unresponsive high-risk early stage bladder cancer.

Adstiladrin is a gene therapy drug. Gene therapy is a new generation of medicine that uses genes to treat, prevent or cure medical disorders.

The approval of Adstiladrin was based on results from a clinical trial enrolling 157 patients who met the abovementioned criteria. Patients participating in the trial received Adstiladrin once every 3 months for up to one year, and afterwards according to their follow-up assessment.

At 3 months from the beginning of the trial, more than half of the patients treated with Adstiladrin had complete response (i.e. no evidence of disease). Half of them remained in complete response for at least one year while, with longer follow-up, recurrence became much rarer.

Regarding toxicity, most patients had manageable side effects, except for 3 patients (1.9%) who stopped the medication secondary to treatment effects.

The researchers believe that Adstilatrin provides promising results regarding novel therapy for patients with unresponsive high-risk early stage bladder cancer.