The field of gene therapy has reached a significant milestone with the development of an inhalable gene therapy for lung cancer. This groundbreaking treatment has shown promising results in clinical trials and has been fast-tracked for potential approval.
The therapy involves genetically modifying lung cells by using a virus to carry immune-boosting genes into the cells. This modification enables the cells to better fight tumors on their own, leading to shrinkage of lung tumors in patients. What sets this therapy apart is not only its mode of action but also its mode of delivery – it is inhaled rather than swallowed or injected. By directly delivering the therapy into the lungs, it overcomes the challenge of getting treatments to reach the lungs effectively.
The therapy utilizes a modified herpes virus to deliver two key genes, interleukin-2 and interleukin-12, into lung cells. These genes help suppress tumor growth by restoring their production in the body. The therapy has shown success in reducing tumor size and halting tumor growth in patients who have exhausted all other treatment options.
Despite some mild side effects like chills or vomiting, the therapy has shown no severe safety concerns. This positive outcome has led to the therapy receiving a “regenerative medicine advanced therapy designation” from the US FDA, expediting its approval process for patient access.
While the therapy targets tumors confined to the lungs, ongoing trials are exploring its combination with immunotherapies and chemotherapies to address tumors that have spread to other parts of the body. The company behind this breakthrough, Krystal Biotech, is also working on gene therapies for cystic fibrosis and alpha-1 antitrypsin deficiency.
In conclusion, the development of an inhalable gene therapy for lung cancer marks a significant advancement in the field of gene therapy. With promising results and expedited approval processes, this therapy offers hope for patients with advanced lung cancer and paves the way for future innovations in gene therapy treatments.

