The US Food and Drug Administration has granted fast-track status to an innovative inhalable gene therapy for advanced lung cancer, following promising early trial results. Developed by Krystal Biotech, the treatment uses a modified virus to deliver immune-boosting genes directly to lung cells via inhalation. In initial tests, it shrank tumors in some patients and halted growth in others.
A novel gene therapy designed for inhalation has shown potential in treating lung cancer, prompting the US Food and Drug Administration to expedite its review process. The therapy, developed by Krystal Biotech, involves a harmless modified herpes virus that carries genes for interleukin-2 and interleukin-12 into lung cells. These proteins, naturally produced by the body to combat tumors, are often depleted in cancer patients, and the treatment aims to restore their levels locally in the lungs.
Since 2024, researchers led by Wen Wee Ma at the Cleveland Clinic in Ohio have tested the therapy in patients with advanced lung cancer who had no other options left. The treatment is administered as a nebulized mist, allowing direct delivery to the lungs, which addresses a key challenge in traditional oral or intravenous therapies that often fail to reach the site effectively.
At a recent meeting of the American Society of Clinical Oncology in Chicago, Ma reported encouraging outcomes from the trial involving 11 patients. "Very encouragingly, the hypothesis was proven – that there was actually shrinkage of the tumours in the lungs," Ma stated. The therapy reduced tumor sizes in three participants and prevented growth in five others. Side effects included chills and vomiting, but no serious safety issues emerged.
This week, the FDA awarded the therapy its regenerative medicine advanced therapy designation, aiming to speed up approval for patient access. However, it currently targets only tumors confined to the lungs. To expand its reach, Ma's team is now combining it with immunotherapies and chemotherapies in larger trials involving about 250 patients.
Krystal Biotech, known for its prior success with a topical gene therapy for the rare skin condition recessive dystrophic epidermolysis bullosa, is also developing similar inhalable treatments for cystic fibrosis and alpha-1 antitrypsin deficiency.
