FDA fast-tracks inhalable gene therapy for advanced lung cancer
The FDA fast-tracked an inhalable gene therapy for advanced lung cancer after an early 11-patient trial showed tumour shrinkage in three patients and disease stabilization in five. Larger studies involving about 250 patients are underway.
KB707, an inhalable gene therapy for lung cancer, has now been fast-tracked by the U.S. Food and Drug Administration after early clinical results suggested it may shrink tumours. In an early clinical trial involving 11 patients with advanced lung cancer, all of whom had exhausted other treatment options, tumours shrank in three patients and in five others, the cancer stopped growing.
Patients inhale the treatment as a fine mist through a nebulizer. Instead of directly attacking the tumour, the gene therapy delivers genetic material into lung cells, essentially turning them into tiny immune-boosting factories.
It uses a modified herpes virus altered so it cannot spread or cause disease as a delivery vehicle. That virus carries two important genes that produce proteins called interleukin-2 and interleukin-12. These proteins are natural immune messengers.
While some experienced side effects such as chills or vomiting, no major safety concerns were reported. The FDA granted it Regenerative Medicine Advanced Therapy designation, putting it on a faster review track if results continue to hold up in larger studies.
Right now, the treatment appears to work only when tumours are confined to the lungs. It does not target cancer that has spread elsewhere in the body. Larger trials involving about 250 patients are now underway, including studies combining the therapy with chemotherapy and immunotherapy.
The treatment was developed by Krystal Biotech, a company that previously pioneered a gene therapy applied directly to the skin. The therapy is not yet approved.