The U.S. Food and Drug Administration (FDA) has granted fast track designation to KB707 from Krystal Biotech, the first cancer gene therapy administered by inhalation. The drug is delivered to the lungs as an aerosol using a nebulizer.
The technology is based on a modified, safe virus that carries the IL-2 and IL-12 genes into cells. These stimulate a local immune response: cells begin producing signals that activate the body’s defenses against the tumor. At the same time, the therapy does not destroy the cancer directly, but changes the environment around it.
The advantage of the approach is that the drug is delivered directly to the lungs, bypassing the bloodstream, which may reduce toxicity. In addition, the therapy can be repeated. This matters because many drugs do not reach lung tissue well.
In an early study involving 11 patients, three showed tumor shrinkage and five had stable disease. The development is still at an early stage and has not yet been approved, but the fast track designation points to its potential significance.
