A therapy for patients to inhale has been found to shrink lung tumors by inserting immune-boosting genes into surrounding cells, receiving the FDA’s “fast track” to approval.
A pioneering inhalable gene therapy for lung cancer that genetically modifies lung cells has been fast-tracked for potential approval following promising clinical trial results.
As explained by , the treatment is innovative because it uses a virus to transport genes that strengthen the immune system to lung cells, making them more effective in fighting tumors on their own. Gene therapy typically involves replacing defective copies of essential genes.
“Very encouragingly, the hypothesis was proven – there was a reduction in tumors in the lungs”, praised the leader of the investigation, Wen Wee Mafrom the Cleveland Clinic, in Ohio, at one from the American Society of Clinical Oncology, in Chicago.
Another unusual aspect is the fact that it is inhaled rather than ingested or injected. The advantage of this mode of administration is that it delivers the therapy directly to the lungs.
One of the reasons why lung cancer is the deadliest form of the disease is precisely the fact that treatments administered orally or intravenously have difficulty reaching the lungs.
How does therapy work?
The new therapy contains a herpes virus which has been modified to become harmless and unable to spread to other people.
The virus is responsible for transporting two genes, one that encodes the protein interleukin-2 and the other that encodes interleukin-12, to lung cells. These substances are produced naturally in the body and help to suppress tumor growth. However, tumors often react and deplete them, so gene therapy has been designed to restore their production.
During administration, a liquid containing gene therapy is nebulized, that is, converted into a fine mist that people inhale directly into their lungs from a device.
The team announced that gene therapy reduced the size of lung tumors in three of 11 people and stopped them from getting bigger in another five. Some patients experienced side effects such as chills or vomiting, but no serious safety concerns were identified.
Based on these positive results, gene therapy this week received the designation of “advanced regenerative medicine therapy” of the Food and Drug Administration (FDA). In other words, the agency will seek to speed up its approval so that patients can have access quickly.
One disadvantage of gene therapy is that it only targets tumors confined to the lungs, not when they have spread to other parts of the body.
