The gene therapy is administered as a mist that is inhaled
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A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked toward potential approval after promising clinical trial results.
“Very encouragingly, the hypothesis was proven — that there was indeed shrinkage of the tumors in the lungs,” Wen Wee Ma of the Cleveland Clinic in Ohio told a recent meeting of the American Society of Clinical Oncology in Chicago.
The treatment is novel because it uses a virus to carry immune-enhancing genes into lung cells, making them better at fighting tumors on their own. Gene therapy usually involves replacing defective copies of important genes.
Another unusual aspect is that it is inhaled rather than swallowed or injected. “It’s such a different way to provide anti-cancer treatment,” Ma said. The advantage of this delivery method is that it gets the therapy directly into the lungs. One reason lung cancer is the deadliest form of the disease is that treatments given orally or intravenously struggle to reach the lungs.
The new therapy contains a herpes virus that has been modified to make it harmless and unable to spread to other people. The virus has the task of dragging two genes, one that codes for the protein interleukin-2 and the other that codes for interleukin-12, into lung cells. These are produced naturally in the body and help to suppress tumor growth. However, tumors often fight back and clear them, so the gene therapy is designed to restore production.
Since 2024, Ma and his colleagues have been testing the gene therapy in people with advanced lung cancer who have exhausted all other treatment options. To administer it, a liquid containing the gene therapy is atomized, meaning it is turned into a fine mist that people inhale directly into their lungs from a device.
At the oncology meeting, Ma announced that the gene therapy had reduced the size of lung tumors in three of 11 people, and prevented them from growing larger in another five people. Some patients experienced side effects such as chills or vomiting, but no serious safety concerns were identified.
Based on these positive results, the gene therapy received “regenerative medicine advanced therapy designation” from the US Food and Drug Administration this week. This means that the agency will seek to expedite approval so that patients can access it as soon as possible.
A disadvantage of gene therapy is that it only targets tumors that are confined to the lungs, not when they have spread to other parts of the body. To solve this, Ma and his colleagues are now testing it in combination with immunotherapy and chemotherapy, in trials that will involve around 250 patients.
Krystal Biotech, the company that developed the gene therapy, previously created the first approved gene therapy that is rubbed into the skin. This uses the same modified herpes virus to carry collagen genes into the skin of people with a rare skin disease called recessive dystrophic epidermolysis bullosa, helping to repair their skin.
The company is also working on inhalable gene therapies for cystic fibrosis and a genetic lung condition called alpha-1 antitrypsin deficiency.
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