FDA Fast-tracks First Inhalable Gene Therapy for Cancer Treatment

An innovative inhaled gene therapy targeting lung cancer is rapidly advancing toward potential approval following encouraging results from clinical trials.

Dr. Wen Wee Ma at the Cleveland Clinic highlighted the findings at a recent American Society of Clinical Oncology conference in Chicago, stating, “Very encouragingly, this proves our hypothesis that the lung tumor actually shrunk.”

This groundbreaking treatment employs a virus to introduce immune-boosting genes into lung cells, enhancing their natural ability to combat tumors. Unlike traditional gene therapies, which often replace defective genes, this method focuses on modifying existing lung cells.

The unique inhalation delivery method represents a significant advancement in cancer treatment. “This is a completely different approach to anti-cancer treatment,” said Ma. Directly targeting the lungs enhances the efficiency and effectiveness of the treatment, particularly since lung cancer is notoriously difficult to treat with standard oral or intravenous therapies.

The therapy utilizes a harmless, modified herpes virus to inject two critical genes into lung cells: interleukin-2 and interleukin-12. These proteins, naturally produced by the body, help inhibit tumor growth. Unfortunately, tumors often diminish their effectiveness, necessitating the need for gene therapy to restore their production.

Since 2024, clinical trials have been ongoing with patients suffering from advanced lung cancer who have exhausted all other treatment options. The therapy is administered via a fine mist inhaled directly into the lungs.

At the oncology conference, Ma reported that the gene therapy has successfully reduced lung tumor sizes in three out of eleven trial participants, while also halting growth in another five. Although some patients reported side effects, such as chills and vomiting, no severe safety issues were noted.

Based on these promising outcomes, the U.S. Food and Drug Administration recently granted “Regenerative Medicine Advanced Therapy Designation” to the gene therapy, facilitating expedited approval processes for patient access.

However, it is important to note that this gene therapy is specifically designed for lung tumors and does not address tumors that have metastasized to other body parts. To expand its efficacy, Ma and his team are exploring combinations with immunotherapy and chemotherapy in a trial involving approximately 250 patients.

Crystal Biotech, the developer of this gene therapy, previously introduced the first FDA-approved gene therapy targeting the skin, using a similar modified herpes virus to treat patients with recessive dystrophic epidermolysis bullosa, a rare skin condition. The company is also developing inhaled gene therapies for cystic fibrosis and alpha-1 antitrypsin deficiency, both inherited lung diseases.