FDA Approves New Gene Therapy for Rare Soft Tissue Cancer
A new, innovative gene therapy is now available for patients with metastatic synovial sarcoma, a rare cancer that forms in the body's soft tissues, such as muscles and ligaments. The U.S. Food and Drug Administration approved Tecelra (afamitresgene autoleucel), making it the first engineered T-cell receptor (TCR) therapy approved for a solid tumor.
Synovial sarcoma affects approximately 1,000 people in the U.S. annually, primarily targeting adult males in their 30s or younger. The disease typically manifests in the soft tissues of the extremities but can spread to other parts of the body. Traditional treatments include surgery, radiotherapy and chemotherapy, but these methods often fall short, particularly for metastatic cases.
Tecelra, an autologous T-cell immunotherapy, uses a patient's own T cells, which are genetically modified to target the MAGE-A4 antigen in synovial sarcoma cells. Moffitt Cancer Center participated in the clinical trials, and the results have been promising. Among the 44 clinical trial patients who received TCR therapy, the overall response rate was 43.2%, with a median duration of response of six months.
“Tecelra is a new therapeutic strategy that uses the patient's immune T cells to recognize and fight against the cancer, administered in only one infusion. This is significantly different from the cytotoxic systemic therapeutic options used to treat this rare tumor for such a long time. This therapy represents a milestone in the development of cell therapies for solid tumors in general and so much needed for patients with synovial sarcoma,” said Mihaela Druta, MD, a medical oncologist at Moffitt specializing in soft tissue malignancies and one of the trial’s principal investigators.
While this accelerated FDA approval offers patients earlier access to this therapy, ongoing clinical trials are necessary to verify its clinical benefits.
