by in St. Louis has demonstrated the effectiveness of T cell immunotherapy in treating certain patients with rare cancers affecting the soft tissues of the body. Published in The Lancet, the study focused on the treatment of synovial sarcoma and myxoid round cell liposarcoma (MRCLS), two uncommon forms of sarcoma that have limited treatment options and poor five-year survival rates when they have spread.
Dr. Brian A. Van Tine, the senior author of the study and a professor of medicine at Washington University, highlighted the urgent need for more effective therapies for these rare sarcomas, noting that standard immunotherapies have shown limited success. The T cell immunotherapy used in the trial involved genetically modifying patients’ T cells to target and destroy cancer cells. While some patients exhibited significant and lasting responses to the treatment, others experienced temporary responses or no response at all, emphasizing the need to refine and broaden the therapy’s effectiveness in more patients.
The investigational immunotherapy, known as afamitresgene autoleucel (afami-cel), entailed the collection of a patient’s T cells, which were then engineered in the laboratory using a viral vector to introduce a specific protein onto their surface. This protein, a T cell receptor, enabled the modified T cells to identify and eliminate cancer cells, specifically targeting a protein called MAGE-A4 found on the surface of most sarcomas.
Developed by the biopharmaceutical company Adaptimmune, the immunotherapy was the focus of an international clinical trial funded by the company. Siteman Cancer Center, a leading sarcoma referral center in the United States, was a key participant in the trial, which involved 52 patients with synovial sarcoma and MRCLS across multiple medical centers in North America and Europe.
Of the patients treated, around 36% showed positive responses to the therapy, with tumors either shrinking or disappearing. The treatment demonstrated greater efficacy in patients with synovial sarcoma compared to MRCLS, with the duration of tumor response averaging over 11 months for synovial sarcoma and over four months for MRCLS. While most patients experienced cancer recurrence after a period of remission, a small number remained cancer-free after their tumors vanished following treatment.
On average, patients in the trial survived just over 15 months, with a one-year survival rate of 60% across all participants. Patients with synovial sarcoma who achieved a complete response to the initial therapy had survival rates of 90% at one year and 70% at two years. The most common side effects of treatment involved blood cell count abnormalities and cytokine release syndrome, a common inflammatory reaction to T cell therapies, with treatment-related deaths attributed to disease progression rather than the investigational therapy.
Dr. Van Tine noted that some patients continued to exhibit sustained responses to the treatment, underscoring the potential of engineered T cells to eradicate cancer cells and establish long-lasting remission. Ongoing research aims to identify factors contributing to favorable treatment outcomes, with the goal of expanding the therapy’s benefits to more patients in the future.
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1. Source: Coherent Market Insights, Public sources, Desk research
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