Oncology News Central (ONC)FDA Approves First Engineered Cell Therapy for a Solid Tumor

Novel T-cell therapy offers new treatment avenue for rare soft tissue cancer, potentially paving the way for cell-based approaches in other solid tumors

The US Food and Drug Administration (FDA) has granted accelerated approval to afamitresgene autoleucel (afami-cel, Tecelra) for the treatment of metastatic synovial sarcoma in adults. This approval marks the first engineered cell therapy for a solid tumor, representing a significant advancement in cancer treatment. Afami-cel utilizes a patient’s own immune cells, which are genetically modified to target cancer cells, offering a new option for patients with this rare soft tissue cancer.

Key Points:

• Afami-cel is approved for adults with unresectable or metastatic synovial sarcoma who have received chemotherapy.
• Patients must be HLA-A02:01P, HLA-A02:02P, HLA-A02:03P, or HLA-A02:06P positive.
• Tumors must express the melanoma-associated antigen A4, determined by FDA-authorized diagnostic devices.
• The treatment involves a one-time infusion of genetically re-engineered immune cells.
• In the SPEARHEAD-1 trial supporting FDA approval:
  • Overall response rate (ORR) was 43.2% among 44 participants
  • Complete response rate was 4.5%
  • Median duration of response (DoR) was 6 months
  • 39% of responders had a DoR of 12 months or longer
• Efficacy is comparable to current best chemotherapy options, according to expert opinion.
• Important safety information includes a boxed warning for cytokine release syndrome (CRS):
  • CRS occurred in 75% of patients in the clinical trial
  • CRS may be severe or life-threatening
  • Immediate evaluation and supportive care are necessary at first signs of CRS
• Common CRS symptoms include fever, tachycardia, hypotension, nausea/vomiting, and headache.
• Afami-cel should not be used in adults who are heterozygous or homozygous for HLA-A*02:05P.
• Adaptimmune plans to establish 6-10 authorized treatment centers this year, expanding to 30 within two years.
• Synovial sarcoma predominantly affects young adults, with a current five-year survival rate as low as 36%.
• For patients with metastatic disease at diagnosis, the five-year survival rate can be as low as 20%.
• This approval provides the first new therapy option for synovial sarcoma in more than a decade.

In 2021, there were an estimated 167,276 people living with soft tissue cancer in the United States. (National Cancer Institute)

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