EvidenceSarcomas (soft tissue, bone, GIST)

Afami-cel showed that T cells can be redirected against an intracellular cancer antigen, something CAR-T cannot do, and produced meaningful responses in a rare sarcoma with few options. It opens a path to TCR-T against other shared antigens and neoantigens. Restriction to one HLA type and one antigen, plus complex manufacturing, means it will help a small, defined group.

Patients with advanced synovial sarcoma, a rare cancer of young adults with few effective drugs, now have an approved cell therapy that produces responses lasting about a year in a substantial minority, if their tissue type and tumour antigen match. It proves that engineered T cells can work against a solid tumour when a good target is present, which had been elusive. It is not a cure for most, requires specialised centres, and only a minority of patients are eligible.

This trial validated the radioenhancer mode of action and supported the 2019 European CE mark for NBTXR3 (Hensify) in soft-tissue sarcoma. Whether more pathological complete responses translate into fewer recurrences or longer survival was not shown here; the 2022 follow-up paper reported more R0 resections and no harm to quality of life.

Interval-compressed VDC/IE is the standard first-line chemotherapy for localised Ewing sarcoma in North America; Euro Ewing 2012 later adopted it in Europe.