FDA approves cell therapy for solid tumours

Until now, the success of CAR-T or TCR-T cell therapies has been limited to blood cancers. Despite numerous clinical trials, their benefit in the treatment of solid tumours, which account for more than 90% of all cancers, remained unclear. This is now history with the accelerated approval of the TCR-T therapy afamitresgene autoleucel (ADP-A2M4, Tecelra) from UK-based Adaptimmune Ltd for the treatment of HLA-A*02-positive, metastatic or unresectable synovial sarcoma, which is directed against the MAGE-A4 antigen frequently expressed on tumours.

In February, the FDA granted accelerated approval to the cell therapy lifileucel from Iovance Biotherapeutics Inc as a second-line treatment for inoperable malignant melanoma. However, the tumour-infiltrating lymphocytes isolated from tumour cells floating in the blood and then multiplied are not genetically modified like Adaptimmune and are therefore – contrary to what Iovance claims – in no way similar to CAR-T or TCR-T cell therapies, except for the fact that they are autologous.

However, following pre-approval of the autologous therapy, which is based solely on clinical evidence of a 39% response rate and increased tumour infiltration, Adaptimmune must provide efficacy data in order to receive full approval. Crucial for the accelerated approval: it is the first new therapy for synovial sarcoma in ten years.

In April this year, Roche’s US subsidiary terminated a collaboration with Genentech Inc in 2021 to develop five MAGE- A4-based allogeneic TCR-T therapies based on induced pluripotent stem cells (hiIPSCs).

The list price for the one-off autologous therapy is USD 727,000. In the USA, there are around 400 patients per year who are eligible for treatment.