Sarcomas are cancers that grow in connective tissue -- cells that connect or support other kinds of tissue in your body. There are more than 50 types of sarcoma, they can be grouped into two main kinds: soft tissue sarcoma and bone sarcoma, osteosarcoma. About 12,000 cases of soft tissue sarcoma and 3,000 cases of bone sarcomas were seen in the U.S. in 2014.
Osteosarcoma, the most common type of bone cancer, affects predominately children and adolescents. There is a huge unmet need for treatment, current treatment usually involves amputation of a limb and heavy chemotherapy, and patients have a poor prognosis.
The first ever CAR T Cell Phase I/II clinical trial in Sarcoma was completed. Aurora’s CAR T Cell AU-101 met its safety endpoint, with no severe adverse events with 19 Sarcoma patients (16 osteosarcomas, 1 Ewing sarcoma, 1 primitive neuro ectodermal tumor, and 1 desmoplastic small round-cell tumor). There were strong signs of tumor killing and survival benefit. In one Partial Response patient, 90% of the tumor biopsied after CAR T Cell therapy was necrotic. There were 3 patients who outlived the median survival historic control, alive around three years.
PET images before (top arrow) and 6 weeks after (bottom arrow) AU101 infusion.
A phase II trial with AU101 combined with lymphodepletion in Osteosarcoma patients is currently ongoing. Experimental findings indicate that lymphodepletion prior to adoptive transfer of tumor-specific T lymphocytes plays a key role in enhancing treatment efficacy by eliminating regulatory T cells and competing elements of the immune system.