Teratoma formation assay in immunocompromised mice to evaluate risk of pluripotent stem cell-derived cell therapy products

STP 2026 -- Human pluripotent stem cell (hPSC) derived products show promise in regenerative medicine for treating and modeling a variety of diseases. Pluripotent stem cells have the potential to self-renew indefinitely and can differentiate into tissues from all three germ layers. The hPSCs can be derived from human embryonic stem cells, or now more commonly, via reprogramming human somatic cells into induced pluripotent stem cells (iPSCs). However, safety concerns exist from residual undifferentiated cells forming teratomas, tumors comprised of tissues from three different germ layers. The International Stem Cell Initiative recommends that all new hPSC lines be assessed for pluripotency. To assess tumorigenicity risk of hPSC-derived therapies, an in vivo teratoma formation assay was developed.