Clinical staging and oversight

Regulation and oversight should be proportional to risk and intended use. This guidance focuses on autologous cell therapy where the adult stem cell source is functionally homologous with the therapeutic target.

Minimal culture expansion is defined by incubation period and passage count, with clinical staging used to balance patient safety with treatment availability.

  • Focus on autologous adult stem cell therapy.
  • Minimal culture expansion is described as no more than 60 days of incubation and no more than 10 passages after colony formation.
  • Safety tracking includes transplant safety, such as tumorigenesis, transplant-site monitoring, and new disease monitoring.
  • Safety tracking also includes treatment-specific risks based on transplant location, disease treated, and cell type.
  • Clinical staging categories include PICL, EICL, LICL, ECCL, LCCL, and CG.
  • Patients treated with clinical-grade cell lines are entered into the ICMS Re-implantation Registry.

Excluded or not-ready cell types

IPS cells, embryonic or cord stem cells, and manipulated-gene autologous adult stem cells are not included in this clinical translation pathway without current scientific, clinical, and regulatory review.

Clinical staging labels

Clinical staging categories include Pre-Investigational Cell Line, Early Investigational Cell Line, Late Investigational Cell Line, Early Clinical Cell Line, Late Clinical Cell Line, and Clinical Grade.

  • PICL: no animal data is available and the line should not be used in humans until animal data is available.
  • EICL: animal models show efficacy and safety but no human data exists; early human studies involve 5-10 patients with at least six months of follow-up.
  • LICL: larger human testing, typically 20-50 patients with at least six months of follow-up.
  • ECCL: early clinical treatments in 50-200 patients with follow-up and no significant complications.
  • LCCL: clinical treatments in 100-300 patients with follow-up and no significant complications.
  • CG: the framework's Clinical Grade category follows completion of its staging phases and includes registry tracking. This designation is not regulatory approval or permission for unrestricted use.