At the invitation and request of the U.S. Food & Drug Administration (FDA), Klearchos Papas, Ph.D., presented on “Assessment of Islet Quality Pre-Transplant” at the advisory committee meeting of the Center for Biologics Evaluation and Research (CBER), Office of Tissues and Advanced Therapies (OTAT), and Cellular, Tissue, and Gene Therapies (CTGT) on April 15, 2021.
Dr. Papas addressed the goals for pre-transplant islet quality assessment, such as; Is it safe to Transplant?, What is the Purity?, What is the “potency” or “dose”? E.g. Number of Viable, Functional βCells /Kg BW recipient, and Can we predict transplantation outcome using a set of real-time
assays?
Pancreatic islets have a complex, “glomerular-like” network of blood vessels with a high capacity for exchange and are necessary for islet function such as nutrient sensing and hormone dispersal.
Islets are assessed for transplantation for safety, identity, viability and potency.
Dr. Papas concludes that “while great progress has been made in the field:
- There is a need to further develop and refine real-time predictive potency tests for clinical islet allotransplantation.
- Islet nuclei counts and DNA measurements may further improve islet dosing especially when combined with measurements or β-cell (and α-cell) fraction.
- Measurements of islet preparation purity should be further refined and the relationship between islet purity and transplant outcome should be further explored.
- Viability and potency assays based on mitochondrial function (i.e. Oxygen Consumption Rate) appear to be useful and should be further explored.
- Attempts to correlate to clinical outcome should take into account viable (and functional) β-cell dose”.
The meeting was held with the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER), Office of Tissues and Advanced Therapies (OTAT), Cellular, Tissue and Gene Therapies (CTGT) Advisory Committee Meeting April 15, 2021.
For additional information, the meeting agenda and slide deck are linked below.
https://www.fda.gov/media/147599/download (Papas slide deck)
https://www.fda.gov/media/147591/download (meeting agenda)