Regulatory Challenges in Manufacturing of Pancreatic Islets

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

At the present time, transplantation of pancreatic islet cells is considered an experimental therapy for a selected cohort of patients with type 1 diabetes, and is conducted under an Investigational New Drug (IND) application. Encouraging results of the Edmonton Protocol published in the year 2000 sparked a renewed interest in clinical transplantation of allogeneic islets, triggering a large number of IND applications for phase I clinical trials. Promising results reported by a number of centers since then prompted the Food and Drug Administration (FDA) to consider the possibility of licensing allogeneic islets as a therapeutic treatment for patients with type 1 diabetes. However, prior to licensure, issues such as safety, purity, efficacy, and potency of the islet product must be addressed. This is complicated by the intricate nature of pancreatic islets and limited characterization prior to transplantation. In this context, control of the manufacturing process plays a critical role in the definition of the final product. Despite significant progress made in standardization of the donor organ preservation methods, reagents used, and characterization assays performed to qualify an islet cell product, control of the isolation process remains a challenge. Within the scope of the FDA regulations, islet cells meet the definition of a biologic product, somatic cell therapy, and a drug. In addition, AABB standards that address cellular therapy products apply to manufacturing facilities accredited by this organization. Control of the source material, isolation process, and final product are critical issues that must be addressed in the context of FDA and other relevant regulations applicable to islet cell products.

Original languageEnglish
Pages (from-to)424-426
Number of pages3
JournalTransplantation Proceedings
Volume40
Issue number2
DOIs
StatePublished - Mar 1 2008

Fingerprint

Islets of Langerhans
Investigational New Drug Application
United States Food and Drug Administration
Licensure
Type 1 Diabetes Mellitus
Transplantation
Organ Preservation
Islets of Langerhans Transplantation
Clinical Trials, Phase I
Investigational Therapies
Drug and Narcotic Control
Homologous Transplantation
Cell- and Tissue-Based Therapy
Biological Products
Therapeutics
Tissue Donors
Organizations
Safety
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Surgery
  • Transplantation

Cite this

Regulatory Challenges in Manufacturing of Pancreatic Islets. / Linetsky, Elina; Ricordi, Camillo.

In: Transplantation Proceedings, Vol. 40, No. 2, 01.03.2008, p. 424-426.

Research output: Contribution to journalArticle

@article{5d1e70a8daa74927aeeaf5743b1ac702,
title = "Regulatory Challenges in Manufacturing of Pancreatic Islets",
abstract = "At the present time, transplantation of pancreatic islet cells is considered an experimental therapy for a selected cohort of patients with type 1 diabetes, and is conducted under an Investigational New Drug (IND) application. Encouraging results of the Edmonton Protocol published in the year 2000 sparked a renewed interest in clinical transplantation of allogeneic islets, triggering a large number of IND applications for phase I clinical trials. Promising results reported by a number of centers since then prompted the Food and Drug Administration (FDA) to consider the possibility of licensing allogeneic islets as a therapeutic treatment for patients with type 1 diabetes. However, prior to licensure, issues such as safety, purity, efficacy, and potency of the islet product must be addressed. This is complicated by the intricate nature of pancreatic islets and limited characterization prior to transplantation. In this context, control of the manufacturing process plays a critical role in the definition of the final product. Despite significant progress made in standardization of the donor organ preservation methods, reagents used, and characterization assays performed to qualify an islet cell product, control of the isolation process remains a challenge. Within the scope of the FDA regulations, islet cells meet the definition of a biologic product, somatic cell therapy, and a drug. In addition, AABB standards that address cellular therapy products apply to manufacturing facilities accredited by this organization. Control of the source material, isolation process, and final product are critical issues that must be addressed in the context of FDA and other relevant regulations applicable to islet cell products.",
author = "Elina Linetsky and Camillo Ricordi",
year = "2008",
month = "3",
day = "1",
doi = "10.1016/j.transproceed.2008.01.027",
language = "English",
volume = "40",
pages = "424--426",
journal = "Transplantation Proceedings",
issn = "0041-1345",
publisher = "Elsevier USA",
number = "2",

}

TY - JOUR

T1 - Regulatory Challenges in Manufacturing of Pancreatic Islets

AU - Linetsky, Elina

AU - Ricordi, Camillo

PY - 2008/3/1

Y1 - 2008/3/1

N2 - At the present time, transplantation of pancreatic islet cells is considered an experimental therapy for a selected cohort of patients with type 1 diabetes, and is conducted under an Investigational New Drug (IND) application. Encouraging results of the Edmonton Protocol published in the year 2000 sparked a renewed interest in clinical transplantation of allogeneic islets, triggering a large number of IND applications for phase I clinical trials. Promising results reported by a number of centers since then prompted the Food and Drug Administration (FDA) to consider the possibility of licensing allogeneic islets as a therapeutic treatment for patients with type 1 diabetes. However, prior to licensure, issues such as safety, purity, efficacy, and potency of the islet product must be addressed. This is complicated by the intricate nature of pancreatic islets and limited characterization prior to transplantation. In this context, control of the manufacturing process plays a critical role in the definition of the final product. Despite significant progress made in standardization of the donor organ preservation methods, reagents used, and characterization assays performed to qualify an islet cell product, control of the isolation process remains a challenge. Within the scope of the FDA regulations, islet cells meet the definition of a biologic product, somatic cell therapy, and a drug. In addition, AABB standards that address cellular therapy products apply to manufacturing facilities accredited by this organization. Control of the source material, isolation process, and final product are critical issues that must be addressed in the context of FDA and other relevant regulations applicable to islet cell products.

AB - At the present time, transplantation of pancreatic islet cells is considered an experimental therapy for a selected cohort of patients with type 1 diabetes, and is conducted under an Investigational New Drug (IND) application. Encouraging results of the Edmonton Protocol published in the year 2000 sparked a renewed interest in clinical transplantation of allogeneic islets, triggering a large number of IND applications for phase I clinical trials. Promising results reported by a number of centers since then prompted the Food and Drug Administration (FDA) to consider the possibility of licensing allogeneic islets as a therapeutic treatment for patients with type 1 diabetes. However, prior to licensure, issues such as safety, purity, efficacy, and potency of the islet product must be addressed. This is complicated by the intricate nature of pancreatic islets and limited characterization prior to transplantation. In this context, control of the manufacturing process plays a critical role in the definition of the final product. Despite significant progress made in standardization of the donor organ preservation methods, reagents used, and characterization assays performed to qualify an islet cell product, control of the isolation process remains a challenge. Within the scope of the FDA regulations, islet cells meet the definition of a biologic product, somatic cell therapy, and a drug. In addition, AABB standards that address cellular therapy products apply to manufacturing facilities accredited by this organization. Control of the source material, isolation process, and final product are critical issues that must be addressed in the context of FDA and other relevant regulations applicable to islet cell products.

UR - http://www.scopus.com/inward/record.url?scp=40949147459&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=40949147459&partnerID=8YFLogxK

U2 - 10.1016/j.transproceed.2008.01.027

DO - 10.1016/j.transproceed.2008.01.027

M3 - Article

C2 - 18374089

AN - SCOPUS:40949147459

VL - 40

SP - 424

EP - 426

JO - Transplantation Proceedings

JF - Transplantation Proceedings

SN - 0041-1345

IS - 2

ER -