Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy

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Grant Award Details

Grant Number:
TRAN1-14609
Investigator(s):
Name:
Institution:
Type:
PI

Disease Focus:
Award Value:
$6,054,165
Status:
Active

Grant Application Details

Application Title:

Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy

Public Abstract:
Translational Candidate

'Pseudoislets' derive from human islets, but compared to islets have superior survival, function, and diabetes reversal after transplantation.

Area of Impact

Pseudoislets could transform islet replacement strategies in diabetes by increasing the number and durable function of transplanted islet cells.

Mechanism of Action

Transplantation of replacement human islet cells is approved in type 1 diabetes and chronic pancreatitis. Sadly, this approach is limited by the scarcity of donor islets, and poor islet survival after transplantation. Pseudoislets - clusters of islet cells derived from human islets - could address this need. Compared to islets, pseudoislets are smaller, resilient, and interactive after transplantation with blood vessel cells, leading to improved islet cell survival and lasting function.

Unmet Medical Need

Loss of islet transplant function in subjects electing total pancreatecomy and islet autotransplantation (TPIAT) is an unmet medical need. Autologous pseudoislet transplantation could prolong islet graft survival and function in TPIAT subjects, extending insulin independence and euglycemia.

Project Objective

PreIND meeting on auto-pseudoislet transplantation

Major Proposed Activities

  • Develop manufacturing and regulatory processes to generate GMP human pseudoislets for autotransplantation in subjects at risk for diabetes mellitus.
  • Assess the stability, function, and the capacity for diabetes reversal by GMP human pseudoislets in transplantation studies of diabetic mice.
  • File a pre-pre-IND for human pseudoislet transplantation to the U.S. FDA
Statement of Benefit to California:
Multiple benefits to California and its citizens would ensue from successful conclusion of innovative studies proposed here. This includes (1) improvements in patient care, especially for those requiring islet transplantation, (2) emergence of pancreatic islet autotransplantation programs that would foster increased consultation and use of California health care systems by citizens and outside clients, (3) enhanced support for academic training and research in pancreatic islet transplantation.