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Cell-Replacement Therapy After Total Pancreatectomy Gets Orphan Drug Status
The FDA has granted orphan drug status for a process involving autologous insulin-producing (AIP) cells as cell-replacement therapy in patients with severe hypoglycemia-prone diabetes from total pancreatectomy (TP), as a result of chronic pancreatitis.
After TP, 100% of patients go on to develop diabetes. This leads to immediate and lifelong insulin-dependence, with the loss of both endogenous insulin secretion and glucagon. Glycemic control after TP is particularly difficult with standard therapy because of total insulin dependence and loss of glucagon-dependent counter-regulation. Patients who have this type of diabetes suffer from both severe hyperglycemic and hypoglycemic episodes.
The proprietary transdifferentiation (cell reprogramming) process, designed by Oregenesis, involves converting one adult tissue or cell into another type of cell. The novel therapy employs this process and the company’s point of care (POCare) liver expansion technology to transform liver cells into AIP cells.
First, cells are obtained from a small sample of the patient’s liver cells, then expanded in a liver-cell bank based on specific POCare cell-expansion capabilities. When required, the cells can be converted into functional glucose-regulated AIP cells through the transdifferentiation process, and returned to the patient’s liver through transfusion.
It is hoped that the therapy will provide a “practical cure” for various forms of insulin-dependent diabetes, enabling long-term insulin independence without any need for concomitant immunosuppressive therapy.
Source: SeekingAlpha, June 17, 2019