Orphan drug status to autologous insulin producing cells

August 6, 2019 0 By FM

The US FDA has granted Orphan Drug designation for autologous insulin producing (AIP) cells as a cell replacement therapy for the treatment of severe hypoglycaemia-prone diabetes resulting from total pancreatectomy (TP) due to chronic pancreatitis (CP), Orgenesis Inc said.

The company’s patented transdifferentiation (or cell reprograming) process involves the conversion of one adult tissue or cell into another type of cell, with its distinct phenotype and function.

The company has developed a novel therapy utilising its transdifferentiation process and its Point of Care (POCare) liver expansion technology to transform liver cells into AIP cells. The cells are first derived from a small sample of the patient’s liver cells and expanded in a liver cell bank based on POCare cell expansion capabilities.

At the appropriate time, the cells may be converted into functional glucose-regulated AIP cells and returned to the patient’s liver via a transfusion.

The incidence of diabetes following TP is 100 percent, resulting in immediate and lifelong insulin-dependence with the loss of both endogenous insulin secretion and that of the counter-regulatory hormone, glucagon. Glycaemic control after TP is difficult with conventional insulin therapy due to complete insulin dependence and loss of glucagon-dependent counter-regulation, the company said.