Diabetes is a syndrome defined by high blood glucose levels caused by either reduction in number of insulin-producing cells (Type 1 diabetes), or the ability of our cells to respond to insulin in combination with declined numbers of insulin-producing cell number (Type 2 diabetes). Thus, a cure for diabetes should entail replacement of insulin-producing β-cells. There have been tremendous efforts throughout the years to generate β-cells from different sources, not only from embryonic stem cells, and adult stem or somatic cells, but also from non-β-cells residing in the pancreas. One such cell type is the amylase-producing pancreatic acinar cell. These cells are primarily responsible for producing and secreting enzymes that would help us digest of the food we eat. Reprogramming of acinar cells toward functional β-like cells would offer an abundant and autologous source of insulin-producing cells.
Description
Researchers at the University of Pittsburgh have discovered a compound which, when administered to diabetic mice or non-human primates (NHP) in therapeutic amounts, has the potential to normalize blood glucose by transforming pancreatic acinar cells into beta cells. These acinar-derived beta cells migrate into and embed themselves within the microenvironment of the islets of Langerhans, conferring distinct advantages beyond insulin production, including proximity to blood vessels that provide for increased efficiency of insulin secretion. The acinar-derived insulin-positive cells also express Glut2, suggesting that these cells share features with mature insulin-producing cells.
Applications
· β-cell replacement therapy for treating diabetes.
Advantages
· Converts native cells into beta cells, avoiding concerns over immunogenicity from foreign cells or tumorigenicity from regenerative therapies
· Normalizes blood glucose in diabetic mice and NHP within ten days
· Observations so far indicate that treatment over a short time may be sufficient, significantly reducing the risk of potential side effects
· As a repurposed drug, this compound has passed the phase (I) clinical trial; thus, the timeline for a transfer to a clinical trial for diabetes will be reduced
Invention Readiness
In vivo data
IP Status
https://patents.google.com/patent/US20200046700A1