One-step diabetes cure

We seem to be in the midst of a boom era for innovative Type 1 diabetes treatments (for mice at least).

Previously we had commented on Doug Melton’s in vivo reprogramming of adult pancreatic cells into insulin-producing beta cells using a three gene approach.

Now an international group of scientists have been able to induce beta cell formation in vivo with transfer of a single gene (Pax4).  The new method stimulated bystander cells in the pancreas (alpha cells, delta cells, PP cells) to develop into insulin-producing beta cells.

Why is this important?

Lack of beta cells is the key problem in Type 1 diabetes – when the cells are missing the body cannot regulate blood sugar which in turn creates a host of serious complications.

Type 1 diabetics are forced to frequently measure their blood glucose levels and self-inject insulin to avoid dire health consequences.  Beta cell transfer from cadavers (Edmonton Protocol) allows diabetics to escape from the rigors of disease management for a year or more, but they need to take powerful immunosupprressive drugs instead.  In addition, there is a serious shortage of donor cells for transplant.

The ideal approach would seem to be to create new patient-specific beta cells inside the body which would restore normalcy.  The current method brings that promise closer than before.  However, there are some important caveats we should bear in mind:

• these finding are all from a mouse system – humans and mice are not the same and there is no guarantee that the findings will translate
• the gene transfer was applied to embryos – it is unknown whether the same approach would work with more mature subjects
• gene transfer has been a major problem in the past

Having said that, our understanding of diabetes and the biology of the beta cells has never been greater.  Stay tuned for more exciting news!