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

Hope for diabetics
Hope for diabetics

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!

John McCulloch

John provides assistance to life sciences entrepreneurs in business strategy, management, intellectual property, financing and licensing. See more…