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!