Gene Alteration Provides Hope for Type 1 Diabetics

June 30, 2014
Adam Hochron

Early research at Columbia University could provide a breakthrough in the treatment of diabetes.

Early research at Columbia University could provide a breakthrough in the treatment of diabetes.

Working at the Naomi Berrie Diabetes Center scientists have shown that turning off a single gene can convert human gastrointestinal cells into insulin-producing cells. If further studies prove the theory it could mean with the right medication a person’s own cells could produce additional insulin.

Published in the journal Nature Communications the research looks promising according to senior author Domenico Accili, MD in a statement from the school. “People have been talking about turning one cell into another for a long time, but until now we hadn’t gotten to the point of creating a fully functional insulin-producing cell by the manipulation of a single target.”

The results of the study could mean that in the future patients with type 1 diabetes might be able to replace lost cells through reeducation of existing cells rather than needing the transplantation of new cells from an embryo or harvested stem cells.

With the work at Columbia the group continued what has been a two decade process to make insulin producing cells to combat the elimination of naturally made cells by a patient’s immune system. Accili’s work in the past had shown that mice could have their intestinal cells transformed to make insulin and his current work has shown its applicability to humans.

The gene needed to be turned off was located in the gut and is known as the FOXO1 gene. Working with postdoctoral fellow Ryotaro Bouchi they made a tissue model of the human intestine with human pluripotent stem cells. They then deactivated the gene and after seven days saw some cells releasing insulin, but only in response to glucose according to the statement.

Acilli said the work of the two studies shows good potential for future treatment. “By showing that human cells can respond in the same way as mouse cells, we have cleared a main hurdle and can now move forward to try to make this treatment a reality.”

Study co-author Rudolph L. Leibel, MD, the Christopher J. Murphy Memorial Professor of Diabetes Research, professor of pediatrics and medicine, and co-director of the Naomi Berrie Diabetes Center at Columbia said he was also encouraged by the results. “This work provides a new research tool for investigating the basic biology underlying the important relationships between the gut and insulin-producing cells, as wella s a clear indication of the potential clinical utility of stem cell-based approaches to diabetes

The next big step in the process will be finding a drug to inhibit FOXO1 in gastrointestinal cells, which Acilli said he is currently working on.