A Promising Step Towards Insulin Independence in Type 1 Diabetes
Key takeaways:
- Initial results of Sana Biotechnology’s new gene-editing technique demonstrate that transplanted islet cells successfully produced insulin in one individual with type 1 diabetes – without the need for immunosuppression or anti-rejection drugs.
- This very early research could be a potential breakthrough as the need for immunosuppressants, which come with serious health risks, is a big barrier to its widespread success.
- While these early clinical results are exciting, further research is essential to fully understand the long-term safety, efficacy, and broader impact of this procedure as progress continues toward the goal of a cure for type 1 diabetes.
Islet cell transplantation has long been considered a promising approach to treating (and potentially curing) type 1 diabetes, but there are several major obstacles, one being the need for lifelong immunosuppressant medications.
However, a recent breakthrough from Sana Biotechnology points to a potential path to restore insulin production without the need for immunosuppressive drugs.
What is islet cell transplantation?
Current research in islet cell transplantation seeks to bring us one step closer to a type 1 diabetes cure. The aim of this procedure is to restore the body's natural ability to produce insulin by transferring healthy islet cells from a donor pancreas to an individual with type 1 diabetes.
Islet cell transplantation has been shown to be successful – as is the case for Lantidra, the first FDA-approved cell therapy (though not currently available) approved in 2023. However, there are multiple barriers to the widespread use of the procedure:
- One is the need for lifelong anti-rejection drugs to prevent the body from attacking or rejecting the transplanted islet cells. The downside of these medications is they increase the risk of serious potential complications including infections, kidney damage, and cancer.
- Another is that the procedure requires islet cells from deceased organ donors, which are in very limited supply. Not all donor islet cells are viable either; insulin-producing cells from multiple donors are sometimes required for just one procedure.
- It’s also yet to be determined the best location for the transplanted cells. The current approach infuses cells into the liver, which is slightly more invasive, while the Sana trial injected the cells intramuscularly.
One solution to the limited supply of islet cells is using modified stem cells designed to produce insulin instead. This transplant method is currently being tested by Vertex in their VX-880 trial. The therapy has shown great promise, with almost a dozen participants now producing their own insulin, eliminating the need for insulin injections. People treated with VX-880 also experienced significant improvements in A1C and time in range.
However, the investigational VX-880 therapy also requires immunosuppressive therapy. For this reason, Sana’s recent achievement in enabling transplanted islet cells to produce insulin in a person with type 1 diabetes without immunosuppression marks a potential major milestone in the field.
How does it work?
The new process from Sana involves modifying donor-derived islet cells so that they can evade immune detection. That is, transplanted cells won’t be recognized by the body’s immune cells, avoiding destruction and enabling ongoing insulin production.
Cells were engineered using a genetic editing process called CRISPR-Cas9, a technique the company has dubbed hypoimmune technology. Initial results from an early clinical trial with modified cells implanted into the forearm muscles of a person with type 1 diabetes were promising. After 28 days, researchers observed the individual was successfully producing their own insulin – without the need for anti-rejection medications.
What does this mean for people with type 1 diabetes?
While these clinical results are very early and only represent one individual’s response to this innovative potential therapy, Sana’s achievement symbolizes a significant step forward in the field of islet cell transplantation.
Similar to Lantidra, Sana’s process requires cells from deceased donors, which aren’t widely available. Sana has said the research from their current study is meant to eventually be combined with a separate stem cell-derived pancreatic islet cell program to treat type 1 diabetes. The goal of this program, similar to Vertex’s newer VX-264 trial, is to vastly increase the available supply of insulin-producing cells without the need for immunosuppression medicines. More research is needed to understand the effects of hypoimmune technology on additional individuals and to determine its potential as a cure for people with type 1 diabetes.
Learn more about emerging treatments for type 1 diabetes here: