Stem Cells: A Glimpse at a Possible Cure for Type 1 Diabetes

Living with type 1 diabetes (T1D) requires constant attention and relentless efforts to manage blood sugar levels, administer insulin multiple times a day, count carbs, worry about hypo- and hyperglycemia and avoid long-term complications, with no break, 24/7, 365 days a year.

So, people with T1D get understandably excited when they hear about new burden-reducing treatments and technologies; they are eager and hopeful to one day see a cure and kiss diabetes goodbye.

To date, the only treatments that can restore insulin production in patients with T1D are pancreas transplants and islet transplants from deceased donors. However, not only is there a long waiting list, but the high-risk treatment is also not suitable for all patients.

Currently, the most promising treatments in the search for a cure are those derived from stem cells. Here’s what you need to know.

What is stem cell therapy?

The stem cells naturally produced by our bone marrow are also called “mother cells,” i.e., cells from which all the cells in our body develop. They have the ability to multiply endlessly and to form different types of cells depending on the needs of the tissues where they are found, such as blood cells (e.g., white blood cells, red blood cells) in the bone marrow or insulin-secreting beta cells in the pancreas.

For this reason, stem cells are studied for the treatment of many diseases (e.g., leukemia, different types of cancer). The goal of stem cell-derived treatments is to develop healthy cells from stem cells in order to replace diseased or defective cells in the body. Stem cell transplants using cells from another organism are also possible.

In the case of T1D, these treatments can replace beta cells in the pancreas that have stopped producing insulin with new cells, enabling the body to resume producing insulin to meet its needs.

Who can benefit from these treatments and how do they work?

Because the stem cells used in current studies on T1D come from another human organism, immunosuppressive drugs are needed to prevent the body from rejecting the stem cells. This enables the transplanted healthy cells to produce insulin and maintain blood sugar levels within normal range.

Due to immunosuppressive drugs being a lifelong, heavy treatment with many adverse side effects (e.g., risk of serious infections, cancers), stem cell treatments are not suitable for all people with T1D. They are currently being studied in people who experience hypoglycemic unawareness, have recurrent severe hypoglycemia, or have frequent and extreme fluctuations in blood sugar levels despite the use of technology (e.g., continuous glucose monitor, insulin pump) and help from their healthcare team. These patients are considered at high risk of developing complications.

The way stem cells are transplanted depends on the type of treatment. For some, the cells are injected into the portal vein, which is located just below the liver, while for others, a small surgical procedure is required.

Promising research

Over the past 20 years, significant advances in stem cell research have led to promising methods in the hope of one day curing T1D.

Among the ongoing research, the Vertex Group’s VX-880 therapy, mentioned in a previous article, has been in the news a lot this past year. Early results announced by the company show that the treatment could restore insulin production and improve blood sugar control. The treatment has already shown that it may significantly decrease the amount of insulin required, or even eliminate the need for insulin for some participants. The study is still ongoing and is currently recruiting participants in Canada. Two Montreal-based teams are involved in this research project.

Another stem cell therapy, called PEC-Direct from the ViaCyte organization, was recently presented at the Endocrine Society’s annual meeting (ENDO 2022) in Atlanta, Georgia. Participants who received the treatment saw a significant increase in their C-peptide count (produced in the pancreas along with insulin), indicating that the body is resuming insulin production. In addition to the increase in C-peptides, a decrease in glycated hemoglobin (HbA1c) and insulin needs were also observed.

Still a long way to go…

While the initial results of these studies are based on small groups of participants, they still show the potential of stem cells in the treatment of T1D. This could be an interesting alternative to pancreas transplantation, which, while not very accessible and risky, still shows very good long-term results.

However, due to the immunosuppressive drugs required for stem cell treatments, this possible cure is not, for the moment, suitable for everyone.

In the meantime, other embryonic research is trying to find treatments using stem cells from the patient rather than from another human organism. This could avoid the need for immunosuppressive drugs and make a cure accessible to all people with T1D.