Managing type 1 diabetes is a lifelong task that cannot be done just by adhering to a particular lifestyle or using prescription drugs or medical devices. Since there’s currently no cure for type 1 diabetes, it’s a no-brainer that people are seeking different ways to deal with it.
One of the exciting emerging treatments¹ for type 1 diabetes is stem cell therapy. Continue reading to learn more about stem cell therapy and its treatment of type 1 diabetes.
We make it easy for you to participate in a clinical trial for Type 1 diabetes, and get access to the latest treatments not yet widely available - and be a part of finding a cure.
Type 1 diabetes is a chronic disease in which the pancreas is destroyed, disrupting its ability to make insulin. Insulin helps the body’s cells to utilize glucose for energy. Once you’ve eaten, insulin stimulates the passing of glucose from the blood to your body cells so you can use it to fuel your activities.
Type 1 diabetes is an autoimmune disease. This means that the body attacks the beta cells (the insulin-producing cells), mistaking them to be as infectious agents. Since there’s no known cause for type 1 diabetes, there are no successful treatments for it, either.
While you can manage type 1 diabetes with diet, exercise, and insulin, there’s no current cure (yet) for it. Research is underway to find ways to reverse the disease so that people can live healthily without medication. Most of these studies focus on restoring the body’s ability to produce insulin naturally.
Stem cells are undifferentiated cells that can generate other types of cells with specific functions. The first two cells generated are referred to as daughter cells. The daughter cells can either become a new stem cell or be the initial cell for developing specialized cells, such as the brain, bone, or blood cells.
Researchers are relying on stem cells due to the following advantages:
When researchers observe how stem cells generate nerves, muscles, bones, and other organs, they get a better understanding of how a certain disease or condition develops.
Researchers can guide stem cells to specific tissues to repair damaged cells.
Researchers can use stem cells to test the drugs for safety and quality. In these tests, stem cells are programmed into tissue-specific cells to try a new drug.
The stem cell is then programmed to acquire the properties of the cells targeted by the drug. Studies to program these cells to create specific ones are still underway.
Stem cell therapy treats various diseases such as neurological conditions, orthopedic, autoimmune, and inflammatory conditions. There are studies conducted about its use to treat multiple sclerosis, chronic obstructive pulmonary disease, amyotrophic lateral sclerosis, Crohn’s disease, stroke recovery, and type 1 diabetes.
Stem cell therapy doesn’t necessarily cure these conditions. Stem cells are instead used to allow the body to heal itself enough to reduce the specific disease symptoms for an extended period.
Stem cell therapy is being used to explore the complex ways of treating diabetes from its root cause, including:
Why the immune system targets the beta cells and no other cells
The cause of insulin resistance in type 2 diabetes
Recent studies are showing progress in generating insulin-producing cells² (beta cells) from embryonic stem cells (ESCs) and induced pluripotent stem cells (iPS).
Laboratory studies have helped better understand diabetes progression, the potential genetic cause, and the similarities and differences among patients. Researchers use the information to diagnose people earlier, avoid disease progression, and treat diabetes.
Furthermore, ESCs and iPS-derived beta cells can replace beta cells, providing a potential cure for diabetes if researchers can transplant them into patients. Particularly on iPS, since they can be patient-derived, there are very few chances of transplant rejection, although patients with type 1 diabetes might experience an attack on their beta cells.
From the research conducted by Melton Lab, Vertex Pharmaceuticals have developed the VX-880. This is an investigational stem cell-derived pancreatic islet cell replacement therapy specific for type 1 diabetes.
The transplant of pancreatic islet cells from a donor pancreas already exists in a therapy known as an islet transplant. However, islet transplant depends on donated pancreas cells, which are in short supply.
To solve this problem, stem cells can be the answer. With this approach, Vertex Pharmaceuticals initiated the first human trial³ in March 2021. In this treatment, scientists injected the patient with a half dose of the VX-880 along with immunosuppressive therapy.
This therapy was meant to avoid immune rejection of the new islet cells.
The first patient involved in this human trial has lived with type 1 diabetes for 40 years and has managed it through insulin injections. The experiment was successful, and the patient experienced an intensive improvement in various measures from the test. This includes improved glycemic control and decreased exogenous insulin requirement.
These improvements indicate a restoration of the insulin-producing islet cells.
However, these are just early findings from one person. There’s still a lot of work needed to ensure that the treatment is effective and safe. In this sense, large-scale clinical trials need to be conducted to determine stem cell therapy’s effectiveness in managing blood sugar levels, especially in the long term.
Stem cell therapy in regenerative medicine promises to treat many diseases, such as type 1 diabetes. Stem cells address the need to replace beta cells and control the autoimmune response to insulin-producing cells.
Currently, beta cells suitable for transplant are derived from pluripotent cells (i.e., cells that have the capacity to self-renew) from various sources, such as the pancreas and liver. Researchers have maintained a protocol that produces cells with molecular characteristics that resemble insulin-secreting cells.
Unfortunately, these cells are unresponsive to glucose. This issue is being addressed through some molecular strategies in clinical trials.
According to the FDA, unproven stem cell therapies can be unsafe.⁴ Their warnings cut across different stem cell researches, with the following concerns:
Reaction due to the administration site
The ability for the cells to move from the placement site and change into inappropriate cells and multiply
Failure of the stem cells to work as expected
Growth of tumors
There are chances of experiencing these safety risks even when the stem cells are from your cells. You should also note that manipulating the cells after removal may contaminate them, and they wouldn’t be effective as expected.
It may take years to get a genuinely effective stem cell-based treatment for diabetes. The main challenges are finding enough insulin-producing cells and protecting the cells from attacking the immune system.
Regardless of these challenges, researchers have been able to produce insulin-producing cells from iPS and embryonic stem cells.
The following approaches are being used to replace lost beta cells and avoid further damage to them:
Embryonic and iPS cells can be made on a large scale and manipulated to become any cell type in the body, such as insulin-producing beta cells. Recent technologies make this a promising avenue to generate large volumes of replacement beta cells.
Beta cells can make copies of themselves in the pancreas, although this process slows as you grow older. Researchers are looking for a drug that would enhance self-renewal to treat type 1 diabetes at its early stages.
Bioengineers and immunologists are finding ways to protect transplanted stem cells from immune attacks. One of their approaches is using cellular engineering to make the cells more resistant to immune attacks.
Another approach is encapsulating the cells with a semi-permeable membrane. This membrane can only allow glucose or insulin to pass through and protect the cells from immune attacks.
Again, these approaches are still in the preliminary studies. Therefore, there’s a need for further research to establish whether they prove to be safe and effective before they’re rolled on a large scale.
Stem cell therapy brings hope to the treatment of type 1 diabetes. However, this approach is in the preliminary stages, and there’s more to be done. With further research being conducted by Vertex Pharmaceuticals and other companies, there are high chances that this approach will be approved sooner than expected.
Diabetes clinical trials | Diabetes Research Institute
FDA warns about stem cell therapies | U.S. Food and Drug Administration