Islet Cell Transplants: How Regenerative Medicine is Tackling Diabetes

Managing diabetes may no longer mean lifelong insulin injections. Islet cell transplants, a process where healthy insulin-producing cells are introduced into the body, are offering new hope for people with type 1 diabetes. The FDA's 2023 approval of Donislecel (Lantidra) marked a major step, showing that nearly 9 out of 10 recipients achieved better blood sugar control within a year, with many no longer needing daily insulin.

Key advancements include:

• Stem Cell Research: Lab-grown beta cells are reducing reliance on donor organs.
• Personalized Therapies: Reprogrammed stem cells from patients' own tissue are showing promise.
• Immune Protection: Techniques like gene editing and encapsulation aim to prevent rejection without immunosuppressants.

These therapies are evolving rapidly, offering hope to millions with type 1 and insulin-dependent type 2 diabetes. While challenges like immune rejection and donor shortages remain, the future of diabetes care is shifting toward long-term solutions that restore natural insulin production.

What Are Islet Cell Transplants?

Islet cell transplantation is a minimally invasive procedure aimed at restoring the body’s ability to produce insulin. This approach is part of regenerative medicine, focusing on replacing lost cellular function instead of just managing symptoms. Unlike a full pancreas transplant, which requires major surgery, this method involves delivering clusters of specialized cells directly into the liver. It’s currently approved for adults with type 1 diabetes who experience severe and unpredictable drops in blood sugar, a condition known as hypoglycemia unawareness. Let’s take a closer look at the role of islet cells and how this procedure works.

Islet Cells and Their Role in Insulin Production

Within the pancreas lie the islets of Langerhans, which contain beta cells responsible for producing insulin. These beta cells constantly monitor blood sugar levels and release insulin as needed. For a successful transplant, approximately 1 million islets are required - usually extracted from two donor pancreases - to restore this critical function in an average 154-pound (70 kg) adult.

The Islet Cell Transplant Procedure

The process begins with the recovery of a pancreas from a deceased organ donor. In the lab, technicians inject purified enzymes into the pancreatic duct to separate the islets from the surrounding tissue. After enzymatic processing and gentle agitation, the islets are purified and assessed for quality before being prepared for transplantation.

During the procedure, which is done under local anesthesia and sedation, a radiologist uses imaging to guide a catheter into the liver’s portal vein. The islets are then infused into the liver through the catheter, relying on gravity to ensure a slow and steady transfer. Once in the liver, the islets settle and begin integrating into the tissue. Over the next two weeks, new blood vessels form connections with the transplanted islets, enabling them to monitor blood sugar levels and release insulin into the bloodstream. Throughout the process, doctors carefully monitor portal vein pressure to minimize complications. These precise steps highlight both the potential and the challenges of islet cell transplantation.

Benefits and Drawbacks of Islet Cell Transplants

One of the main benefits of islet cell transplantation is its ability to prevent life-threatening hypoglycemic episodes. In a Phase 3 clinical trial involving 48 participants, 88% were free from severe hypoglycemic events one year after the procedure, and 52% achieved complete insulin independence. Between 1999 and 2023, the Collaborative Islet Transplant Registry reported 1,477 recipients across 40 centers worldwide, with 50% to 70% maintaining insulin independence five years post-transplant.

However, there are challenges. Recipients must take lifelong immunosuppressive medications, such as tacrolimus and sirolimus, to prevent organ rejection. These drugs come with significant risks, including kidney damage, heightened infection risk, and a greater chance of developing certain cancers. Additionally, islet function can decline over time due to inflammation, oxygen deprivation in the liver, or autoimmune attacks. Another major hurdle is the limited availability of donor organs. Of the approximately 8,000 pancreas donations in the U.S. each year, fewer than one-third are suitable for isolating islets.

"Islet transplantation serves as a crucial platform for the next generation of β-cell replacement therapies, including stem cell–derived islets, genetically engineered immune-evasive products, encapsulation technologies, and precision immunotherapy." - Lorenzo Piemonti, MD

New Developments in Regenerative Medicine for Diabetes

Stem cell and immune therapies are opening new doors for diabetes treatment. Advances in cell engineering and immune protection are transforming the way we approach managing this disease.

Creating Islet Cells from Stem Cells

Scientists are now able to grow insulin-producing cells from human pluripotent stem cells (hPSCs), including both embryonic and induced pluripotent stem cells. By replicating the natural development process of the pancreas, they use specific chemical signals - such as Activin A, Wnt, and retinoic acid - to turn these stem cells into functional beta cells capable of producing insulin.

In June 2025, Vertex Pharmaceuticals' FORWARD trial delivered promising results. Out of 12 patients who received a full dose of these cells, 10 achieved insulin independence within a year. All participants avoided severe hypoglycemia and maintained glucose levels within the target range of 70 to 180 mg/dL [NEJM, 2025].

Another milestone came in late 2024 at Nankai University. Researchers led by Wang performed an autologous transplant on a 25-year-old woman with type 1 diabetes. They chemically reprogrammed her fat-derived stem cells into pluripotent cells, which were then transformed into islet cells. By day 75 post-surgery, she no longer needed insulin, a state she maintained for over a year. Her HbA1c levels dropped from 7.57% to 5.37% by day 120, and her time-in-range improved from 43.18% to over 98% [Signal Transduction and Targeted Therapy, 2024].

However, one major challenge remains: protecting these cells from immune system attacks.

Preventing Immune System Rejection

Lab-grown islet cells are at risk of being targeted by the immune system. To address this, researchers are using gene editing and encapsulation techniques to protect the cells without requiring lifelong immunosuppression.

In August 2025, a team led by Per-Ola Carlsson at Uppsala University in Sweden performed the first human transplant of gene-edited "hypoimmune" islets. Using CRISPR technology, they removed HLA class I and II genes and added CD47 to help the cells avoid immune detection. These cells, implanted into a 42-year-old man's forearm muscle, survived for 12 weeks while producing insulin.

"This is the most exciting moment of my scientific career. [The procedure] opens the future possibility of treating not only diabetes but other autoimmune diseases." - Per-Ola Carlsson [NEJM, 2025]

Other approaches include encapsulation devices that shield the cells while allowing nutrients and insulin to pass through. Innovations like VX-264, which uses non-perforated membranes, aim to eliminate the need for immunosuppressive drugs entirely. Meanwhile, researchers at the University of British Columbia have engineered stem cell lines with eight protective genes and a safety switch that can be activated with Ganciclovir to control unwanted cell growth. Autologous transplants, where a patient's own cells are used to create islets, also show promise, as the body is less likely to reject these cells.

Using These Treatments for Different Types of Diabetes

These regenerative therapies are now being tested for insulin-dependent type 2 diabetes, particularly in cases of beta cell failure.

In April 2024, researchers at the Shanghai Institute of Biochemistry and Cell Biology treated a 59-year-old man with a 25-year history of type 2 diabetes. They used his own stem cells to create islets, which were transplanted via the portal vein. By week 11, he no longer needed insulin, and by week 56, he had stopped all oral diabetes medications. At a 116-week follow-up - over two years post-transplant - his blood sugar remained well-controlled, with 99% of his time spent in the tight target range [Cell Discovery, 2024].

Type 2 diabetes offers a unique testing ground for these therapies since it lacks the autoimmune component of type 1 diabetes. This allows researchers to better evaluate the function of transplanted cells. However, more studies are needed to understand how these treatments work in the presence of insulin resistance and to assess their long-term benefits for a larger group of patients.

Determining If Islet Cell Transplants Are Right for You

With advancements in regenerative medicine transforming diabetes care, figuring out if islet cell transplants are a good fit for you is an important step. While this treatment offers promising results, it’s not suitable for everyone. Knowing the qualifications and what the process entails can help you have meaningful discussions with your healthcare provider about whether this option aligns with your needs.

Assessing Your Diabetes Type and Treatment Goals

Islet cell transplants are currently approved for adults with type 1 diabetes who struggle with severe, recurring low blood sugar episodes. To qualify, candidates must also experience hypoglycemia unawareness. If you’ve documented two or more severe hypoglycemic events within six months despite optimal diabetes management, you may be a candidate.

Your treatment goals play a key role in determining if this procedure is right for you. While some patients achieve complete insulin independence after the transplant, that’s not the primary goal for everyone. Clinical trials have shown that nearly 90% of recipients maintained an A1C level below 7% and avoided severe low blood sugar episodes one year after the procedure. About 50% of patients were insulin-independent at the one-year mark, though this number often falls to 40% by the second year. If your primary focus is preventing life-threatening hypoglycemia rather than eliminating insulin use altogether, this treatment could still provide meaningful benefits. Next, let’s look at the eligibility criteria and factors that may affect your candidacy.

Who Qualifies and What to Consider

To qualify, candidates must be 18 to 65 years old, have been insulin-dependent for over five years, and have developed type 1 diabetes before the age of 40. Additionally, negligible C-peptide levels are required to confirm eligibility.

Certain conditions may disqualify you from the procedure. These include portal venous pressure above 20 mmHg, active infections, a history of cancer within the last five years, severe untreated heart or liver disease, and pregnancy or plans to become pregnant. A high body mass index (BMI) may also be a concern, as it can lead to increased insulin resistance, potentially overworking the transplanted islets.

Another key consideration is the need for lifelong immunosuppressive therapy, which comes with its own risks. Most programs also require a psychological evaluation to ensure you’re prepared to commit to the strict medication and monitoring regimen.

Questions to Ask Your Healthcare Team

Once you’ve confirmed your eligibility, it’s important to ask the right questions to guide your decision-making process. Start by asking about the center’s 3- and 5-year insulin independence rates. Clarify whether the primary goal in your case is to eliminate severe hypoglycemia or to achieve full insulin independence. Having a clear understanding of what to expect can help you make an informed decision.

It’s also helpful to ask about specific risk factors. For example, you might ask, “Am I at an increased risk for portal vein thrombosis based on my current vascular health?” or “How will immunosuppressant side effects, like high blood pressure or tremors, impact my daily life compared to my current diabetes complications?”

Finally, explore newer treatment options. Ask if there are clinical trials for technologies like stem cell–derived islets or encapsulation methods that you might qualify for. These approaches could address challenges like donor shortages or reduce the need for immunosuppressants. Don’t forget to check if your insurance covers the procedure and how you’ll manage the ongoing costs of anti-rejection medications.

How Newborn Stem Cell Banking Supports Future Diabetes Treatments

Islet cell transplants currently depend on cadaveric donors, but newborn stem cell banking provides a way to preserve cells that could play a role in diabetes treatments. Stem cells collected at birth have unique properties researchers are studying to develop insulin-producing cells and regulate immune responses. Let’s break down what newborn stem cell banking involves.

What Is Newborn Stem Cell Banking?

Newborn stem cell banking involves preserving cord blood, cord tissue, placental cells, and exosomes collected at birth. Each of these materials has specific benefits:

• Cord blood contains hematopoietic stem cells (HSCs), which can develop into blood and immune cells.
• Cord tissue is rich in mesenchymal stem cells (MSCs), which show promise in modulating immune responses.
• Placental tissue provides another source of MSCs.
• Exosomes are signaling molecules that help with cell communication.

These materials are cryogenically stored for future use in regenerative therapies. For example, Americord Registry's Cord Blood 2.0™ technology collects up to twice as many stem cells as the industry average. This increased collection capacity allows treatment for patients weighing up to 165 pounds, making it a long-term resource as children grow into adults.

Research on Banked Stem Cells for Diabetes

Ongoing research highlights several ways newborn stem cells could contribute to diabetes treatments. Scientists are working on differentiating these cells into insulin-producing beta cells, offering an alternative to the limited supply of donor islets. MSCs from cord and placental tissue are also being studied for their potential to suppress autoimmune responses in type 1 diabetes and reduce transplant rejection risks.

One example of progress comes from a Phase II trial conducted by NextCell Pharma in September 2020. Their study, called ProTrans, involved 15 adults newly diagnosed with type 1 diabetes. Ten patients received high-dose MSC treatments from umbilical cord tissue, and during the first year, they retained 90% of their natural insulin production. In comparison, the five placebo patients lost 47%.

Another area of exploration involves genetic modifications to make banked stem cells less visible to the immune system. This could reduce or even eliminate the need for long-term immunosuppressive drugs. Additionally, umbilical cord blood cells naturally carry a lower risk of graft-versus-host disease, which may lead to fewer complications compared to other donor sources.

Americord Registry Services for Family Health Planning

Preserving newborn stem cells is a forward-thinking step for families, especially as regenerative medicine continues to evolve. Americord Registry provides several options for stem cell banking, including cord blood, cord tissue, placental tissue, and exosome banking. Their 5-compartment storage bags allow families to divide a single collection for use in multiple therapies or clinical trials, rather than depleting the entire sample at once.

For families interested in comprehensive coverage, Americord's Ultimate Family Plan includes all four banking options at a cost of approximately $293 per month over 24 months. Banked stem cells are a 100% genetic match for the baby and can be a partial match for siblings or parents, offering a potential resource for treating diabetes or other conditions. Placental tissue banking, in particular, provides MSCs that are a perfect genetic match for the mother, which could support her health needs in the future.

With about 9 million people living with type 1 diabetes today - a figure projected to rise to 17.4 million by 2040 - preserving these biological resources at birth gives families more options as medical advancements continue.

Conclusion: What's Next for Regenerative Medicine in Diabetes Care

Islet cell transplantation has evolved from a research concept into an FDA-approved therapy. The focus is now shifting toward stem cell-derived islets, which could provide a steady supply of insulin-producing cells. This shift is critical, especially with approximately 9 million people currently living with type 1 diabetes - a number that could climb to 17.4 million by 2040. These advancements aim to address the shortage of donor organs and meet the growing need for effective treatments.

Gene editing tools like CRISPR are being harnessed to create immune-evasive cells. These cells have the potential to avoid immune system attacks, making lifelong immunosuppressive drugs unnecessary. In June 2025, Vertex Pharmaceuticals shared encouraging updates from its FORWARD study at the American Diabetes Association's 85th Scientific Sessions. All 12 participants in the study successfully restored their own insulin production and eliminated severe hypoglycemic events. By early 2026, further research highlighted rapid insulin independence with sustained blood sugar control - an exciting step forward for future therapies.

"Stem cell therapy is showing tremendous promise in transforming type 1 diabetes care, offering real hope for insulin independence. With advances in genetic engineering, these therapies may one day also evade immune attack - eliminating the need for immunosuppressive drugs." - Marlon Pragnell, Vice President of Research and Science, American Diabetes Association

As gene editing continues to refine cell therapy, newborn stem cell banking is emerging as a proactive option for personalized care. This approach preserves mesenchymal stem cells (MSCs), which researchers are exploring for their ability to modulate the immune system and support transplanted islet cells. By moving toward therapies that use a patient’s own cells, banked stem cells could play a pivotal role in creating personalized regenerative treatments.

To stay at the forefront of these developments, keep an eye on ClinicalTrials.gov for updates on Phase 3 studies. Discuss emerging therapies with your healthcare team, especially as the FDA’s 2023 approval of islet transplantation opens the door for broader use of stem cell-derived treatments. Staying informed will help you make timely decisions about treatment options and stem cell preservation.

FAQs

How long do islet cell transplants typically last?

Islet cell transplants typically remain effective for up to 8 years. Research indicates that their longevity can vary based on individual circumstances and improvements in transplant procedures.

Can I avoid anti-rejection drugs with newer islet therapies?

Recent developments in islet cell transplantation for type 1 diabetes are offering hope for reducing or even eliminating the need for lifelong immunosuppressive drugs. Early trials with experimental therapies, such as gene-edited hypoimmune islet cells and stem cell-derived islet cells, have shown encouraging results in avoiding immune rejection without relying on these medications. While these methods are still in the experimental phase, they mark a step forward in potentially decreasing dependence on anti-rejection drugs in the years ahead.

How can newborn stem cell banking help with future diabetes treatments?

Newborn stem cell banking holds promise for future diabetes treatments, particularly type 1 diabetes. Stem cells preserved from umbilical cord blood or tissue can potentially be transformed into insulin-producing islet cells. These cells have the ability to replace damaged pancreatic beta cells, helping to restore insulin production. By banking stem cells early, families secure a personalized resource that could minimize immune rejection risks and contribute to advancing regenerative approaches in diabetes care as research continues to evolve.