Cell Therapy Sustains Insulin Production for 6 Years in Type 1 Diabetes
Umbilical cord-derived cell therapy preserved endogenous insulin production in T1DM patients.
As autoimmune diseases continue to place a growing burden on healthcare systems worldwide, the field has started to move beyond immunosuppressive therapies that slow decline without meaningfully changing disease trajectories.
After first emerging as a concept in the early 2000s, cell-based immunomodulation has resurfaced as a promising therapeutic modality for autoimmune diseases, though not without concerns surrounding durability, scalability, and standardization.
Few leaders are as well-positioned to address these apprehensions as Dr. Lindsay Davies, chief scientific officer at NextCell Pharma and co-founder and chief executive officer of QVance.
Drawing on more than two decades of experience working with mesenchymal stromal cells (MSCs), Davies shared her insights with Technology Networks at Advanced Therapies UK. She explained how NextCell’s ProTrans therapy is challenging assumptions about what autoimmune treatment should look like, why umbilical cord-derived MSCs are biologically distinct from other sources, and how regulatory frameworks are forcing the field to clarify mechanistic explanations.
ProTrans: Redefining autoimmune therapy for Type 1 diabetes
Type 1 diabetes is an autoimmune disease in which the immune system destroys insulin-producing beta cells in the pancreas. Without sufficient insulin, individuals cannot adequately regulate blood glucose levels.
If left untreated, dysregulated glycemic control can damage blood vessels and nerves over time. This increases the risk of cardiovascular events as well as chronic wounds that may not heal properly and could lead to limb amputation. Short-term complications, such as diabetic acidosis, can be life-threatening. Therefore, people with Type 1 diabetes require lifelong insulin treatment and intensive monitoring to manage their blood glucose.
NextCell is hoping to change that with ProTrans—an umbilical cord-derived MSC therapy for Type 1 diabetes. The company reports that Phase 1/2 studies demonstrated preservation of endogenous insulin production for at least six years following a single dose, supporting its use as a durable, disease-modifying Type 1 diabetes therapy.
“That’s quite phenomenal. ProTrans administration doesn’t need a specialist team. It can be given in an outpatient clinic, which makes it scalable from a patient access perspective, wherever we are in the world.” — Dr. Lindsay Davies.
That simplicity is not accidental. ProTrans was deliberately designed as an allogeneic, off‑the‑shelf product to avoid the logistical and economic barriers associated with autologous cell therapies.
The implications for global access are central to NextCell’s strategy. Davies explained: “We don’t want to just serve the richest and most affluent countries. We recognize the potential for patients across the globe.”
ProTrans could reframe Type 1 diabetes management:
- Efficient: A single, outpatient infusion with no hospitalization requirements
- Long-lasting: Durable immune modulation in adult Type 1 diabetes
ProTrans repeat dosing shows potential to extend beta-cell preservation
While ProTrans is positioned as a disease-modifying, not regenerative, treatment, the long-term preservation of endogenous insulin production has opened the door to new hypotheses.
“That’s when we started asking, what if we did repeated treatment? Could we halt progression for even longer, or give the body enough space to initiate self‑repair of remaining beta cells?” Davies said.
In a Phase 1 repeated-dose study, a small cohort receiving two infusions maintained an average 91% preservation of insulin production over 6 years. Additionally, NextCell recently announced that patients in the same group maintained preservation of endogenous insulin production for up to 7.5 years following their first infusion. Importantly, the long-term repeated-dose data are not yet peer-reviewed.
While the team focuses on single-dose treatment for now, Davies emphasized that these results lay a promising foundation for future work.
Preliminary data demonstrate long-term benefits:
- Early repeat‑dosing data suggest ProTrans significantly extends preservation of endogenous beta‑cell function
- These findings suggest that immune reprogramming has the potential to halt disease progression
- The repeated‑dose data are preliminary, so larger, controlled studies are needed to validate these findings
Umbilical cord MSCs are not like any other MSCs
While MSCs from different tissue sources share immunomodulatory capacity, their developmental origins matter profoundly. Tissue source influences how the cells interact with the immune system, how they are tolerated by the host, and, consequently, what therapeutic windows are feasible.
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Davies explained: “We use umbilical cord MSCs specifically because the cells retain a memory of where they came from and what their role was. Umbilical cord MSCs remember fetal–maternal tolerance.”
Fetal–maternal tolerance
Fetal–maternal tolerance refers to the specialized immune state established during pregnancy that allows the mother’s immune system to tolerate the fetus, which carries foreign antigens, without mounting a destructive immune response.
In umbilical cord-derived MSCs, this “tolerance memory” has beneficial clinical consequences. Unlike bone marrow or adipose‑derived MSCs, umbilical cord MSCs do not induce anti‑human leukocyte antigen (HLA) antibody formation, a crucial determinant of antibody-mediated therapy rejection.
“This is quite a unique phenomenon. Other MSC sources do generate HLA antibodies. We don’t see that in our studies with umbilical cord.” — Dr. Lindsay Davies.
From a safety perspective, the implications are significant. The absence of sensitization could support repeat dosing and long-term use without increasing immunological risk.
The advantages of umbilical cord MSCs:
- Retention of fetal–maternal immune tolerance and absence of anti‑HLA antibody formation
- Improved safety profile compared with bone marrow or adipose MSCs
MSC therapy is finally standardizing
Over the past two decades, MSC therapy research has progressed despite controversies. The umbrella term “MSC” masked profound biological variability across products and knowledge gaps limited reliable translation from bench to bedside—the therapeutic potential was there, but the mechanism was poorly understood.
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According to Davies, that era is ending. She explained: “We can’t just say ‘MSC’ anymore. The cell source matters, the patient matters, the manufacturing process matters, and the delivery route matters. All of this impacts the mechanism of action.”
A major inflection point, Davies noted, was the new chapter adopted by the European Pharmacopoeia: Cell-based preparations for human use. The chapter provides a comprehensive framework on MSC production and characterization and came into force on April 1, 2026.
Davies was particularly pleased to see that the guidance promotes the use of functional assays over traditional potency assays. “A functional assay recognizes that you are putting a living cell into a dynamic environment,” she explained.
The shift toward standardization reflects a maturing field, as well as regulatory influence. Academic consensus reports, once siloed, are now influencing regulatory expectations, closing the loop between discovery, development, and approval. Davies reasoned that this collaboration results in clarity that benefits everyone: developers, regulators, clinicians, and, ultimately, patients.
MSC mechanism‑of‑action understanding is evolving
- Regulatory frameworks require explicit cell‑source definition
- Functional assays are prioritized over simplistic potency markers
- Mechanism is increasingly understood as context‑dependent, not universal
Scientific maturity meets real‑world impact
Ultimately, advances in our understanding of MSC mechanisms, coupled with regulatory advances, are reshaping what is achievable in autoimmune therapy.
As researchers continue to establish scientific feasibility, emphasis should also be placed on translating durable, non-immunosuppressive MSC therapies into clinical settings.
Key takeaways:
- ProTrans demonstrates that long‑acting, low‑burden autoimmune therapy is biologically and operationally achievable
- Umbilical cord MSCs offer unique immunological advantages
- Standardization of mechanism‑of‑action frameworks marks a turning point for MSC credibility and scalability across global markets
This content includes text that has been created with the assistance of generative AI and has undergone editorial review before publishing. Technology Networks' AI policy can be found here.
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