At December’s Cell and Gene Therapy International Europe conference, NextCell presented research on its mesenchymal stem cell (MSC) therapy aimed at transforming treatment for type 1 diabetes mellitus (T1DM).
T1DM affects approximately two million Americans, including over 300,000 children. Current management relies on insulin replacement via daily injections or pumps, a regimen that demands constant attention and may lose effectiveness as patients develop insulin resistance over time.
MSCs are multipotent stem cells known for their immunomodulatory effects, influencing T-cells and B-cells to regulate immune responses. NextCell’s Chief Scientific Officer, Lindsay Davies, highlighted the company’s efforts to use MSCs to improve diabetes care.
“There is a real need for therapies that can slow or even reverse disease progression in type 1 diabetes,” Davies said. “Most drug development today focuses solely on restoring insulin production.”
NextCell is developing ProTrans, an MSC therapy designed to modulate the autoimmune response that destroys insulin-producing pancreatic beta cells.
“Type 1 diabetes involves crosstalk between beta cells and the immune system, leading to cell destruction,” Davies explained. “Injected MSCs interact with phagocytes and macrophages, which then influence adaptive immune cells, creating a tolerance effect that protects pancreatic tissue.”
ProTrans is designed as an off-the-shelf therapy, requiring no post-processing or GMP-compliant facility. “We source MSCs from umbilical cords, generate a cell bank from multiple donors, and use a patented algorithm to select the optimal mix,” Davies said. “The product can be cryopreserved, thawed at bedside, and infused intravenously.”
The therapy is fully allogeneic, avoiding the need for patient-specific cells and offering advantages over autologous treatments. “From an industrial perspective, the process is robust and scalable, with strong potential for pre-commercial applications,” Davies added.
Despite skepticism in the scientific community—only 12 MSC therapies are approved worldwide despite more than 1,600 trials—the company has presented supporting clinical data.
In a Phase I/II double-blind study (NCT03406585) of men aged 18-40, ProTrans was compared to placebo. Efficacy was measured by C-peptide levels, which indicate beta-cell activity. After 12 months, placebo patients saw a 47% decline in C-peptide levels, while ProTrans patients experienced only a 10% decrease after a single infusion.
“The therapy halted disease progression,” Davies said. “In observational follow-up over five years, the effect persisted.”
However, ProTrans is intended for stage-three, newly diagnosed patients with residual beta-cell function—typically around 20%. Stage-four patients, who are fully insulin-dependent, are unlikely to benefit.
NextCell is now expanding trials to the pediatric population, where the unmet need is high. “We have launched a pediatric trial with 66 patients, bringing our total treated to 90,” Davies announced. “We expect results by next September.”
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