A New Option to Halt Diabetes Shows Promising Results in Phase II Trial

A team of researchers from Uppsala University in Sweden has been investigating the potential of using a particular type of cells, known as mesenchymal stromal cells (MSCs), to slow the advancement of type 1 diabetes in newly diagnosed patients.

The findings of their study were published in the scientific journal Diabetologia.

This study involved a relatively small group of 24 participants. The team used a unique source of MSCs for their research, taken not from the study’s patients (allogeneic) but instead derived from a substance known as Wharton’s jelly.

Wharton’s jelly is a gel-like tissue that envelopes the blood vessels within the umbilical cord, which connects a developing fetus to the placenta. MSCs harvested from this jelly have the potential for regeneration, can differentiate into a limited range of cell types, possess self-renewal abilities, and have the capacity to communicate with other cells leading to immunomodulation.

What makes these Wharton’s jelly-derived MSCs particularly fascinating is their immunomodulatory properties, which prevent the body from rejecting them as foreign tissue. This unique characteristic makes these MSCs suitable for creating allogeneic “off-the-shelf” treatments.

The study utilized a unique selection method known as ProTrans to gather the Mesenchymal Stem Cells (MSCs) required. This technique was developed by NextCell, a Swedish bio-pharmaceutical firm linked with various scientists who contributed to the research.

The investigation included a merged Phase I/II trial that started with a dose escalation and proceeded to a randomized, double-blind, placebo-controlled study.

The examination contrasted the treatment using ProTrans-derived allogeneic MSCs with placebo in adults who were recently diagnosed with type 1 diabetes.

The research aimed to observe alterations in C-peptide during a mixed meal tolerance test one year after the ProTrans or placebo infusion, compared to the baseline results prior to treatment.

In individuals treated with the placebo, there was a notable decline in C-peptide levels by 47%, whereas those who received the ProTrans treatment only experienced a drop of 10%. Likewise, insulin requirements rose by a median of 10 U/day in placebo-treated participants but did not change in individuals treated with ProTrans throughout the 12-month follow-up.

This research implies that the use of allogeneic MSCs derived from Wharton’s jelly through ProTrans can provide a safe treatment option for newly diagnosed type 1 diabetes, with the potential for long-term preservation of beta cell function.

There is currently no other treatment available on the market or under clinical trials that have demonstrated the capability to halt the progression of diabetes.

About the study model

A study model, often known as a double-blind approach, is constructed in a manner where both the participants and the investigators are uninformed about whether a proposed intervention or a control substance is in use. This methodology is advantageous as it minimizes experimental bias, and it becomes paramount for the authenticity of the findings when the investigators or the study itself have affiliations with a commercial entity that might profit from the study’s outcome.

Before the study began, a web-based code was developed to implement randomization, with participants assigned to either the ProTrans or placebo treatment groups. In the clinic, sealed envelopes containing corresponding code allocations were securely stored in a locked room. These envelopes were opened by the study staff during initial visits. Through this approach, participant randomization was accomplished even before they interacted with the study personnel, thereby eliminating the possibility of assignment bias.

The identities of the groups and the nature of the treatments in the study were unknown to all those involved, including both participants and research staff. The key to the randomization was only revealed once all of the data had been gathered and assessed. This was subsequently examined by the Medical Statistics Unit at Karolinska Institute.

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