Diabetes Research Institute Revises Cure Strategy

February 27, 2025

Two years have passed since Dr. Matthias von Herrath, M.D., took over as Scientific Director of the Diabetes Research Institute (DRI). This is the first update since JDCA’s interview with von Herrath shortly after he assumed his new role. In 2023, Herrath emphasized the DRI would remain focused on finding a biological cure for T1D, building on the strong foundation set by his predecessor, Dr. Camillo Ricordi, M.D.

This report looks at the changes DRI has undergone within the past two years, addressing broad strategic realignments, human trial progress, and goals for the next several years.
 

About the Institute

The DRI is located at the University of Miami Miller School of Medicine (UM). Founded in 1971, its mission is “to develop and rapidly apply the most promising research to treat and cure those now living with diabetes.” Its research goal is to restore natural insulin production and normal blood sugar levels without adverse side effects, referred to as a biological cure. The DRI maintains its primary focus on T1D, even though this is not directly expressed in its mission.

The institution is funded by several sources, namely the UM and the Diabetes Research Institute Foundation (DRIF). DRIF is a nonprofit created in 1971 with the sole purpose of providing the DRI funding to cure diabetes. In FY23, DRIF provided $5 million to DRI’s cure research.

Herrath joined the DRI in 2023 as scientific director after a two-year search, succeeding Ricordi who had held the position since 1993. Ricordi’s specialty in islet transplantation and immunology shaped the organization throughout his tenure and beyond, keeping the primary focus on islet transplantation to this day. The DRI actively collaborates with the research community, including pharma and other research centers.
 

Shift in Strategy

Under Herrath’s leadership, DRI redefined its cure research strategies.

The previous strategies, “Site, Sustain, Supply,” were held in place for over a decade and squarely focused on islet cell transplantation. These referenced the need for a hospitable site to house cells, the ability to sustain cell health without rejection, and a sufficient supply of cells for islet transplantation. These were the pillars underpinning DRI’s BioHub initiative, introduced in 2013, which aimed to cure T1D by creating an integrated mini-organ that mimicked the native pancreas, producing insulin without antirejection drugs. Though the initiative did not formally end, the lack of news since 2016 suggests it has been phased out.

The new strategies are “Therapy, Target, Technology.” These refer to accelerating the development of combination therapies to protect beta cells (latent and transplanted), discovering new targets to better understand diabetes and the immune response, and utilizing the most effective technology (immune cell interface and gene editing, to name a few).

The new strategies omit reference to cell supply completely, seeming to focus on stopping the autoimmune attack. This is a good thing. While cell supply solutions seem to be taken up by commercial enterprises, cell protection solutions are still in the early stages where a lead horse has yet to emerge.
 

DRI Research: Results and Performance

Overall

Within the past two years, there have been no fundamental human trial breakthroughs stemming from DRI-driven research.

However, there are sixteen clinical trials currently housed at DRI:

• One Practical Cure (another has completed)
• Eight are for established T1D
• Six for newly diagnosed
• One to prevent T1D onset
• One for T2D

DRI states they are collaborating with Eledon Pharmaceuticals in an additional trial, housed at the University of Chicago. The phase I/II trial is testing Tegoprubart, an anti-CD40L antibody, as an alternative to traditional immunotherapy.

Some projects are sponsored and led by DRI scientists while others are owned by outside entities and conducted at the institute. The most developed human trials conducted at the institute are in phase III but are not sponsored by DRI scientists.
 

Eight Trials for Established T1D Diabetes

Research focus in established human trials is as follows:

• (3) conducting islet transplantation
• (2) observational studies following transplant
• (1) improving outcomes after complete islet graft failure
• (1) improving outcomes for T1D with kidney disease
• (1) glucose management

The T2D Trial

One trial underway at DRI focuses on T2D, indicating added scope that von Herrath may be bringing to the DRI. This is a longitudinal study tracking the effect of metformin on delaying T2D progression. The presence of this trial raises the question of whether or not the DRI will move more into T2D research, where research grants may be more plentiful.
 

Practical Cure Trials

One trial taking place at DRI qualifies as a Practical Cure: VX-264. This is a phase I/II study testing the safety and efficacy of stem cell-derived islets inside an encapsulation device to protect cells from the immune system. This trial is sponsored by Vertex Pharmaceuticals and is not led by the DRI.

DRI’s POSEIDON trial, a potential Practical Cure, was completed in the summer of last year. The purpose was to halt or stop beta cell destruction with vitamin D and omega-3. No results have been made public, and there is no indication the project will progress to a new phase. 
 

Forward-Looking Goals

DRI states that there are two wide-sweeping goals for the next two years:

1. Advancing cure-focused research
2. Enhancing clinical care and education

In addition, DRI has identified what it calls, “three must-win battles” necessary for a T1D cure. These are the development of macroencapsulation devices to protect transplanted islets, delivering tolerable immune therapy, and enabling the augmentation of adult beta cells (increasing beta cell mass). Research efforts will be focused on these priorities, they say.