Breakthrough T1D’s Project ACT: A Dedicated Cure Initiative?

January 14, 2025

On November 14th of last year, Breakthrough T1D (formerly JDRF) announced the launch of Project ACT (Accelerate Cell Therapies). It is described as “the flagship initiative to Accelerate Cell Therapies, an anticipated pathway to Cures for Type 1 Diabetes.”

On the surface, this initiative appears to be a good development, bringing a dedicated focus to cell supply and cell protection solutions. But, there are many open questions and some genuine concerns that need to be addressed. In the best case, Project ACT will be empowered to break barriers and dramatically accelerate a cure. In the worst case, it is marketing topspin re-packaging work that is already underway.

This report looks closer at the details of Project ACT.
 

What Is Project ACT?

Project ACT's stated mission is to “dramatically speed cell therapy products as T1D cures through coordinated efforts to simultaneously advance research, development, regulatory, access, and adoption.”
 
Breakthrough T1D states that cell therapies are a viable pathway to an eventual T1D cure, and it wants ACT to be the “roadmap” to this end. The roadmap is comprehensive, addressing five principal workstreams:

1. Research and Development: Invest in protection strategies, fund research, and build on current work within the existing Centers of Excellence.
2. Clinical Testing: Create guidelines for trial models to expedite eventual approval.
3. Regulatory Policy: Influence and de-risk regulatory pathways.
4. Health Policy: Build and expand access and coverage for future therapies.
5. Medical Affairs: Ensure therapies can be quickly implemented when approved.

The research and development portion of ACT intends to address both cell supply and cell protection, as follows:

1. Developing an unlimited source of stem cell-derived beta cells for transplantation.
2. Protecting transplanted cells from the immune system’s attack without broad immunosuppression.

Timing

The project was initiated with $75 million in gifts from three donors, which may have precipitated the timing. It is unclear if Breakthrough T1D was seeking donors to underwrite Project ACT, or if the donors required the money be used for this type of dedicated initiative. The level of Breakthrough T1D’s monetary contribution to the project is also unclear.
 
According to Breakthrough T1D, the initiative has not begun, and will not begin until the first $100 million of the total budget ($345 million) has been secured. The nonprofit seeks high-net-worth philanthropic partners to secure funding and underwrite the initiative with gifts of at least $5 million. As it stands, the average donor cannot give directly to the project. This prevents the majority of the cure-focused T1D community from giving, losing a collective powerhouse of smaller but dedicated potential donors. 
 

Cell Supply and Cell Protection: What Should Be the Focus?

Cell supply and protection are two key research pathways that, when achieved together, should result in a Practical Cure for T1D. This is not a new understanding, and one must ask why Breakthrough is launching this initiative now, as opposed to years ago. The glass half-full perspective is ‘better late than never.’ The other perspective wonders why it took so long.

Today, more than ever before, work addressing cell supply is largely manned by commercial enterprises. There is enough business potential in developing stem cell-derived beta cells that large and small companies alike are interested, with many already in aggressive development. Vertex Pharmaceuticals recently announced a phase III trial for VX-880, testing its proprietary cell line, as the last step before FDA approval and the product is marketed. Large nonprofits are no longer needed to get cell supply research into commercial development—that ship has sailed.
 
On the other hand, cell protection remains an open field. There is no clear leader, and projects are all over the developmental map from bench to early human trials. Other pathways are being tested, including gene-edited cells, encapsulation, and immunomodulation, but none have demonstrated sustained success in humans. This seems like the optimal area for Breakthrough T1D to put the lion’s share of its focus. The verbiage in Project ACT’s roadmap suggests more emphasis will be placed on cell protection solutions, but this is unconfirmed.
 
JDCA hopes that Breakthrough T1D will place its primary focus on solving cell protection, as this is truly where the nonprofit can deliver the greatest impact. Additionally, we hope research support is extended beyond their Centers of Excellence to reach any principal investigators who demonstrate real progress.
 

The Inspiration: Artificial Pancreas Project 

Project ACT was inspired by Breakthrough T1D’s Artificial Pancreas Project (APP). It was created in 2005 and led by Jeffrey Brewer and Aaron Kowalski, later CEOs of the nonprofit in 2011 and 2019, respectively. 

The program was a multimillion-dollar initiative to accelerate the development and FDA approval of hybrid-closed loop systems. $171 million was invested, supporting R&D, clinical trials, and lobbying to expedite approval and insurance coverage.

In 2016, Medtronic released the MiniMed 670G, called (controversially) the first Artificial Pancreas by integrating an insulin pump with a CGM. Breakthrough T1D claims a degree of credit for this launch, and for the competitor products that soon followed.
 

The Practical Cure Initiative We’ve Been Waiting For?

Project ACT is still quite new, and as a result, there are many unknowns.
 
If it is a well-managed and prioritized initiative, it could make a genuine impact. If it is a marketing spin on already existing programs and activities, then we will only get more of the same. The real test will be to evaluate the progress of Project ACT at the six-month and one-year marks.

Key Questions

Leadership: Who will lead the initiative?
Donations: Why is there no opportunity for donors to give directly to Project ACT?
Performance and Accountability: What are the short and long-term metrics to measure success?
Recipients: What research stage (in vitro, animal, human) and what researchers (academic, commercial) will receive priority funding?
Funding: Will Breakthrough T1D use its $188 million cash surplus to bolster the new project, or will all funding originate from outside entities?