This is the first in a series of reports on the top five largest T1D research centers in the U.S. It will feature a profile of the City of Hope (COH) Diabetes & Metabolism Research Institute, located in California.
In addition to our initial profile, last week the JDCA attended a COH sponsored research symposium and met with Dr. Bart Roep, the Founding Chair of the Department of Diabetes Immunology and Dr. Debbie Thurmond, Chair of the Department of Molecular & Cellular Endocrinology at COH to discuss institution goals and research. Several other notable researchers were in attendance, including but not limited to, Doug Melton, Ed Damiano, Jane Reusch (the ADA president-elect), and Mark Atkinson (Head of the JDRF Research Advisory Committee and Professor at the University of Florida).
Earlier this year, the JDCA published a report on a significant $50m donation to COH, which catapulted COH into a position of being one of the most well-funded institutions working on T1D in the country (click here to view). As part of the gift, COH established the Wanek Family Project to Cure Type 1 Diabetes and set a bold goal of curing type 1 diabetes within six years.
COH STRUCTURE AND T1D RESEARCH PATHWAYS
City of Hope first established a Division of Diabetes in 1971. It has continued to explore diabetes-related research topics for the past five decades with the goal of understanding the disease, advancing islet cell transplantation, developing drugs, and studying the relationship between diabetes and cancer. It is comprised of six major departments (T1 and T2 focused), including most recently The Wanek Family Project for Type 1 Diabetes, led by Roep.
The Wanek Project pursues four main research pathways and the division of the $50m gift across the core focus areas is dependent upon progress, with priority given to clinical trials, innovation, and acceleration. The core focus areas are:
1. Immune Modulation
- Studying mixed chimerism (combining donor and recipient immune systems) to reverse autoimmunity in type 1 diabetes in mouse models.
- Exploring nano cell products that protect beta cells from destruction.
- Examining similarities between T1D and cancer patient immune systems.
2. Beta Cell Expansion and Replacement
- Demonstrating an ability to significantly improve the survival of beta cells.
- Studying proteins that are found at abnormally low levels in patients with diabetes.
- Expanding an islet cell transplantation program and working to create a lasting and sustainable supply of insulin-producing cells.
3. Preventing and Reversing Complications
- Identifying at risk pre-diabetic patients.
- Using precision medicine approaches to help patients with, or at risk for, type 1 diabetes.
4. Innovation Program – This program consists of studies prioritized by the impact that seed funding can be expected to have on research advancement.
- Catapult projects: Early stage projects that can be accelerated by an influx of funding.
- Incubator projects: Funding for these projects will advance critical studies to fill in gaps of understanding and determine the clinical promise of the project.
Outside of the Wanek project, which began in January and has not yet advanced any projects into clinical trials, COH is overseeing three active T1D trials in human testing. None of the current trials are considered Practical Cure research. (See appendix A for trial status and NIH number)
TIMELINE FOR T1D TESTING
The Wanek gift and program is milestone driven, with only the first two years of funding guaranteed, and the remaining four tied directly to progress, as evaluated by an Oversight and Advisory Committee. According to Roep, the funding for the remaining four years will be assigned on basis of progress and promise, as well as new projects arising in the first two years. Roep and Thurmond both expressed optimism about the Wanek family project during the JDCA interview at the COH symposium and felt the six-year timeline was important to its success and progress.
A gift of this size provides an institution like COH unique research capabilities. In addition to improvements in research technology and manpower, it also allows COH to pursue their research paths independent of NIH and JDRF oversight and regulation. Still, it is important to note that once COH trials reach human testing, it is subject to the same FDA oversight as all other institutions.
APPENDIX A: COH CLINICAL TRIALS
1. N2-(1-carboxyethyl)-2'Deoxyguanosine (CEdG) a Potential Biomarker for Diabetes
- The purpose of this study is to try and identify CEdG as a potential biomarker for diabetes. (T1 & T2)
2. Islet Transplantation Alone (ITA) in Patients With Difficult to Control Type I Diabetes Mellitus Using a Glucocorticoid-free Immunosuppressive Regimen
- Active, not recruiting
- The purpose of this study is to evaluate the safety and effectiveness of islet cell transplantation.
3. Islet Cell Transplant for Type 1 Diabetes
- The purpose of this study is to determine if islet cell transplantation using ATG, along with additional medications to prevent the body from rejecting the transplanted cells, is a safe and effective treatment for type 1 diabetes.