Diabetes Fact Sheet

CIRM funds many projects seeking to better understand diabetes and to translate those discoveries into new therapies.

Description

Diabetes comes in two forms: type 1 (also known as juvenile) and type 2 (also known as adult). Approximately 1.25 million people in the U.S. have type 1 diabetes, which is the form primarily being targeted by stem cell research.

Type 1 diabetes is an autoimmune disorder where the body’s own immune system destroys the cells in the pancreas that make the hormone insulin. Insulin normally circulates in the bloodstream after a meal and allows cells of the body to take up sugar and use it for food. Without insulin, cells starve and the sugar builds up in the bloodstream where it can damage the kidneys, blood vessels and retina.

Any potential cure for type 1 diabetes requires replacing the lost insulin-producing cells of the pancreas. Currently, the only cells that can be used for such a transplant come from donated organs, which are in short supply. Such insulin-producing cell transplants are also risky because the cells can be rejected by the recipient’s body if they don’t receive immune suppressing drugs.

To solve the first problem, groups of CIRM-funded researchers have developed methods to make replacement insulin-producing cells derived from human embryonic stem cells, which can be grown in large amounts. Implanted into mice and rats these cells are able to regulate blood sugar.

To get around the problem of rejection, CIRM-funded teams have placed donor progenitor cells in a device that implants under the skin and shields the cells from the patient's immune system. Other groups are studying how to regulate the immune system to make stem cell-derived transplants safer.

Clinical Stage Programs

UC San Francisco

Transplantation of beta cells, contained in donor pancreatic islets, can reverse the symptoms of diabetes.  However, due to a poor islet survival rate, transplants require islets from multiple donors.  Since islet cells are transplanted directly into the vessels that enter the liver, it is extremely difficult to monitor and retrieve these cells should the need arise. Peter Stock and his team at UCSF are using parathyroid glands to aid in the success and viability of the transplant procedure. Co-transplantation of islets and parathyroid glands, from the same donor, substantially increases beta cell survival, potentially enabling adequate long-term insulin production and removing the need for multiple donors.  The co-transplantation will occur in the patient’s forearm, which allows for easier monitoring and improves the effectiveness and accessibility of islet transplants for patients.

Caladrius Biosciences

Caladrius is targeting the immune system as an alternative strategy for treating patients with type 1 diabetes. This disease causes the immune system to destroy the insulin-producing cells of the pancreas. The team is developing a stem cell-based therapy using the patient's own cells. They will take cells, called regulatory T cells (Tregs), from the patient’s own immune system, expand the number of those cells in the lab and enhance them to make them more effective at preventing the autoimmune attack on the insulin-producing cells.

ViaCyte

ViaCyte is developing cell therapies to replace lost beta cells for people with type 1 diabetes (T1D). The therapies are derived from human embryonic stem cells, which are partially matured into becoming pancreatic tissues (the type destroyed in T1D). The cells are inserted into a small pouch that is transplanted under the patient’s skin. The transplanted cells will develop into fully matured beta cells that secrete the hormone insulin, which is needed to keep blood sugar levels at a healthy level. CIRM is funding ViaCyte’s two Phase 1/2 trials testing different product candidates. The first product, VC-01, encapsulates the cells and protects them from the patient’s immune system. The second product, VC-02, allows the patient’s blood vessels to make direct contact with the implanted cells. VC-02 is being developed for patients with high-risk T1D.

Learn More:

Progress and Promise in Diabetes Research

CIRM Grants Targeting Diabetes

Researcher name Institution Grant Title Grant Type Award Amount
Maike Sander University of California, San Diego Deciphering transcriptional control of pancreatic beta-cell maturation in vitro Basic Biology IV $1,258,560
Richard Jove City of Hope, Beckman Research Institute Innovation and Translational Stem Cell Therapy for Diabetes and Neurological Diseases: Paving the way for real life solutions Conference $14,878
Howard Foyt ViaCyte, Inc. Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes Strategic Partnership I $9,475,070
Douglas Losordo Caladrius Biosciences Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus Clinical Trial Stage Projects $8,175,946
Mark Anderson University of California, San Francisco Generation of a functional thymus to induce immune tolerance to stem cell derivatives Basic Biology V $1,191,000
Esther Latres JDRF International JDRF Encapsulation Consortium Fall 2017 Meeting Conference II $42,425
David Tirrell California Institute of Technology Engineered matrices for control of lineage commitment in human pancreatic stem cells Basic Biology V $526,896
Howard Foyt ViaCyte, Inc. Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes Clinical Trial Stage Projects $19,752,463
Jeffrey Bluestone University of California, San Francisco A CIRM Disease Team for the Treatment and Cure of Diabetes Disease Team Planning $55,000
Yang Xu University of California, San Diego Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs Quest - Discovery Stage Research Projects $1,960,560
Howard Foyt ViaCyte, Inc. Clinical Development of a Cell Therapy for Diabetes Accelerated Development Pathway I $8,783,852
Alan Agulnick ViaCyte, Inc. Preclinical development of an immune evasive islet cell replacement therapy for type 1 diabetes Quest - Discovery Stage Research Projects $1,470,987
Roslyn Isseroff University of California, Davis Scaffold for dermal regeneration containing pre-conditioned mesenchymal stem cells to heal chronic diabetic wounds Preclinical Development Awards $4,620,144
Shuvo Roy University of California, San Francisco Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatment Quest - Discovery Stage Research Projects $1,113,000
Felicia Pagliuca Semma Therapeutics Personalized Cell Therapy for Diabetes Therapeutic Translational Research Projects $597,333
Peter Butler University of California, Los Angeles Personalized Cell Therapy for Diabetes Therapeutic Translational Research Projects $1,494,896
Senta Georgia Children's Hospital of Los Angeles Developing a personalized approach to beta cell replacement for patients with a genetic form of diabetes Inception - Discovery Stage Research Projects $179,995
Ronald Evans Salk Institute for Biological Studies Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes Quest - Discovery Stage Research Projects $1,637,209
Allan Robins ViaCyte, Inc. Cell Therapy for Diabetes Disease Team Research I $22,999,933
Kevin D'Amour ViaCyte, Inc. Stem cell-derived islet cell replacement therapy with immunosuppression for high-risk type 1 diabetes Late Stage Preclinical Projects $3,544,721
Peter Stock University of California, San Francisco Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site: PARADIGM Clinical Trial Stage Projects $11,083,012
Didier Stainier University of California, San Francisco Endodermal differentiation of human ES cells SEED Grant $611,027
Tejal Desai University of California, San Francisco Thin Film Encapsulation Devices for Human Stem Cell derived Insulin Producing Cells Quest - Discovery Stage Research Projects $1,092,063
Yang Xu University of California, San Diego Developing induced pluripotent stem cells into human therapeutics and disease models Early Translational I $5,165,028
Julie Sneddon University of California, San Francisco Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells Quest - Discovery Stage Research Projects $2,006,076
Olivia Kelly ViaCyte, Inc. Methods for detection and elimination of residual human embryonic stem cells in a differentiated cell product Early Translational I $5,405,397
Fouad Kandeel City of Hope, Beckman Research Institute 2009 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes Biology, Immunology and Cell Therapy Conference $15,000
Evert Kroon ViaCyte, Inc. Development of the Theracyte Cellular Encapsulation System for Delivery of human ES Cell-derived Pancreatic Islets and Progenitors. Tools and Technologies I $827,072
Fouad Kandeel City of Hope, Beckman Research Institute 2013 Rachmiel Diabetes and Obesity Levine Symposium - Advances in Diabetes Research Conference $27,750
Jeffrey Bluestone University of California, San Francisco Stem cell tolerance through the use of engineered antigen-specific regulatory T cells Transplantation Immunology $1,152,768
Fouad Kandeel City of Hope, Beckman Research Institute 2011 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes Research Conference $15,000
Charles King University of California, San Diego Biological relevance of microRNAs in hESC differentiation to endocrine pancreas Basic Biology III $1,313,649
Fouad Kandeel City of Hope, Beckman Research Institute 2012 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes Research Conference $15,000
Total:
$117,623,710.00

CIRM Diabetes Videos

Resources

Find Out More:
Stem Cell FAQ | Stem Cell Videos | What We Fund