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 NameInstitutionGrant TitleGrant TypeAward Amount
Katy DigovichMinutia, Inc.Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patientsQuest – Discovery Stage Research Projects$1,198,550
Ronald Mark EvansSalk Institute for Biological StudiesExtending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1DQuest – Discovery Stage Research Projects$1,523,285
Prof. Song LiUniversity of California, Los AngelesiPSC Extracellular Vesicles for Diabetes TherapyQuest – Discovery Stage Research Projects$1,354,928
Dr Eiji Yoshihara PhDLundquist Institute for Biomedical Innovation at Harbor – UCLA Medical CenterDual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetesQuest – Discovery Stage Research Projects$250,000
Ronald Mark EvansSalk Institute for Biological StudiesTherapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 DiabetesQuest – Discovery Stage Research Projects$1,637,209
Dr. Shuvo RoyUniversity of California, San FranciscoSilicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatmentQuest – Discovery Stage Research Projects$1,113,000
Dr. Alan D. AgulnickViaCyte, Inc.Preclinical development of an immune evasive islet cell replacement therapy for type 1 diabetesQuest – Discovery Stage Research Projects$1,470,987
Dr Yang XuUniversity of California, San DiegoDevelopment of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCsQuest – Discovery Stage Research Projects$1,924,791
Dr. Julie B SneddonUniversity of California, San FranciscoDesigning a cellular niche for transplantation of human embryonic stem cell-derived beta cellsQuest – Discovery Stage Research Projects$2,006,076
Dr. Tejal Ashwin DesaiUniversity of California, San FranciscoThin Film Encapsulation Devices for Human Stem Cell derived Insulin Producing CellsQuest – Discovery Stage Research Projects$1,092,063
Dr. Senta GeorgiaChildren’s Hospital of Los AngelesDeveloping a personalized approach to beta cell replacement for patients with a genetic form of diabetesInception – Discovery Stage Research Projects$179,995
Walter ParkStanford UniversityEnhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus TherapyTherapeutic Translational Research Projects$6,054,165
Dr. Peter Cawood ButlerUniversity of California, Los AngelesPersonalized Cell Therapy for DiabetesTherapeutic Translational Research Projects$1,494,896
Dr. Felicia PagliucaSemma TherapeuticsPersonalized Cell Therapy for DiabetesTherapeutic Translational Research Projects$597,333
Dr. Everett H. MeyerStanford UniversityCellular Immune Tolerance SymposiumConference II$31,225
JDRF Esther LatresJDRF InternationalJDRF Encapsulation Consortium Fall 2017 MeetingConference II$42,425
Peter StockUniversity of California, San FranciscoPancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site: PARADIGMClinical Trial Stage Projects$11,083,012
Dr. William SietsemaCaladrius BiosciencesPhase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes MellitusClinical Trial Stage Projects$8,175,946
Manasi JaimanViaCyte, Inc.Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetesClinical Trial Stage Projects$13,607,002
Dr. Tim KiefferViaCyte, Inc.Stem cell-derived islet cell replacement therapy with immunosuppression for high-risk type 1 diabetesLate Stage Preclinical Projects$3,544,721
Dr. Roslyn Rivkah IsseroffUniversity of California, DavisScaffold for dermal regeneration containing pre-conditioned mesenchymal stem cells to heal chronic diabetic woundsPreclinical Development Awards$4,620,144
Howard FoytViaCyte, Inc.Clinical Development of a Cell Therapy for DiabetesAccelerated Development Pathway I$8,783,852
Richard JoveCity of Hope, Beckman Research InstituteInnovation and Translational Stem Cell Therapy for Diabetes and Neurological Diseases: Paving the way for real life solutionsConference$14,878
Fouad R KandeelCity of Hope, Beckman Research Institute2012 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes ResearchConference$15,000
Fouad R KandeelCity of Hope, Beckman Research Institute2011 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes ResearchConference$15,000
Fouad R KandeelCity of Hope, Beckman Research Institute2013 Rachmiel Diabetes and Obesity Levine Symposium – Advances in Diabetes ResearchConference$27,750
Fouad R KandeelCity of Hope, Beckman Research Institute2009 Rachmiel Levine Diabetes and Obesity Symposium: Advances in Diabetes Biology, Immunology and Cell TherapyConference$15,000
Dr. Jeffrey A BluestoneUniversity of California, San FranciscoA CIRM Disease Team for the Treatment and Cure of DiabetesDisease Team Planning$55,000
Dr. David Anthony TirrellCalifornia Institute of TechnologyEngineered matrices for control of lineage commitment in human pancreatic stem cellsBasic Biology V$526,896
Dr. Mark S. AndersonUniversity of California, San FranciscoGeneration of a functional thymus to induce immune tolerance to stem cell derivativesBasic Biology V$1,191,000
Howard FoytViaCyte, Inc.Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetesStrategic Partnership I$9,475,070
Maike SanderUniversity of California, San DiegoDeciphering transcriptional control of pancreatic beta-cell maturation in vitroBasic Biology IV$1,258,560
Dr. Charles C. King Dr.University of California, San DiegoBiological relevance of microRNAs in hESC differentiation to endocrine pancreasBasic Biology III$1,313,649
Dr. Olivia G. KellyViaCyte, Inc.Methods for detection and elimination of residual human embryonic stem cells in a differentiated cell productEarly Translational I$5,405,397
Dr. Yang XuUniversity of California, San DiegoDeveloping induced pluripotent stem cells into human therapeutics and disease modelsEarly Translational I$5,165,028
Dr. Jeffrey A BluestoneUniversity of California, San FranciscoStem cell tolerance through the use of engineered antigen-specific regulatory T cellsTransplantation Immunology$1,152,768
Dr. Evert KroonViaCyte, 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
Dr. Didier Y StainierUniversity of California, San FranciscoEndodermal differentiation of human ES cellsSEED Grant$611,027
Allan RobinsViaCyte, Inc.Cell Therapy for DiabetesDisease Team Research I$0
Total:
$98,854,699.64

CIRM Diabetes Videos

Diabetes: Progress toward stem cell therapies, a Live Google Hangout

Ron Piran, Sanford-Burnham – CIRM Stem Cell #SciencePitch

Eugene Brandon, Viacyte, Inc. – CIRM Stem Cell #SciencePitch

Diabetes: Progress and Promise in Stem Cell Research

Diabetes: Advancing Stem Cell Therapies – 2011 CIRM Grantee Meeting

Alan Lewis Talks About Getting an Embryonic Stem Cell-Based Therapy to Patients

Spotlight on Type 1 Diabetes: Welcoming Remarks

Spotlight on Type 1 Diabetes: Seminar by Ed Baetge, Ph.D.

Spotlight on Type 1 Diabetes: Seminar by Peter Butler, M.D.

Spotlight on Type 1 Diabetes: Seminar by Joelle Johnson

Stem Cell Based Diabetes Treatment: Alan Lewis – CIRM Science Writer’s Seminar

Resources

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