Heart Disease Fact Sheet

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

Description

Heart disease strikes in many forms, but collectively it causes one third of all deaths in the U.S. Many forms of heart disease have a common result—cardiomyopathy. While this is commonly called congestive heart failure (CHF), it is really just the heart becoming less efficient due to any number of causes, but the most common is loss of functioning heart muscle due to the damage caused by a heart attack. An estimated 4.8 million Americans have CHF, with 400,000 new cases diagnosed each year. Half die within five years.

Numerous clinical trials are underway testing a type of stem cell found in bone marrow, called mesenchymal stem cells or MSCs, to see if they are effective in treating the form of CHF that follows a heart attack. While those trials have shown some small improvements in patients the researchers have not found that the MSCs are creating replacement heart muscle. They think the improvements may be due to the MSCs creating new blood vessels that then help make the existing heart muscle healthier, or in other ways strengthening the existing tissue.

California’s stem cell agency has numerous awards looking into heart disease (the full list is below). Most of these involve looking for ways to create stem cells that can replace the damaged heart muscle, restoring the heart’s ability to efficiently pump blood around the body. Some researchers are looking to go beyond transplanting cells into the heart and are instead exploring the use of tissue engineering technologies, such as building artificial scaffolds in the lab and loading them with stem cells that, when placed in the heart, may stimulate the recovery of the muscle.

Other CIRM-funded researchers are working in the laboratory, looking at stem cells from heart disease patients to better understand the disease and even using those models to discover and test new drugs to see if they are effective in treating heart disease. Other researchers are trying to make a type of specialized heart cell called a pacemaker cell, which helps keep a proper rhythm to the heart’s beat.

We also fund projects that are trying to take promising therapies out of the laboratory and closer to being tested in people. In some cases, these awards also fund the early phase clinical trials to show that they are safe to use and, in some cases, show some signs of being effective.

Clinical Stage Programs

Capricor (Heart failure and Duchenne Muscular Dystrophy-related heart failure)

The company is using donor cells derived from heart stem cells developed by Cedars-Sinai to treat patients developing heart failure after a heart attack. In early studies the cells appear to reduce scar tissue, promote blood vessel growth and improve heart function.

In a second trial, the company is using the same donor cells derived from heart stem cells to treat patients developing heart failure due to Duchenne Muscular Dystrophy. In early studies the cells appear to reduce scar tissue, promote blood vessel growth and improve heart function.

Cedars-Sinai Medical Center (Cardiomyopathy)

This team developed a way to isolate some heart-specific stem cells that are found in adult heart muscle. They use clumps of cells called Cardiospheres to reduce scarring caused by heart attacks. Initially they used cells obtained from the patient’s own heart but they later developed methods to obtain the cells they need from donor organs, which allows the procedure to become an off-the-shelf-therapy, meaning it can be available when and where the patient needs it rather than having to create it new each time. The company, working with the Cedars-Sinai team, received FDA approval to begin a clinical trial in June 2012. 

Stanford School of Medicine (Heart Failure)

This team plans to turn embryonic stem cells into what are called cardiomyocytes, the kind of cells that can become heart muscle. They plan to develop methods for producing sufficient quantities for clinical therapy and to do all the laboratory work and preliminary testing needed to gain FDA approval of a clinical trial by the close of the grant. They are proposing to carry out a trial with patients who have disease that is so advanced that they are on a waiting list for heart transplants.

Video: Bruce Conklin of the Gladstone Institute of Cardiovascular Disease talks about using stem cells to screen drugs for heart side effects

CIRM Grants Targeting Heart Disease

Researcher name Institution Grant Title Grant Type Approved funds
Joseph Wu Stanford University Drug Discovery & Stem Cell Models for Cardiovascular Disease Conference Conference II $7,500
Jane Lebkowski Geron Corporation Preclinical Development and First-In-Human Testing of GRNCM1 in Advanced Heart Failure Disease Team Therapy Planning I $0
John Cashman Human BioMolecular Research Institute Discovering Potent Molecules with Human ESCs to Treat Heart Disease SEED Grant $688,274
Irving Weissman Stanford University Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs Tools and Technologies II $1,463,814
Deborah Lieu University of California, Davis Induction of Pluripotent Stem Cell-Derived Pacemaking Cells Basic Biology IV $1,333,689
Linda Cambier Cedars-Sinai Medical Center Exosomal Y-RNAs as mediators of bioactivity of cardiac-derived cell therapy Inception - Discovery Stage Research Projects $181,063
Huei-sheng Chen Sanford-Burnham Medical Research Institute Development of Neuro-Coupled Human Embryonic Stem Cell-Derived Cardiac Pacemaker Cells. SEED Grant $695,680
Deepak Srivastava Gladstone Institutes Mechanisms of Direct Cardiac Reprogramming Basic Biology III $1,572,380
Sheng Ding Gladstone Institutes A new paradigm of lineage-specific reprogramming Basic Biology IV $1,568,395
Phillip Yang Stanford University Activation of patient-specific endogenous myocardial repair through the exosomes generated from the hypoxic iPSC-derived cardiomyocytes (iCMs). Inception - Discovery Stage Research Projects $234,619
Michelle Khine University of California, Irvine Micro Platform for Controlled Cardiac Myocyte Differentiation SEED Grant $156,426
Ali Nsair University of California, Los Angeles Characterization and Engineering of the Cardiac Stem Cell Niche Basic Biology III $1,127,741
Eduardo Marbán Cedars-Sinai Medical Center Mechanism of heart regeneration by cardiosphere-derived cells Basic Biology IV $1,367,604
Arjun Deb University of California, Los Angeles Targeting progenitors in scar tissue to reduce chronic scar burden Inception - Discovery Stage Research Projects $230,400
Phillip Yang Stanford University In Vivo Molecular Magnetic Resonance Imaging of Human Embryonic Stem Cells in Murine Model of Myocardial Infarction SEED Grant $629,952
Farah Sheikh University of California, San Diego Molecular Mechanisms Underlying Human Cardiac Cell Junction Maturation and Disease Using Human iPSC Basic Biology III $1,341,955
Ali Nsair University of California, Los Angeles Human Induced Pluripotent Stem Cell-Derived Cardiovascular Progenitor Cells for Cardiac Cell Therapy. New Faculty Physician Scientist $2,316,894
Deepak Srivastava Gladstone Institutes Use of Human iPSC-derived Endothelial Cells for Calcific Aortic Valve Disease Therapeutics Quest - Discovery Stage Research Projects $2,400,048
Benoit Bruneau Gladstone Institutes Induction of cardiogenesis in pluripotent cells via chromatin remodeling factors New Faculty II $2,723,653
Sean Wu Stanford University Elucidating Molecular Basis of Hypertrophic Cardiomyopathy with Human Induced Pluripotent Stem Cells Basic Biology III $1,260,537
Reza Ardehali University of California, Los Angeles Preclinical evaluation of human embryonic stem cell-derived cardiovascular progenitors in a large animal model New Faculty Physician Scientist $2,930,388
Deborah Ascheim Capricor, Inc Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy Clinical Trial Stage Projects $3,376,259
Ching-Pin Chang Stanford University VEGF signaling in adventitial stem cells in vascular physiology and disease New Faculty II $3,005,695
John Laird University of California, Davis Phase I study of IM Injection of VEGF Producing MSC for the Treatment of Critical Limb Ischemia Disease Team Therapy Planning I $76,066
Joseph Wu Stanford University Tissue Collection for Accelerating iPSC Research in Cardiovascular Diseases Tissue Collection for Disease Modeling $1,291,832
Michael Lewis Cedars-Sinai Medical Center Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells Clinical Trial Stage Projects $7,354,772
Kara McCloskey University of California, Merced Building Cardiac Tissue from Stem Cells and Natural Matrices New Faculty II $1,656,083
Robert Robbins Stanford University Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure Disease Team Therapy Planning I $73,030
Thomas Novak Cellular Dynamics International Generation and characterization of high-quality, footprint-free human induced pluripotent stem cell lines from 3,000 donors to investigate multigenic diseases hiPSC Derivation $16,000,000
Deepak Srivastava Gladstone Institutes Direct Cardiac Reprogramming for Regenerative Medicine Quest - Discovery Stage Research Projects $2,400,048
Irving Weissman Stanford University Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells Comprehensive Grant $2,471,386
Deepak Srivastava Gladstone Institutes Direct Cardiac Reprogramming for Heart Regeneration Early Translational III $5,795,871
Deborah Requesens Coriell Institute for Medical Research The CIRM Human Pluripotent Stem Cell Biorepository – A Resource for Safe Storage and Distribution of High Quality iPSCs hPSC Repository $9,942,175
John Cashman Human BioMolecular Research Institute Improving Existing Drugs for Long QT Syndrome type 3 (LQT3) by hiPSC Disease-in-Dish Model Early Translational IV $6,361,369
Brian Black University of California, San Francisco Weinstein Cardiovascular Development Conference Conference $35,000
Randall Lee University of California, San Francisco Embryonic Stem Cell-Derived Therapies Targeting Cardiac Ischemic Disease Comprehensive Grant $2,424,353
Joseph Wu Stanford University Heart Repair with Human Tissue Engineered Myocardium Early Translational III $4,396,738
Christian Metallo University of California, San Diego Metabolic regulation of cardiac differentiation and maturation Basic Biology V $1,124,834
Alice Tarantal University of California, Davis 11th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases Conference $16,850
Harold Bernstein University of California, San Francisco Modeling Myocardial Therapy with Human Embryonic Stem Cells Comprehensive Grant $2,134,694
Walter Boyd University of California, Davis Extracellular Matrix Bioscaffold Augmented with Human Stem Cells for Cardiovascular Repair Early Translational III $4,631,754
Michelle Khine University of California, Merced Micro Platform for Controlled Cardiac Myocyte Differentiation SEED Grant $193,700
Alice Tarantal University of California, Davis 9th Annual Gene Therapy Symposium for Heart, Lung and Blood Diseases Conference $12,000
Robb Maclellan University of California, Los Angeles Human Cardiovascular Progenitors, their Niches and Control of Self-renewal and Cell Fate Basic Biology I $917,667
Christopher Zarins Stanford University Engineering a Cardiovascular Tissue Graft from Human Embryonic Stem Cells Comprehensive Grant $2,454,490
Eric Adler University of California, San Diego Identification of Novel Therapeutics for Danon Disease Using an iPS Model of the Disease Early Translational III $1,701,575
Todd McDevitt Gladstone Institutes Engineering microscale tissue constructs from human pluripotent stem cells Research Leadership $5,884,058
Alice Tarantal University of California, Davis 10th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases Conference $18,300
Eduardo Marbán Cedars-Sinai Medical Center Autologous cardiac-derived cells for advanced ischemic cardiomyopathy Disease Team Research I $5,560,232
Deepak Srivastava Gladstone Institutes microRNA Regulation of Cardiomyocyte Differentiation from Human Embryonic Stem Cells Comprehensive Grant $2,994,719
Yang Xu University of California, San Diego Human ES cell based therapy of heart failure without allogenic immune rejection Early Translational III $1,857,600
Michael Snyder Stanford University Center of Excellence for Stem Cell Genomics - Stanford Genomics Centers of Excellence Awards (R) $22,796,609
Joseph Wu Stanford University A Novel, Robust and Comprehensive Predictive Tool Using Human Disease-Specific Induced Pluripotent Stem Cells for Preclinical Drug Screening Tool Translational Research Projects $1,000,000
Bruce Conklin Gladstone Institutes Induced Pluripotent Stem Cells for Cardiovascular Diagnostics New Cell Lines $1,708,560
Mark Mercola Sanford-Burnham Medical Research Institute Chemical Genetic Approach to Production of hESC-derived Cardiomyocytes Comprehensive Grant $2,832,000
Rachel Smith Capricor, Inc Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction Disease Team Therapy Development - Research $14,405,857
Joseph Gold City of Hope Development of a scalable, practical, and transferable GMP-compliant suspension culture-based differentiation process for cardiomyocyte production from human embryonic stem cells. Tools and Technologies III $891,518
Sonja Schrepfer University of California, San Francisco Hypo-immunogenic cardiac patches for myocardial regeneration Inception - Discovery Stage Research Projects $238,500
Michael Longaker Stanford University Derivation and analysis of pluripotent stem cell lines with inherited TGF-b mediated disorders from donated IVF embryos and reprogrammed adult skin fibroblasts New Cell Lines $1,406,636
Patrick McDonough Vala Sciences, Inc. Optimization in the Identification, Selection and Induction of Maturation of Subtypes of Cardiomyocytes derived from Human Embryonic Stem Cells Tools and Technologies I $870,717
Joseph Wu Stanford University Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure Disease Team Therapy Development - Research $19,060,330
Shaochen Chen University of California, San Diego Development of 3D Bioprinting Techniques using Human Embryonic Stem Cells Derived Cardiomyocytes for Cardiac Tissue Engineering Tools and Technologies III $1,368,517
Kevin Healy University of California, Berkeley Human Cardiac Chip for Assessment of Proarrhythmic Risk Quest - Discovery Stage Research Projects $816,908
Sylvia Evans University of California, San Diego Specification of Ventricular Myocyte and Pacemaker Lineages Utilizing Human Embryonic Stem Cells SEED Grant $585,600
Mohammad Pashmforoush University of Southern California Transcriptional Regulation of Cardiac Pacemaker Cell Progenitors New Faculty I $2,816,578
Huei-sheng Chen Sanford-Burnham Medical Research Institute Studying Arrhythmogenic Right Ventricular Dysplasia with patient-specific iPS cells Basic Biology IV $1,579,250
Joseph Wu Stanford University Macaca mulatta as advanced model for predictive preclinical testing of engineered cardiac autografts and allografts Tools and Technologies III $1,689,744
Mark Mercola Stanford University Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI Quest - Discovery Stage Research Projects $1,809,234
Krishna Shenoy Stanford University Technology for hESC-Derived Cardiomyocyte Differentiation and Optimization of Graft-Host Integration in Adult Myocardium SEED Grant $572,891
Andrew Putnam University of California, Irvine A Novel Engineered Niche to Explore the Vasculogenic Potential of Embryonic Stem Cells New Faculty I $395,764
Benoit Bruneau Gladstone Institutes Epigenetic regulation of human cardiac differentiation Basic Biology IV $1,568,148
Total:
$198,807,523.00

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