Heart Failure


Heart disease strikes in many forms; collectively, heart disease causes one third of all deaths in the U.S. The prevalence of heart disease is widespread: one out of every three men, and one out of every four women, will develop heart disease in their lifetime. Many forms of heart disease have a common result: heart failure, or cardiomyopathy. Heart failure occurs when the heart becomes unable to pump blood efficiently to the body; millions of Americans suffer from this affliction. [1] One of the most common causes of heart failure is the loss of functioning heart muscle, due to damage caused by a heart attack. Damaged areas irreversibly turn into scar tissue after the initial attack; this scarring predisposes patients to suffer or die from consequences of their heart disease. Scarring leads to a worsening of general and cardiovascular health, as well as a diminishment in quality of life. [2]

An estimated 5.1 million Americans suffer from heart failure, with 400,000 new cases diagnosed each year. [3] [4] For victims of end-stage heart failure, the 2-year survival rate with standard medical therapy is only 50%; this dismal rate is worse than that of patients suffering from AIDS, liver cirrhosis, and stroke. [5]

Beyond the incalculable human costs of suffering associated with serious heart disease, heart failure costs the nation an estimated $32 billion each year. This total includes the cost of health care services, medications and lost productivity in term of missed days of work. [6]

Depending on the severity of a patient’s heart failure, the current standard of care involves device or drug therapy (e.g. pacemakers) or organ transplantation. Organ transplantation is extremely limited in terms of scalability; the U.S. faces a serious organ shortage, especially for vital organs such as hearts, kidneys and livers. Moreover, the recipients’ immune system will sometimes reject the donors’ hearts, leading to what is called Graft-versus-Host disease, which is often fatal. Stem cell therapies offer new hope to avoid these grave problems by transplanting cells, rather than whole organs.

Progress towards a cure

To date, CIRM has awarded more than $183 million in funding to heart disease research. [7]

One promising CIRM-funded research project at Stanford, led by Dr. Joseph Wu, is working to develop a stem cell therapy using embryonically-derived heart cells to treat victims of heart attack. The team intends to direct these embryonic stem cells—which can differentiate into any of the more than 200 cell types in the body, including heart muscle cells—to develop into healthy heart cells (called cardiomyocytes). If successful, transplantation of healthy cardiomyocytes could improve cardiac function following a heart attack.

Another CIRM-supported project, led by Dr. Eduardo Marbán at Cedars-Sinai Medical Institute, focuses on recovery following major heart attacks. Marbán’s team has developed an approach using patients’ own stem cells to regenerate scarred tissue caused by heart attack. These cells were used in a clinical trial called CADUCEUS, which demonstrated, for the first time, that therapeutic regeneration is possible after heart muscle is destroyed. In this trial, researchers used cells made from the patients’ own tissue (autologous); the process required only a simple procedure called a biopsy to take healthy tissue from the heart, isolate that tissue and use it to grow new cells. [8] These cells are known as CDCs (cardiosphere derived cells) and have been developed for clinical use over the course of many years. More than 100 peer-reviewed scientific publications ascertain the power of these cells to regenerate and heal damaged heart tissue.

Building on the foundation of Dr. Marbán’s work, along with the support of a $19.7 million CIRM Disease Team Grant, a team led by Dr. Rachel Smith and Michelle Kreke of Capricor Therapeutics, is conducting an ALLSTAR clinical trial. This trial represents the potential to take cell therapy to a new level, by developing an “off-the-shelf” CDC product, so patients can have quick and easy access to cells that already been manufactured using Good Manufacturing Practices (GMP) and tested for purity, potency and sterility. This trial is testing the same patient population as in the CADUCEUS trial: patients who have had a major heart attack—and persistent reductions in heart function as a result—and therefore are at greater risk of subsequent heart failure. The treatment is simple: the CDCs are delivered through single brief catheterization procedure, requiring a only local anesthetic and insertion of a tube (or catheter) into the affected blood vessel in the heart. [9] This procedure is performed hundreds of thousands of times per day across the USA to treat heart attack as it occurs. The overarching goal of this approach is to prevent patients who have had a heart attack from developing heart failure over time. Early-stage trial results indicate that this therapeutic approach has the potential to reduce scar tissue, and to promote generation of new, healthy heart tissue.

These trials began in June 2012; they have since moved into Phase 2 trials at many sites across the country. Some patients from the Phase 1 portion have already shown significant recovery. In January of 2014, Capricor signed a $337.5 million exclusive license option and collaboration agreement with Janssen Biotech, a division of pharmaceutical giant Johnson & Johnson. [10] This public-private collaboration increases the likelihood of continual, reliable funding for future trials, leveraging initial funding from CIRM.

Selected disease and research progress information provided by the California’s stem cell research funding agency, the California Institute for Regenerative Medicine (CIRM). Visit www.cirm.ca.gov for more updates.


[1] Smith, Rachel and Michelle Kreke.  “Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction.”  California Institute for Regenerative Medicine.  Web.  https://www.cirm.ca.gov/our-progress/awards/allogeneic-cardiac-derived-stem-cells-patients-following-myocardial-infarction

[2] Id.

[3] Go, AS et al. “Heart disease and stroke statistics—2013 update: a report from the American Heart Association.” Circulation. 2013;127:e6–e245.  http://circ.ahajournals.org/content/127/1/e6

[4] “Heart Disease Fact Sheet.”  California Institute for Regenerative Medicine.  Web.  Accessed 16 February 2016.  https://www.cirm.ca.gov/our-progress/disease-information/heart-disease-fact-sheet

[5] Wu, Joseph and Deepak Srivastava. “Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure.” California Institute for Regenerative Medicine.   Web.  Accessed 15 February 2016. https://www.cirm.ca.gov/our-progress/awards/human-embryonic-stem-cell-derived-cardiomyocytes-patients-end-stage-heart-0

[6] Heidenreich, PA et al. “Forecasting the future of cardiovascular disease in the United States: a policy statement from the American Heart Association.” 1 March 2011. Circulation. 2011;123(8):933–44.  http://www.ncbi.nlm.nih.gov/pubmed/21262990

[7] Author, No.  “Heart Disease Fact Sheet.”  California Institute for Regenerative Medicine.  Web.  Accessed 15 February 2016.  https://www.cirm.ca.gov/our-progress/disease-information/heart-disease-fact-sheet

[8] Id.

[9] Id.

[10] McCormack, Kevin.  “Heartening start to the New Year for stem cell heart therapy.”  6 January 2014.  California Institute for Regenerative Medicine.  Web.  Accessed 16 February 2016.  http://blog.cirm.ca.gov/2014/01/06/heartening-start-to-new-year-for-stem/


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