New gene therapy for Heart Failure shows promise

A new option for combating Heart Failure (or Congestive Heart Failure, also known as CHF) is showing promise as the CUPID Study

Heart Failure occurs with the heart muscle begins to wear out and lose its elastic properties. It isn’t a Congenital Heart Defect (CHD) itself, but a CHD can trigger it as a patient becomes older. And since I have CHF, I’m interested in keeping an eye on it!

The normal heart contains a protein that has a long medical name that I can’t say and can barely type. Thankfully, it is also known as SERCA2a. In a heart going through Heart Failure, the levels of  SERCA2a begin to drop. All is OK for a while, but it is like driving a car without ever checking the oil. You can take care of yourself, eat right, exercise, and write a letter to your mother every week, but if your heart isn’t producing SERCA2a then you’ve got a real problem on your hands. Eventually the CHF wins.

You can’t drink this stuff or take it in pill form. Instead, the gene that makes this protein is inserted into a virus. Not a virus that can make you sick, but acts as a transport mechanism for the gene. The virus (which in its commercial form is called MYDICAR) is inserted directly into the heart by catheter. In the study, there were 39 patients in total and the ones receiving the drug received low, medium, or high doses. And while there were no “adverse outcomes” the results were rather strange. Sometimes patients had the best response to lower doses of the enzyme. That really doesn’t make any sense.

It does look promising, if they can figure out why some people respond better to lower doses of the therapy. But this was a Phase 2 clinical trial studying a limited number of patients. We’ve got a few years to wait before this becomes available.

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One Response to “New gene therapy for Heart Failure shows promise”

  1. Leslie Shipley Says:

    For Immediate Release November 9, 2010
    NEW Gene Therapy for Patients with Congestive Heart Failure 
    A San Diego firm, Renova Therapeutics, announces that researchers at the VA San Diego Healthcare System have initiated clinical trials testing an exciting and promising gene therapy that has the potential to enhance the quality of life for patients with Congestive Heart Failure (CHF).
    CHF is a condition where the heart is unable to pump sufficiently to provide the body with enough blood flow to match its needs.  There are several causes of CHF, but the prognosis is dismal.  Fifty percent of those who have this disease die within 4-5 years.  That’s worse than the mortality rate for most cancers.  Currently there are more than 5 million Americans with this fatal illness.  
    “Based on our preclinical studies, we are hopeful that this novel therapy will someday make a difference in the lives of patients with CHF,” said Dr Kirk Hammond, who leads this research. Dr Hammond is a cardiologist at the VA San Diego Healthcare System and a Professor of Medicine at the University  of California, San Diego. He has worked to find innovative ways to improve the quality of life for people with heart disease since 1987.
    Dr. Hammond’s research has focused on developing gene therapy for CHF in order to increase the heart’s ability to pump blood to meet the body’s needs.  Normal hearts respond to the “flight and fright” hormones called catecholamines by increasing the pumping strength and rate of the heart.  Result?  Cardiac output goes up.  Catecholamines exert their effects by interacting with special receptors on heart muscle cells.  This activates a molecule called AC which leads to increased cyclic AMP inside the cell.  Cyclic AMP causes the heart to beat stronger.   The failing hearts of CHF patients contain low levels of cAMP.   Dr. Hammond’s break-through discovery was that increasing the heart’s content of AC6 improves heart function, and that AC6 has multiple beneficial effects. Hammond and his group sequenced the human AC6 gene and patents subsequently issued. His laboratory has received more than $20 million from the National Institute of Health in support of his research leading up to this trial.
    Dr. Hammond reasoned that if the amount of AC in a failing heart could be increased, it would increase heart function.  The exploration of his hypothesis began with a series of experiments — first in heart muscle cells in a Petrie dish, then in mice and finally in pigs with heart failure.
    The effects seen in the pre clinical studies were robust.  Mice with cardiomyopathy (a type of CHF) showed increased heart function and lived as long as normal, healthy mice when AC6 was increased. In pigs, AC6 gene therapy led to substantial improvements in heart function and reduced dilation of the heart.  This dilation, or enlargement of the heart, is part of how the body compensates for the loss of the heart’s ability to pump blood sufficiently.
    Renova Therapeutics is the company that was formed to make the AC6 treatment available to patients with CHF, should the clinical trials prove safe and effective.  The leadership team at Renova has a depth of expertise in the biotechnology industry.  
    Jack W. Reich, Ph.D., is the CEO and co-founder of Renova Therapeutics. He is a local San Diegan who has played an instrumental role in the development of the biotech industry since the late 80’s, and has been involved with more than 30 biotech companies.  Dr. Reich, Dr Hammond and biotech attorney Craig Andrews co-founded the first cardiovascular gene therapy company called Collateral Therapeutics with Dr Hammond as the lead scientist and Dr Reich as Chairman and CEO.  This company went public on NASDAQ two years after its founding with no venture capital.  Subsequently, Collateral Therapeutics was sold to Schering AG in 2002 in a stock-for-stock transaction at a large premium to market price, one of the highest premiums ever paid for a public biotech company up to that time.
    Having been successful with Collateral Therapeutics, Hammond, Reich and Andrews have teamed again to form Renova Therapeutics.  Asked what lessons were most valuable over the past decades, Dr Reich observed:  
    “One of the biggest factors in determining the clinical success of a gene therapy is the ability to deliver it to a target organ. This is what has held back the field for many years.”   Dr. Hammond discovered a means of delivery of a pivotal gene that is now patented.  He did his original experiments using a common catheter that is used in routine cardiac catheterization.  Hammond simply attached a syringe to the end of the catheter and injected the gene therapy into the coronary arteries of the heart — and it worked!  That discovery is groundbreaking… not only for cardiovascular gene therapy, it has implications for the delivery of gene therapy to other organs as well. 
    AC6 Gene Therapy Update:  Active and retired military are invited to volunteer in the clinical trial. This clinical trial is underway at the VA San Diego Healthcare System, San Diego. Those persons who are active or retired military are invited to participate. They must be between 18 and 80 years old, and diagnosed with CHF. For more information call 858 642 3542 and ask about the AC6 study or go to  and click Participate in the AC6 Study.
    A second clinical trial site has recently opened at the William Beaumont Hospital in Royal Oak, Michigan, which is open to all subjects and is not limited to military.
                                                                    Press contact:
                                                                    Leslie Shipley
                                                                    Renova Therapeutics
                                                                    11452 El Camino Real
                                                                    San Diego, CA 92130
                                                                    mobile: 858 634 0113

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