Washington, D.C., December 4, 2018  – The FDA has recently approved an investigational new drug clinical trial that will start shortly at MedStar Heart & Vascular Institute. The trial, called STEMVAD, is sponsored by MedStar Heart, in partnership with CardioCell, to determine whether stem cell therapy improves myocardial function in patients with severe heart failure—severe enough to require the implantation of a left ventricular assist device, or LVAD. The trial will use CardioCell’s proprietary mesenchymal stem cells (MSCs), and will pioneer the use of a novel delivery strategy, deriving from a new understanding of the causes of progressive heart failure and the mechanisms by which stem cells exert their beneficial cardiac effects.

Approximately six and a half million adult Americans have heart failure, of whom 200,000 to 250,000 are estimated to have end-stage heart failure, and are in need for heart transplantation. However, with the very low supply of donor hearts, LVADs are increasingly used. An LVAD is a small pump that helps circulate the patients’ blood when their heart becomes too weak to pump effectively on its own. Although highly effective in alleviating symptoms and improving longevity, patients with LVAD support still have a high incidence of serious complications. Importantly, persistent inflammation is also a probable major cause of clinical and cardiac deterioration in LVAD patients.

“Innovative therapies to improve heart function and outcomes of patients with advanced heart failure are sorely needed,” said Selma Mohammed, MD, PhD, director of the Heart Failure Research Program at MedStar Heart & Vascular Institute.  

Virtually all previous attempts to use stem cells to treat cardiac disease relied on the concept that the cells had to be delivered directly to the target tissue—the heart—and this, in turn, required catheter-based, invasive delivery strategies. This posed to the MedStar Heart & Vascular Institute investigators a potential major practical issue, as it was their belief that a single injection of stem cells would not lead to a “cure,” but that repeated injections—every few months—would be required for adequate and sustained treatment effect.

“We have developed compelling evidence, using mouse models of heart attack and of heart failure, that one of the major mechanisms leading to progressive myocardial dysfunction in patients with heart failure is the presence of persistent and inappropriate inflammation,” said Stephen Epstein, MD, director of Translational and Vascular Biology Research at MedStar Heart & Vascular Institute. “We also demonstrated not only that MSCs have marked anti-inflammatory effects, but that they exert these effects when injected intravenously. Most importantly, these anti-inflammatory effects led to a marked improvement of heart function.” 

These results led to the design of the study about to be initiated—which involves the first stem cell trial using repeated intravenous administration of MSCs every month to control the inflammation, and to sustain any improvements in heart function.

“The focus of this trial is on safety and feasibility. If successful, pivotal trials will follow to establish whether repeated infusions of MSCs are effective strategies for treating LVAD patients, resulting in improved symptoms and outcomes,” added Dr.  Mohammed. 

“If we are successful in showing intravenously delivered stem cells improve outcomes in LVAD patients, the results would likely extend to the general population of heart failure patients, and in the process, fundamentally transform current paradigms for treating heart failure,” concluded Dr. Epstein.

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 In addition to Drs. Mohammed and Epstein, the following individuals made critically important contributions to the successful initiation and completion of the research leading to this clinical trial:

• Dror Luger, PhD, research scientist, MedStar Heart & Vascular Institute, MedStar Washington Hospital Center
• Michael J. Lipinski, MD, PhD, former MedStar Heart & Vascular Institute research scientist, now with Cardiovascular Associates of Charlottesville, Sentara Martha Jefferson Hospital, Charlottesville, Va.
• Ron Waksman, MD, director, Cardiovascular Research and Advanced Education, MedStar Heart & Vascular Institute, MedStar Washington Hospital Center
• Sergey Sikora, PhD, president and CEO, CardioCell, LLC

Conflict of Interest Statement: Dr. Stephen Epstein is an equity holder in CardioCell, serves on its Board, and consults for the company.

About MedStar Heart & Vascular Institute:
MedStar Heart & Vascular Institute is a national leader in the research, diagnosis and treatment of cardiovascular disease. A network of 10 hospitals and 150 cardiovascular physicians throughout Maryland, Northern Virginia and the Greater Washington, D.C., region, MedStar Heart also offers a clinical and research alliance with Cleveland Clinic Heart & Vascular Institute, the nation’s #1 heart program. Together, they have forged a relationship of shared expertise to enhance quality, improve safety and increase access to advanced services. MedStar Heart & Vascular Institute was founded at MedStar Washington Hospital Center, home to the Nancy and Harold Zirkin Heart & Vascular Hospital. Opened in July 2016, the hospital ushered in a new era of coordinated, centralized specialty care for patients with even the most complex heart and vascular diagnoses.

So Young Pak
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