Gene Therapy Proves Effective in Treating Severe Heart Failure; Holds Potential to Drastically Reduce Healthcare Costs for Heart Failure Patients

/PRNewswire/ -- The 14th Annual Scientific Meeting of the Heart Failure Society of America (HFSA) will feature a discussion titled "Latest Developments in Stem Cell and Gene Therapy in Heart Failure" which includes a presentation by Dr. Roger Hajjar, Director of the Cardiovascular Research Institute, one of the 12 translational science institutes at The Mount Sinai Medical Center in New York. Dr. Hajjar's discussion will focus on the injection of a gene into patients with advanced heart failure to reverse the debilitating and life-threatening condition.

Over ten years, Dr. Hajjar and his team have validated the cardiac sarcoplasmic reticulum calcium ATPase pump, SERCA2a, as a target in heart failure and developed methodologies for cardiac-directed gene transfer. This work has led to the initiation and recent completion of phase 1 and phase 2 First-in-Man clinical trials of SERCA2a gene transfer in patients with advanced heart failure.

Patients treated with high dose therapy have shown 90 percent risk reduction for heart failure-related cardiovascular events such as significantly worsening health, the need for a transplant or cardiovascular device support, intravenous treatment or death.

"The patients receiving this gene therapy have shown marked improvements," said Dr. Hajjar. "Through our tests we've observed heart failure patients' quality of life improves greatly for significantly less cost than traditional therapies."

Patients treated with the gene therapy treatment may result in a large decrease in personal health care costs across their trial period. In nine months, individuals treated with the trial's placebo spent an average of $27,118 on health care, paying for expenses such as hospital stays, emergency medicine, and home care. Comparatively, in the same period of time, individuals treated with gene therapy spent an average of $329 on health care.

"Gene Therapy is a breakthrough in the treatment of heart failure patients that holds the potential to reverse the disease while also making treatment and recovery more affordable than ever," said Dr. Douglas Mann, HFSA President. "It is critical for the medical community to continue to integrate science and clinical medicine so biomedical research can improve patient care."

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