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In a major step forward for the University of Miami’s growing regenerative medicine program, the Miller School of Medicine/UHealth is taking part in an international clinical trial to determine the effectiveness of genetically targeted enzyme replacement therapy for advanced heart failure.

“This is leading-edge 21st century medicine,” said Joshua M. Hare, M.D., Director of the Interdisciplinary Stem Cell Institute. “After decades of basic science research, we are close to proving that a painless single-time genetic treatment can reverse the course of cardiac disease in some patients.”

The gene therapy is designed to restore levels of an important enzyme called SERCA2a, which regulates the production of calcium ions needed for the contraction and relaxation of cardiac muscle cells. Numerous human studies have established a clear association between depleted SERCA2a enzyme in cardiac cells and the progression of end-stage heart failure.

Claudia A. Martinez, M.D., assistant professor of medicine and interventional cardiologist, infuses the investigational product via a standard heart catheterization through the groin or arm. “This is a painless minimally invasive procedure, and most of the patients go home after a few hours,” said Martinez, who performs the one-time outpatient infusion at University of Miami Hospital. Patients receive either the genetically targeted enzyme replacement therapy or a placebo. “We treated our first patient in July and our second patient in August,” she said.

Candidates for the clinical trial are advanced heart failure patients who continue to deteriorate from their disease despite optimal drug therapy. Prescreening blood tests are necessary to determine who is a candidate to receive this potential treatment.

Repairing this molecular defect in preclinical studies reversed the disease and restored cardiac function. “We know based on small studies that this regenerative approach works,” Martinez said. “Now, we are trying to prove that on a large scale.”

Hare, who is also Louis Lemberg Professor of Medicine and the Miller School’s Chief Sciences Officer, added that patients with advanced heart disease may also benefit from stem cell therapy that would restore production of the SERCA2a enzyme. “We now have two options on the table that could be applied separately or combined, depending on the most effective approach,” he said.

Emphasizing the critical relationship between funding basic scientific research and developing new clinical treatments, Hare said, “The growth of regenerative medicine is the result of more than 20 years of basic research. If those funds were to be curtailed, there would be a negative impact on the clinical side in coming decades. It has taken decades to unravel the science of how the heart beats, and to see that a lack of SERCA2a is a problem. Now, we are on the threshold of developing effective gene therapies for these conditions.”

For more information about the gene therapy trial, call the Interdisciplinary Stem Cell Institute at 305-243-9106.

 

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