August 02, 2013
Riya V. Anandwala

New Clinical Trial at SLU to Assess Gene Therapy in Patients with Poor Heart Function

ST. LOUIS — Saint Louis University has enrolled its first patients in a new clinical trial that will further assess the efficacy of gene therapy in patients with advanced heart failure.

Paul Hauptman, M.D., professor of
internal medicine at SLU is the
principal investigator of the study.

The new study, "CUPID 2b", follows the initial exploratory study, "CUPID" that enrolled 40 patients nationally and tested whether MYDICAR — a genetically-targeted regulatory protein replacement therapy — could repair heart muscle that was damaged by a prior heart attack or cardiomyopathy, a heart muscle disease. The new trial will enroll a total of 200 patients in 52 sites worldwide.

"This trial is the next important step in advancing gene therapy as a treatment for advanced heart failure," said Paul Hauptman, M.D., professor of internal medicine at Saint Louis University School of Medicine and principal investigator of the study. "With this new study we will now be able to assess whether the early impressive improvements seen in CUPID can be replicated in a much larger number of patients."

Michael Lim
Michael Lim, M.D., director of the
division of cardiology at SLU,
performs the catheterization

The therapy involves infusing a gene — a type of regulatory protein called SERCA-2a — down the coronary arteries that may help the heart cells better utilize calcium, which is similar to an engine getting better gas mileage. This outpatient procedure is performed in a cardiac catheterization lab and aims to increase the level of SERCA-2a, which is reduced in the individual heart muscle cells in patients with heart failure and can thereby change the way those cells function

"This study provides a truly novel therapy to help the heart improve its ability to function for patients who are suffering from symptoms related to poor heart function," said Michael Lim, M.D., who serves as director of the division of cardiology at Saint Louis University and performs the catheterization procedure.

"A patient will undergo a heart catheterization procedure to have the therapy delivered, which will not be any different from a catheterization procedure that they have received before."

SLU is the only medical center in the region and one of only 33 in the nation that is participating in the study. To participate in the trial, please contact the Cardiology Research Office at Saint Louis University at 314-577-8876.

Established in 1836, Saint Louis University School of Medicine has the distinction of awarding the first medical degree west of the Mississippi River. The school educates physicians and biomedical scientists, conducts medical research, and provides health care on a local, national and international level. Research at the school seeks new cures and treatments in five key areas: infectious disease, liver disease, cancer, heart/lung disease, and aging and brain disorders.

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