MDNews - Cleveland-Akron-Canton

July/August 2015

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serum levels of D4A. D4A levels varied among patients, however, sug- gesting that individuals may differ in their metabolism of Abi to D4A. "More studies are needed to uncover the exact mechanisms involved, but we predict that direct treatment with D4A could prolong survival in some patients with metastatic prostate cancer," says Dr. Sharifi. "Further studies will also help us develop a potential biomarker profile to predict which patients will respond to D4–Abi." Dr. Sharifi holds positions in Cleveland Clinic's Lerner Research Institute, Glickman Urological & Kidney Institute, and Taussig Cancer Institute, and is the Kendrick Family Endowed Chair for Prostate Cancer Research. This research was funded by the Prostate Cancer Foundation, the American Cancer Society, the Department of Defense (U.S. Army Medical Research and Material Command), the Howard Hughes Medical Institute and the National Cancer Institute. GENE THERAPY IMPROVES VENTRICULAR TISSUE REPAIR IN HEART FAILURE PATIENTS Heart dysfunction years after a heart attack is the most common cause of heart failure. Once a patient develops heart failure, there are limited therapies that reverse or improve heart function. A team of researchers from 16 medical centers nationwide, and led by Summa Health's Marc S. Penn, MD, PhD, FACC, Director of Research for the Summa Health Cardiovascular Institute, and Founder and Chief Medical Officer for Juventas Therapeutics, Inc., have identified a gene-therapy treat - ment that, in a Phase II study demonstrates long-term, improved ventricular tissue repair in patients with high-risk heart failure. Known as STOP-HF, the Phase II trial was conducted in a random- ized, double-blind, placebo-controlled manner in 93 patients with symptomatic heart failure for one year after a single treatment. The majority of the enrollees were men in their mid-60s, who experienced their last heart attack 11 years prior to participation. Treatment was performed while patients were under conscious se - dation and involved 15 injections into the heart delivered via a cath- eter inserted through the groin. Patients received either placebo or a 15 or 30 mg dose of the investigational product — a DNA plasmid encoding stromal cell-derived factor-1 (SDF-1), into the peri-infarct region of the myocardium. Following the procedures and follow-up visits, cardiac function in each of the 93 participants was tested at four months. Researchers will present their analyses of the trial's primary endpoints — improvements in walking distance and quality of life — and additional analyses on the effects of drug therapy based on baseline heart function. Dr. Penn and his group have been working on the role of SDF-1 in tissue repair since publication in 2003 of their seminal findings in The Lancet. Since their early discovery, they have demonstrated that SDF-1 over-expression using gene therapy leads to the re - cruitment of the body's own stem cells to the site of tissue injury, thereby leading to stem cell-based repair and regeneration without the need to administer stem cells. The investigational product the team has developed leads to the expression of SDF-1 in the target tissue. To date, the studies have focused on patients with heart failure, critical limb ischemia and cosmetic wound healing following open-heart surgery. In addition to Summa Health's Cardiovascular Institute, other institutions participating in the trial included Linder Center at Christ Hospital, Cincinnati, Ohio; Baycare Health System, Clearwater, Florida; Iowa Heart, Des Moines, Iowa; Johns Hopkins, Baltimore, Maryland; University of Utah, Salt Lake City, Utah; University of Florida, Gainesville, Florida; Center for Therapeutic Angiogenesis, Birmingham, Alabama; Michigan CardioVascular Institute, Saginaw, Michigan; Minneapolis Heart Institute, Minneapolis, Minnesota; Montefiore Medical Center-Einstein Campus, New York, New York; Pepin Heart Institute, Tampa, Florida; Spectrum Health, Grand Rapids, Michigan; Washington University, St. Louis, Missouri; Columbia University, New York, New York; Hospital of University of Pennsylvania, Philadelphia, Pennsylvania; and Juventas Therapeutics, Inc., Cleveland, Ohio. ■ Marc S. Penn, MD, PhD, FACC National and international nominations are being sought for the third annual Harrington Prize for Innovation in Medicine, which honors a medical researcher for notable achievements in innovation, creativity and potential for clinical application. The Harrington Prize, which carries a $20,000 honorarium, is the result of a collaboration between The American Society for Clinical Investigation, one of the nation's oldest and most respected medical honor societies, and the Harrington Discovery Institute at University Hospitals in Cleveland, Ohio — part of The Harrington Project for Discovery & Development. The Harrington Discovery Institute is a nonprofit institute dedicated to enabling physician-scientists to transform their discoveries into therapies that enhance patient care. Nomination guidelines can be found at HarringtonDiscovery.org/ThePrize. NOMINATIONS NOW OPEN FOR HARRINGTON PRIZE FOR INNOVATION IN MEDICINE Deadline Is August 28, 2015 2 0 1 5 J U LY/AU G U S T ■ M D N E W S . CO M ■ MD NEWS Cleveland/Akron/Canton | 2 3

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