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PENN Medicine Clinical Trials

The latest news from the University of Pennsylvania School of Medicine and Health System.

Copyright: 2008, The Trustees of the University of Pennsylvania

Penn Study Verifies Human Gene Therapy in Model of Rare Metabolic Disorder

Thu, 28 Jul 2016 17:15:00 GMT

In an ongoing preclinical program using gene therapy to help cells restore normal levels of IDUA, researchers from the Perelman School of Medicine at the University of Pennsylvania have shown that exposure to the human IDUA protein early in the life of an MPS I canine model increased immune tolerance to the foreign gene.

Penn Medicine to Develop the Next Generation of Viral Vectors -- called AAV 3.0™ -- for Gene Therapies and Genome Editing

Wed, 29 Jun 2016 14:45:00 GMT

The Perelman School of Medicine at the University of Pennsylvania has launched a new program, called AAV 3.0™, to create new viral vectors to find quicker and better treatments for an array of diseases.

Drug Candidate Stops Extra Bone Growth in Animal Model of Rare, Genetic Disease

Wed, 13 Apr 2016 14:45:00 GMT

New preclinical research provides support to a drug that has been repurposed to possibly treat a rare and extremely disabling genetic bone disease, particularly in children. In that disease, fibrodysplasia ossificans progressiva (FOP), a mutation triggers bone growth in muscles, alters skeletal bone formation, and limits motion, breathing, and swallowing, among a host of progressive symptoms.

"Gene Fusion" Mutation Uses Three-Way Mechanism To Drive Childhood Brain Cancers

Tue, 02 Feb 2016 16:45:00 GMT

A powerful, three-way mechanism by which a mutation drives the growth of childhood brain cancers, was discovered by scientists from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia (CHOP).

Two-Hit Therapy for Breast Cancer Tumors Using Approved Drugs Looks Promising in Animal Study, Penn Study Finds

Wed, 07 Oct 2015 13:30:00 GMT

Disabling a cancer-causing pathway and administering an immune-molecule-based mop-up therapy eradicated a specific type of breast tumor in mice, according to researchers in the Perelman School of Medicine at the University of Pennsylvania.

Penn-developed, DNA-based Vaccine Clears Nearly Half of Precancerous Cervical Lesions in Clinical Trial

Wed, 30 Sep 2015 13:30:00 GMT

Using a novel synthetic platform for creating vaccines originally developed in the laboratory of David Weiner, PhD, a professor of Pathology and Laboratory Medicine from the Perelman School of Medicine at the University of Pennsylvania, a team led by his colleagues at the Johns Hopkins University School of Medicine, has successfully eradicated precancerous cervical lesions in nearly half of the women who received the investigational vaccine in a clinical trial.

Penn Medicine Researchers Receive $2 Million Grant from the American Heart Association

Fri, 17 Jul 2015 15:00:00 GMT

Daniel J. Rader, MD, chair of the Department of Genetics at the Perelman School of Medicine at the University of Pennsylvania, and Danish Saleheen, MD, PhD, an assistant professor of Biostatistics and Epidemiology, have been awarded a four-year, $2 million 2015 Grand Challenge Award from the American Heart Association (AHA).

Vision-Restoring Gene Therapy Also Strengthens Visual Processing Pathways in Brain, According to Penn Study

Wed, 15 Jul 2015 18:00:00 GMT

Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia (CHOP), have found evidence that this sight restoration leads to strengthening of visual pathways in the brain, published this week in Science Translational Medicine.

Invitation to Cover: Balancing Act: Conflict of Interest and Scientific Discovery

Wed, 27 May 2015 13:15:00 GMT

How can academic medical centers best manage the emerging issue of conflict of interest among scientists and physicians working to develop the next generation of treatments and cures? How do academic conflict of interest policies affect the process of scientific discovery? Exploring these questions and related issues will be at the heart of the "Conflict of Interest and Scientific Discovery" symposium organized by the Institute for Translational Medicine and Therapeutics at the University of Pennsylvania's Perelman School of Medicine. 

NIH Awards $16 Million to Penn-led Group to Develop Synthetic DNA Vaccines to Fight HIV

Thu, 12 Mar 2015 18:00:00 GMT

The National Institutes of Allergy and Infectious Diseases has awarded $16 million over the next five years for a collaborative study led by scientists from the Perelman School of Medicine at the University of Pennsylvania.

Eczema Medication Unlikely to Increase Risk of Cancer in Children, Penn Team Finds

Thu, 19 Feb 2015 13:15:00 GMT

The topical eczema medicine pimecrolimus appears unlikely to be associated with an increased risk of cancer in children, based on a group of children who were followed for 10 years, according to study published online this week in JAMA Dermatology.

Classification of Gene Mutations in a Children's Cancer May Point to Improved Treatments

Mon, 10 Nov 2014 16:00:00 GMT

Oncology researchers studying gene mutations in the childhood cancer neuroblastoma are refining their diagnostic tools to predict which patients are more likely to respond to drugs called ALK inhibitors that target such mutations.

First Atlas of Body Clock Gene Expression Informs Timing of Drug Delivery

Mon, 27 Oct 2014 19:00:00 GMT

A new effort mapping 24-hr patterns of expression for thousands of genes in 12 different mouse organs – five years in the making – provides important clues about how the role of timing may influence the way drugs work in the body.

Penn Study Examines Power of Exercise to Prevent Breast Cancer

Thu, 02 Apr 2009 17:40:00 GMT

A new federally funded University of Pennsylvania School of Medicine study aims to learn whether women at high risk of breast cancer can use exercise to meaningfully reduce their risk of getting the disease. Building on evidence that reducing estrogen in the body reduces cancer risk, and that elite female athletes experience a drop in estrogen levels that often cause them to stop ovulating and menstruating, the WISER Sister trial will investigate two different levels of regular treadmill exercise as a possible intervention for breast cancer risk reduction.

Penn Scientists Test Novel Medication to Block Progression of Alzheimer’s Disease
Researchers at the University of Pennsylvania School of Medicine are conducting studies on an experimental medication to block nerve damage and inflammation in the brain that can lead to progressive memory loss and behavioral changes in people with Alzheimer’s disease. Current Alzheimer’s disease therapies focus on improving symptoms rather than attacking the root of the disease progression.

Gene Therapy Improves Vision in Patients with Congenital Retinal Disease
In a clinical trial at The Children’s Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.

Zilver Trial Aims to Offer High-Tech Relief for Peripheral Arterial Disease
University of Pennsylvania School of Medicine researchers are the first in the Philadelphia region to begin using an innovative new drug-eluting stent to treat patients with peripheral arterial disease, the clogging and hardening of arteries that supply blood to the legs and feet.

Study Investigating Vaccine to Treat Brain Tumors Underway
Researchers at the University of Pennsylvania School of Medicine and the Abramson Cancer Center have begun ACT III – a Phase II/III Randomized Study – to investigate the addition of CDX-110 vaccine to standard care maintenance chemotherapy in patients with newly diagnosed glioblastoma multiforme (GBM), the most aggressive form of primary brain tumor.

Researchers at Penn Study New Airway Bypass Treatment
Researchers at the University of Pennsylvania School of Medicine are now studying an investigational treatment that may offer a significant new, minimally-invasive option for those suffering from advanced widespread emphysema. The EASE (Exhale Airway Stents for Emphysema) trial focuses on a procedure called airway bypass that involves creating pathways in the lung for trapped air to escape -- and in turn, may relieve emphysema symptoms including shortness of breath.

New Clinical Trial for Deadly Brain Tumors
Physicians initially diagnosed Phil Marfuta, 28, with tension headaches, which seemed reasonable to him since he is a busy graduate student studying physics at Princeton University. However, as the days went on his headaches did not subside, and when a CT scan and an MRI revealed two tumors, Phil underwent emergency surgery at the Hospital of the University of Pennsylvania. One of Phil’s tumors was a grade IV glioblastoma multiforme (GBM), which is the most aggressive form of primary brain tumor. Typically once diagnosed, the median survival time for a patient with a GBM is 12 months.

Fighting HIV With HIV: New Gene Therapy Vector Shows Promise
A protein with the ironic name 'Srcasm' can counteract the effects of tumor-promoting molecules in skin cells, according to new research by investigators at the University of Pennsylvania School of Medicine. Using animal models, the researchers discovered that Srcasm acts like a brake in epithelial cells, preventing uncontrolled cell growth caused by a family of proteins called Src kinases. This finding, published online in the Journal of Biological Chemistry, suggests a target for future gene therapy to treat skin, head, neck, colon, and breast cancers.

Penn Health System in Pursuit of Lung Cancer Vaccine
Researchers at the University of Pennsylvania have initiated a clinical trial directed at developing a better treatment for lung cancer. For patients who undergo surgery for their lung cancer, the treatment involves taking the tumor that was removed and processing it to make a vaccine.

Penn Researchers Examine the Effects of Meditation on Early Cognitive Impairment
Researchers at the University of Pennsylvania School of Medicine are examining the effectiveness of meditation on early cognitive impairment. Once this new study is completed, the results could help answer lingering questions over whether or not stress-reducing techniques and mind exercises can lessen or even prevent cognitive decline.