Drug Delivery

Chronic rejection of transplanted organs is the leading cause of transplant failure, and one that the field of organ transplantation has not overcome in the field to flourish.

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McGowan Institute for Regenerative Medicine affiliated faculty member and Duquesne University Associate Professor of Pharmaceutics Jelena Janjic, PhD, is working with a multidisciplinary team of researchers to explore the use of nanomedicine in organ transplantation and regenerative surgery.

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University of Pittsburgh School of Medicine scientists announced a potential vaccine against SARS-CoV-2, the new coronavirus causing the COVID-19 pandemic. When tested in mice, the vaccine, delivered through a fingertip-sized patch, produces antibodies specific to SARS-CoV-2 at quantities thought to be sufficient for neutralizing the virus.

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McGowan Institute for Regenerative Medicine affiliated faculty member Cecelia Yates, PhD, Associate Professor, Department of Health Promotion & Development, School of Nursing, University of Pittsburgh, with a secondary appointment in the Department of Pathology, School of Medicine, has been awarded $250,000 and support to accelerate her research through an ongoing partnership between global biotechnology leader CSL Behring and the University City Science Center.

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NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of genomic loci or secondary and tertiary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

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McGowan Institute for Regenerative Medicine affiliated faculty members Hang Lin, PhD, and Douglas Weber, PhD, received $1.5 million from the NIH to study the mechanism of osteoarthritis pain and test drugs in a novel microphysiological tissue chip (microJoint). The team will use the microJoint to also assess the impact of opioids on the neural activity and tissue health in the knee joint.

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More than 30 million Americans suffer from diabetes and must inject themselves with insulin two to four times daily. Researchers have been looking for ways to administer the drug orally, and researchers at Carnegie Mellon University (CMU) have now shown such a feat is possible.

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McGowan Institute for Regenerative Medicine affiliated faculty member Keith Cook, PhD, a professor of biomedical engineering at Carnegie Mellon University (CMU), is working to give new hope to patients with chronic lung disease.

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Gavin Jenkins, in his recent article for PittMed, reports on the work of McGowan Institute for Regenerative Medicine affiliated faculty member Louis Falo, Jr., MD, PhD, Professor and Chairman of the Department of Dermatology at the University of Pittsburgh School of Medicine.  Seven years ago, and inspired by the history of scratch vaccinations, Dr. Falo began researching microneedle array technology, which delivers medicine through the skin.  Afterall, our skin is our biggest organ, and our first line of defense against viruses and other pathogens we come across.

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Can nanomedicine play a role in the future of pain treatment?  Several answers to this question were presented during the American Pain Society Scientific Summit in Anaheim, California, earlier this year.  McGowan Institute for Regenerative Medicine affiliated faculty member Jelena Janjic, PhD, Associate Professor of Pharmaceutics in the Graduate School of Pharmaceutical Sciences and Mylan School of Pharmacy at Duquesne University and the Founder and Co-Director of the Chronic Pain Research Consortium at Duquesne, provided one solution through her work on targeted nanomedicine for chronic pain treatment.

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The Controlled Release Society has announced that McGowan Institute for Regenerative Medicine faculty member and University of Pittsburgh professor Steven Little, PhD, is the recipient of its 2018 Young Investigator Award. The honor annually recognizes one individual in the world, 40 years of age or younger, for outstanding contributions in the science of controlled release. Dr. Little is the William Kepler Whiteford Endowed Professor and Chair of the Department of Chemical and Petroleum Engineering at Pitt’s Swanson School of Engineering.

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Overall antibiotic use was not curbed by giving physicians the results of biomarker tests in patients with suspected lower respiratory tract infections, according to findings from the Procalcitonin Antibiotic Consensus Trial (ProACT). The national, randomized clinical trial was coordinated by researchers at the University of Pittsburgh School of Medicine, and funded by the National Institute of General Medical Sciences, part of the National Institutes of Health (NIH). The results, published recently in the New England Journal of Medicine, were presented at the ATS 2018 International Conference in San Diego.  McGowan Institute for Regenerative Medicine affiliated faculty members Derek Angus, MD, MPH, and John Kellum, MD, are co-authors on the study.

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McGowan Institute for Regenerative Medicine affiliated faculty member and Duquesne University Professor Jelena Janjic, PhD, has created the first inflammatory pain nanomedicine that could significantly reduce the need for opioids in treating pain.

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“Vasopressors are critical to treat patients with shock. The critical care community now has another tool to use,” said McGowan Institute for Regenerative Medicine affiliated faculty member John A. Kellum, MD, Director of Center for Critical Care Nephrology, Vice Chair for Research, and Professor of Critical Care Medicine, University of Pittsburgh. “The approval of angiotensin II represents a major advance in the treatment of patients with septic or distributive shock.”

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Low back pain is the leading cause of disability worldwide and becomes increasingly common as human bodies age. Currently, more than 40 percent of low back pain patients are prescribed opioids at some point. Researchers at Pitt want to bring that number down.

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McGowan Institute for Regenerative Medicine affiliated faculty member Marc Simon, MD, MS, FACC, Associate Professor of Medicine, Bioengineering, and Clinical Translational Science, and Director, Heart Failure Research / Clinical Hemodynamics Core Facility, at the Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute at the University of Pittsburgh, is the lead investigator on a 50-patient, open-label Phase 2 study in patients with pulmonary hypertension (PH) from multiple different etiologies.  Dr. Simon recently presented the study’s results in an invited lecture at the 4th Annual Drug Discovery and Development Symposium for Pulmonary Hypertension.  The study’s product, Aironite, is in development by Savara, Inc., a clinical-stage specialty pharmaceutical company focused on the development and commercialization of novel therapies for the treatment of serious or life-threatening rare respiratory diseases.

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Patents are a crucial element in moving academic research to the marketplace, where it can make a difference in the world. Without the economic protection a patent provides, there is little incentive for companies to invest time and risk large amounts of money to further develop early stage discoveries into technologies, products, or life-saving treatments.

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A groundbreaking new study led by researchers from the University of Pittsburgh and UPMC—including McGowan Institute for Regenerative Medicine affiliated faculty member Marc Simon, MD, Associate Professor of Medicine, Director of Heart Failure Research, Medical Director of the Montefiore Clinical & Translational Research Center of the Clinical Translational Science Institute, and an Attending Physician in the Advanced Heart Failure and Cardiac Transplantation Section in the Heart and Vascular Institute of the University of Pittsburgh—has identified a new group of compounds that could have robust effects in treating pulmonary hypertension (PH), an enigmatic but sometimes fatal disease of the blood vessels of the lungs that currently has no cure. The findings, which were published in The Journal of Clinical Investigation, highlight the use of these drugs to alter vessel stiffness and its downstream control of metabolism, a link previously unknown for people suffering from the progressive disease.

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SkinJect, Inc., a Pittsburgh-based company, today announced its completion of a license with the University of Pittsburgh to its novel, minimally invasive treatment for common forms of non-melanoma skin cancer, basal cell and squamous cell carcinoma. Two million new cases of basal cell cancer are reported each year in the United States, and more than half of all patients suffer a recurrence. The new product under development could dramatically change the way these skin cancers are treated.

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The University of Pittsburgh’s Center for Medical Innovation (CMI) awarded grants totaling $140,000 to six research groups through its 2016 Round-1 Pilot Funding Program for Early Stage Medical Technology Research and Development.  Two of the six research groups include projects of McGowan Institute for Regenerative Medicine affiliated faculty members—Justin Weinbaum, PhD, Marina Kameneva, PhD, and Jonathan Waters, MD.  The six latest funding proposals include developing a novel vascular access system, a shunt for treatment of fetal hydrocephalus in-utero, a system for stroke rehabilitation, a cell therapy for treatment of aortic aneurysm, a method for treatment of sickle cell anemia, and a novel mechanical device for use in general surgery.

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Guidelines on lipid-lowering treatment for primary prevention of cardiovascular disease (CVD) are provided by the American College of Cardiology/American Heart Association (ACC/AHA) and the European Society of Cardiology (ESC). The recommendations for lipid-lowering treatment initiation from both guidelines are based on evidence from randomized clinical trials (RCTs) demonstrating the efficacy of statins for primary prevention of CVD. The trial evidence was translated by recommending initiation of treatment for adults with a predicted 10-year risk for CVD exceeding a given threshold.  However, global-risk algorithms were never used as an enrollment criterion for RCTs. Therefore, it has been argued that risk-based allocation of statins does not fully reflect the existing evidence. The degree of overlap and discrepancy between U.S. and European guidelines in light of available trial evidence remains unclear.  The Netherlands and U.S. authors from a recent paper published in JAMA Cardiology, aimed to compare recommendations from the latest ACC/AHA and ESC prevention guidelines with the evidence from 10 major primary prevention RCTs for statins.

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New Processes Could Provide Personalized Pain Treatment

As reported by Karen Ferrick-Roman, Duquesne University Fall 2014 Magazine, pain costs Americans up to $635 billion each year for medical treatment and in lost productivity, says the Institute of Medicine of the National Academies. That hefty price tag is equivalent to the first 10 years of spending for homeland security, illustrating a nationwide problem of giant proportions.  Today, an estimated 116 million Americans live with chronic pain.

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Precise Electronic Drug Release Thanks to Graphene Oxide Nanocomposite Material

McGowan Institute for Regenerative Medicine faculty member Tracy Cui, PhD,

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Pitt Team Aims to Change Tissue Microenvironment to Fend Off Cancerous Tumors

Instead of developing new drugs that directly target tumors, the team has been working on adjuvant agents that alter the immunological microenvironment around the tumor…

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Compounded Medication to Prevent Preterm Birth Not a Safety Risk

A new study published online in the American Journal of Obstetrics and Gynecology by McGowan Institute for Regenerative Medicine affiliated faculty member Raman Venkataramanan, PhD, professor of pharmaceutical sciences at the University of Pittsburgh as well as professor of pathology within the School of Medicine, and a team of researchers from the University of Pittsburgh School of Medicine and the University of Pittsburgh School of Pharmacy, reports that 17-hydroxyprogesterone caproate (17-OHPC), a medication that reduces the rate of preterm birth in high-risk women, did not raise any safety concerns when the medication was prepared and dispensed by independent compounding pharmacies throughout the United States.

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Gum Disease Treated by Using Homing Beacon to Bring Needed Immune Cells to Inflamed Area

The red, swollen, and painful gums and bone destruction of periodontal disease could be effectively treated by beckoning the right kind of immune system cells to the inflamed tissues, according to a new pre-clinical study conducted by researchers at the University of Pittsburgh, including McGowan Institute for Regenerative Medicine faculty members Steven Little, Ph.D., associate professor and chair of the Department of Chemical and Petroleum Engineering, Pitt’s Swanson School of Engineering, and Charles Sfeir, D.D.S., Ph.D., director, Center for Craniofacial Regeneration, and associate professor, Departments of Periodontics and Oral Biology, Pitt’s School of Dental Medicine.  Their findings, published in the early online version of the Proceedings of the National Academy of Sciences, offer a new therapeutic paradigm for a condition that afflicts 78 million people in the U.S. alone.

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McGowan Institute for Regenerative Medicine faculty are trying to open a new front in the battle against gum disease, the leading cause of tooth loss in adults and sometimes termed the most serious oral health problem of the 21st century.

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Clinical Trial: Neuroprotective Agent for Treating Severe Traumatic Brain Injury

At the Neurotrauma Society’s 2012 Annual Meeting, McGowan Institute for Regenerative Medicine affiliated faculty member David Okonkwo, MD, PhD, assistant professor with the Department of Neurological Surgery/University of Pittsburgh Medical Center, director of Neurotrauma and of the Spinal Deformity Program, clinical director of the Brain Trauma Research Center, and most recently, the associate director of the Center for Injury Research and Control, presented results from BHR Pharma’s SyNAPSe® clinical trial.  Dr. Okonkwo, study principal investigator, announced the trial enrollment midpoint of 590 patients, which was the milestone for the global Phase 3, multi-center trial.  This was achieved when a study subject was randomized by Songklanagarind Hospital in Thailand.

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Seed Funds Help Make NIH Funding Possible

A project, originally supported by McGowan Institute for Regenerative Medicine through seed funds from the Pennsylvania Commonwealth, has now received National Institutes of Health (NIH) funding.  The National Institute of General Medical Sciences at the NIH recently announced that Qrono Inc. will receive a Small Business Technology Transfer Grant in 2012.  The grant is entitled “A New In Silico Design Platform for Building Custom Controlled-Release Systems.”

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Renal Cell Carcinoma Focus of Clinical Trial

McGowan Institute for Regenerative Medicine affiliated faculty member Michael Lotze, PhD, professor of surgery and bioengineering, vice chair of research within the Department of Surgery, assistant vice chancellor in the six schools of the health sciences at Pitt, and director of strategic partnerships within the University of Pittsburgh Cancer Institute as well as the Catalyst Program within the recently funded Clinical and Translational Research Institute, is the principal investigator of an ongoing clinical trial focused on renal cell carcinoma (RCC).  The main goal of the research study is to determine whether treating renal cell cancer patients with the study drug, hydroxychloroquine, along with IL-2, a standard treatment of kidney cancer that has spread to other parts of the body, can make the cancer easier to kill and eliminate. Another goal is to see how the study drug affects the body’s immune cells which fight cancer cells.

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Biomimetics Is Focus of Senior Vice Chancellor’s Research Seminar Series Lecture

McGowan Institute for Regenerative Medicine faculty member Steven Little, PhD, chair of the Department of Chemical and Petroleum Engineering, associate professor, and Bicentennial Alumni Faculty Fellow of the University of Pittsburgh Swanson School of Engineering, recently delivered a lecture in the 2012 Senior Vice Chancellor’s Research Seminar series.  The title of Dr. Little’s presentation was “Medicine That Imitates Life through Biomimetic Drug Delivery.”

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