Translational science


C difficile bacteria

Research Reveals Exciting New Possibilities for Microbiome Therapies

February 15, 2018

A major paper just published in Cell Host & Microbe sheds light on a question that has puzzled scientists for years: while we know fecal microbiota transplantation (FMT) works for people suffering from recurrent Clostridium difficile infection, how exactly does it work? The research team behind the paper includes Consortium collaborators Alexander Khoruts and Michael J. Sadowsky, the director of the Biotechnology Institute, a Consortium member center. They used clinical experiments and statistical modeling to uncover the "rules" for how donor bacteria grafts itself to existing gut microbes in the host. One of the outcomes of the research is Strain Finder, a method to predict which types of bacteria will best colonize a microbiome being treated via FMT. In addition to its implications for the treatment of C. diff, the study findings may also help with therapies for metabolic syndrome as well as other common conditions. Prof. Khoruts gave a lecture on The Evolving Human Microbiome that was moderated by Michael J. Sadowsky; it's been viewed thousands of times because it provides a coherent overview of this fascinating subject; you can view it here. More recently, Martin J. Blaser spoke about the effects of antibiotics on the human microbiome; you can view his lecture here


Vercise deep brain stimulation device

Key Parkinson's Brain Stimulation Research Led by U, Funded by MnDRIVE

January 10, 2018

A brain stimulation device just approved by the US Food and Drug Administration (FDA) was developed as part of a multi-site clinical study led by University of Minnesota medical researchers and partly funded by the MnDRIVE program. According to Twin Cities Businessneurology department chair Dr. Jerrold Vitek and his research team successfully implanted the first non-trial patient with the Vercise system at the University of Minnesota Medical Center in December. The device is manufactured by Boston Scientific, and has been hailed as “one the most innovative neuromodulation technologies available today.” Deep brain stimulation is used to reduce the involuntary shaking and stiffness that are among the symptoms of Parkinson's disease. MnDRIVE – Minnesota’s Discovery, Research, and InnoVation Economy – is a partnership between the University and the State of Minnesota that aligns areas of research strength with the state’s key and emerging industries. One of of MnDRIVE's areas of focus is the treatment of brain conditions; the others are robotics, global food and the environment. 


Model of crystal structure for CRISPR-Cas9

Will 2018 Be the Year of CRISPR?

December 27, 2017

An article in MIT Technology Review notes, "In just the past few years, advances in [the gene-editing technology] CRISPR have been happening at a breakneck speed — and companies have sprung up to commercialize the technology. Now, patients in Europe and the U.S. could be treated with CRISPR-based therapies as soon as 2018." Looking at actual research, however, the picture is more mixed: trials are planned at Stanford University, for example, but have already been delayed at the University of Pennsylvania. Alexey Bersenev, director of the Advanced Cell Therapy Lab at Yale New Haven Hospital, cautions “the field is currently over-optimistic about possible results of clinical trials. . . . Every new and hot biomedical technology usually undergoes an inflated expectations phase.”


A film still of Audrey Hepburn wearing a red dress

A Prolific Gender Studies Researcher's Outcomes Questioned

December 14, 2017

According to an article in Ars Technica, "Psychologist Nicolas Guéguen's large body of research is the kind of social psychology that demonstrates, and likely fuels, the Mars vs. Venus model of gender interactions, with its assertions, for example, that men consider women wearing red to be more attractive. But it seems that at least some of his conclusions are resting on shaky ground. Since 2015, a pair of scientists, James Heathers and Nick Brown, has been looking closely at the results in Guéguen's work. What they've found raises a litany of questions about statistical and ethical problems. In some cases, the data is too perfectly regular or full of oddities, making it difficult to understand how it could have been generated by the experiment described by Guéguen." In addition to identifying questionable research outcomes, Heathers and Brown learned that some of Guéguen's methodologies put female researchers in sexualized situations. These types of concerns are central to Research Integrity and Trustworthy Science, a conference that will be held on the University of Minnesota campus on March 8. Eminent, nationally-known presenters will address data fabrication, selective data reporting, predatory journals and concerns about the reproducibility of scientific findings. To learn more and register for free in-person or webinar attendance, click here


Prof Susan M. WOlf

Wolf Joins TOPMed Advisory Panel

November 30, 2017

The National Heart, Lung, and Blood Institute (NHLBI) has appointed Consortium Chair Susan M. Wolf to the External Advisory Panel for the Trans-Omics in Precision Medicine (TOPMed) Program, which is dedicated to the advancement of heart, lung, blood, and sleep precision medicine. Since its launch in 2014, TOPMed has supporting whole genome sequencing and other omics platforms such as metabolomics and proteomics; the project researchers expect to have achieved a total of 150,000 whole genome sequences by the end of 2018. Research conducted on such a scale raises importance governance questions, including how best to ensure the privacy of participants' health data when it's shared with the broader research community. Prof. Wolf is a renowned expert on genomic privacy, having led several research studies that established the legally and ethically preferred methods to manage research findings from biobanks. The External Advisory Panel will provide the NHLBI Director and staff with expert guidance on TOPMed, with the goal of applying systems biology to drive the future of precision medicine. 


Brian Madeux

U Scientist is Among First to Edit Genes Within Body

November 22, 2017

Last week a 44-year-old California man was the first to be implanted with billions of copies of "a corrective gene and a genetic tool to cut his DNA in a precise spot," according to The Guardian. The patient, Brian Madeux, suffers from Hunter syndrome and has endured 26 operations to treat symptoms of the disease. Dr. Chester Whitley of Masonic Cancer Center, a Consortium member, is a lead scientist researching the therapy. It is based on zinc-finger nucleases, which work like "molecular scissors that seek and cut a specific piece of DNA." Dr. Whitley is quoted in the Atlantic, where he notes, “You know exactly where you’re going in the genome. It’s not like using a shotgun hoping you’re hitting a bird. It’s like using a rifle.” While still in clinical trial, if proven effective, zinc-finger nucleases could "kick off a new era for gentic disorders, one where kids never have to suffer their effects in the first place." 


Skin grown in a lab for use as graft

Whole-body Skin Graft Gives Hope to a Boy with a Rare Disease

November 10, 2017

An article in Science Magazine describes the case of a 7-year-old boy who suffers from "a severe form of epidermolysis bullosa (EB), an often-fatal group of conditions that cause skin to blister and tear off at the slightest touch." He has made a dramatic recovery after being treated with genetically modified stem cells that were used to grow new skin, which was then grafted onto his body. Jakub Tolar, MD, is also developing therapies for EB; he is Dean of the Medical School and Director of the Stem Cell Institute, a Consortium member. In the Science article, he notes, that "the grafts . . . can’t repair damage to internal surfaces such as the esophagus, which occurs in some EB cases. Fortunately, that wasn’t an issue for the boy in this study. The treatment is 'a good step in the right direction,' Tolar says, 'but it’s not curative.'” In a separate article in STAT, John Wagner, MD, Director of the University of Minnesota Masonic Children’s Hospital’s blood and marrow transplant program, says the findings have “'extraordinary' potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells — those in blood and bone marrow."



IonE 10 yrs logo

IonE Celebrates Ten Years

October 30, 2017

The Institute on the Environment (IonE), a Consortium member, is celebrating its tenth anniversary with a fundraiser scheduled for the evening of Thursday, Nov. 16. All proceeds support the ongoing work of IonE, a cross-disciplinary institute that focuses on solving major environmental challenges. IonE is a leader in the field of ecological economics — the science and practice of incorporating externalities and the value of nature into decision making. They foster research and engagement; entrepreneurship and leadership development; service to society; and innovative communications like their magazine Ensia


Map marker with My Training text

CTSI Launches New Training Tool for Research with Human Participants

September 25, 2017

The Clinical and Translational Science Institute (CTSI), a Consortium member, has launched a new web-based training tool for University of Minnesota clinical research professionals who work with human participants, and their supervisors. The Human Research Training website is a free, easy-to-use tool to help these researchers identify and maintain the appropriate training, certification, credentials, and immunizations. “Previously there wasn’t a systematic way to identify what research training was necessary or a system to track and maintain training compliance,” said Megan Hoffman, CTSI Workforce Development Program Manager, Clinical Translational Research Services. “This tool provides the linking and connectivity between U Learn and Moodle in one spot — it works to provide training uniformity across our vast research enterprise. The tool doesn’t add to the training requests, it simply tracks them in one place.”


Red glass double helixes

Is it Clinical Care? Is it Research? Navigating the Difference in Genomic Medicine

August 31, 2017

A working group of the Clinical Sequencing Exploratory Research (CSER) Consortium has published the first-ever study describing new approaches to a crucial question: how should genomics investigators handle the increasingly blurry boundary between research and clinical practice? The article, "Navigating the Research-Clinical Interface in Genomic Medicine" appeared in Genetics in Medicine, which is published by ACMG. A new blog post by Consortium Chair Susan M. Wolf, JD and Wylie Burke, MD, PhD (Department of Bioethics and Humanities at the University of Washington) describes the results of CSER's empirical research. A 22-item survey was administered to investigate how investigators were conceptualizing and navigating the research-clinical interface. The results were striking: most of the studies describe a merger of research and clinical care. According to Wolf and Burke, "This challenges the conventional wisdom that while clinical care benefits patients, research seeks only generalizable knowledge." CSER encompasses nine National Institutes of Health (NIH)-funded projects driving the translation of genomic sequencing into clinical care; among these are grants for which Consortium Chair Susan M. Wolf serves as a co-Principal Investigator: Disclosing Genomic Incidental Findings in a Cancer Biobank (with Gloria Petersen of Mayo Clinic and Barbara Koenig of UCSF) and LawSeq: Building a Sound Legal Foundation for Translating Genomics into Clinical Application (with Ellen Wright Clayton of Vanderbilt and Frances Lawrenz of the University of Minnesota). Prof. Wolf was the lead author on the Genetics in Medicine article. 



Nanowarming in an alternating magnetic field

U Researchers Develop New Nanotech to Improve Transplant Outcomes

August 23, 2017

A team led by University of Minnesota researchers has developed a new method for thawing frozen tissue that may enable long-term storage and subsequent viability of tissues and organs for transplantation. The method, called nanowarming, prevents tissue damage during the rapid thawing process that would precede a transplant. The U of MN has long been a leader in organ transplants – 2017 marks the 50th anniversary of the world's first pancreatic transplant, in 1967. According to the study's co-author, Prof. Michael Garwood, PhD, of the University of Minnesota Dept. of Radiology, "Prior to the development of [this nanowarming technology, called] SWIFT, no imaging technique had been capable of quantifying high concentrations of iron-oxide #nanoparticles in tissues non-invasively." Consortium Chair Susan M. Wolf led the team that developed the oversight guidelines for nanotechnology research with human participants; learn more about those here.



Sleeping baby in mother's arms

In a First, Study Demonstrates Effectiveness of a Probiotic Strain in Preventing Disease

August 18, 2017

Probiotics like Lactobacillus have become common supplements, consumed either in pill form or in food. Despite their popularity, however, scientific evidence of probiotic benefits has been scarce. Now, according to an article in The Atlantic, a team from the University of Nebraska-Lincoln has released the results of a large clinical trial demonstrating that babies given a combination of the probiotic Lactobacillus plantarum and sugar "had a significantly lower risk of developing sepsis — a life-threatening condition where infections trigger body-wide inflammation, restricted blood flow, and organ failure." The team, led by Prof. Pinaki Panigrahi, took the novel approach of identifying probiotic strains that actually thrive in the human gut; previously, studies have focused on those that are easy to grow and manufacture. The study may be the first to credibly demonstrate a benefit that had, until now, been theoretically possible but unproven. The lack of hard evidence has left most scientists skeptical of the probiotics craze — for example, Prof. Alexander Khoruts of the University of Minnesota's Microbiota Therapeutics Program has stated his belief that, until further tested, they're a waste of money; see his Consortium-sponsored talk on the evolving human microbiome here. Since this large-scale study shows compelling results, it could mark the beginning of a new era in probiotic therapies.


R. Alta Charo

Charo Explains Breakthrough Genetic Treatment for Leukemia

July 27, 2017

For the first time, an FDA panel has recommended that the agency approve a genetic treatment for leukemia. The therapy has been shown to treat a type of leukemia that sometimes doesn't respond to standard therapies like chemotherapy. It uses genetic engineering – removing cells, editing them and then reintroducing them into the patient's body – to transform the living cells into a weapon against cancer. The clinical trial of 55 people had an 85% effectiveness rate, resulting in remission or possibly a long-term cure. In an interview on Wisconsin Public Radio, Consortium collaborator Prof. Alta Charo (University of Wisconsin Law School) notes the treatment is "not a magic bullet yet." She cites some very serious side effects, such as extreme inflammation that can cause extraordinarily high fevers. In addition, it is expensive, potentially costing as much as $500,000. If approved, the therapy is recommended for ages 3-25; however, it could eventually be rolled out to older patients. Charo describes this as an example of the "medicine of the 21st century [which will use] gene editing in order to personalize treatment . . . changing cells so they can become their own kind of biological drug." Charo is a member of the National Working Group for the LawSeqSM project, for which Consortium Chair Prof. Susan M. Wolf is one of the Principal Investigators. Listen to the entire interview here


Prof. Heidi Rehm of Harvard Medical, lecturing

Rehm Calls for Open Data Sharing to Improve Genomic Interpretation

July 24, 2017

In an article published in Genetics in Medicine, Heidi L. Rehm, Director of the Laboratory for Molecular Medicine at Partners Healthcare Personalized Medicine and Associate Professor of Pathology at Harvard Medical School, calls for the sharing of variant interpretations to advance medicine and improve patient care. Reviewing the progress made over the past five years by ClinVar and other major studies, she highlights the strides than can be made when multiple laboratories collaborate on evaluating the significance of individual genes, assigning categories consistently, and allowing peer review. To that end, Rehm lays out five recommendations for regulators, agencies, providers, laboratories and others. Rehm is a frequent Consortium collaborator – she wrote a major article on improving genomic laboratory practices with Consortium Chair Susan M. Wolf and others. She also spoke about her work to define standards for the interpretation of genomic variants at a lecture hosted by the Consortium in 2015; video of that talk is available here


Earl Bakken

Center for Spirituality & Healing Renamed in Honor of Earl E. Bakken

July 18, 2017

The University of Minnesota has announced it is renaming two health-focused interdisciplinary centers, including Consortium member the Center for Spirituality & Healing (CSH), in honor of Twin Cities inventor and entrepreneur Earl E. Bakken. Bakken is the co-founder of Medtronic, is an alumnus of the U, and was an early mentor for CSH director Mary Jo Kreitzer. According to the University's announcement, "Bakken has a unique appreciation for both the art and science of health care, as demonstrated by his long-standing support for [CSH], a center focused on research, outreach and education of integrative health and wellbeing." The Medical Devices Centeran interdisciplinary program under the Institute for Engineering in Medicine, will also be renamed in Bakken's honor; in 1957, he developed the first wearable, external, battery-powered, transistorized pacemaker for Dr. C. Walton Lillehei, a University of Minnesota heart surgeon.


Rendering of uterus

Consortium Scholar Explores the Ethics of Uterus Transplants

July 6, 2017

In February, 2016, a 26-year-old American woman underwent the nation’s first uterus transplant. While that procedure was ultimately unsuccessful because of a post-operation infection, a Swedish team has conducted a 9-patient trial resulting in 7 pregnancies and 5 deliveries since 2013. During the 2015-2016 academic year, Law School student Katarina Lee received a Consortium Research Award to analyze the medical, legal and ethical ramifications of uterine transplantation. The practice is both fraught with medical risk and raises controversial bioethics questions because, unlike heart or kidney transplants, the operation is not life-saving. Having completed her JD, Lee now works as a clinical ethics fellow at Baylor College of Medicine’s Center for Medical Ethics and Health Policy. To learn more about this and other Consortium Research Awards, click here.