Translational science

News

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

News

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.

News

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.

News

Jonathan Pitre

Breakthrough Bone Marrow Therapy Developed by U Researchers

July 5, 2017

A team of scientists led by Jakub Tolar, director of Consortium member the Stem Cell Institute, believes they've discovered a new therapy to help patients suffering from a devastating skin disease. According to the Minneapolis Star Tribune, "A decade after performing the world’s first bone marrow transplants to treat epidermolysis bullosa (EB) — a rare and potentially fatal skin disease — university researchers believe they have discovered a 'powerhouse' new formula that . . . helps the body grow new skin and will allow patients such as [Jonathan] Pitre, 17, to live longer, less painful lives." EB can cause friction or even a minor bump to become a significant wound; it sometimes leads to severe infections and skin cancer. Over the past several months, Pitre has been treated at the U's Masonic Children's Hospital, undergoing radiation, chemotherapy and two bone marrow transplants. Long-term research has helped doctors identify the most effective cell type for EB treatment — mesenchymal stem cells, are "uniquely good at bullying their way into the body and producing the missing collagen [that causes the disease]. 'This is the first time ever, that I know of, when you are infusing them with the goal that these cells will stay,' Tolar said. 'They will graft into the skin, set up shop there. It’s as if these mesenchymal stem cells are coming home.'"

News

Sharon Terry

Citizen Science Pioneer Diagnoses Medical Research Shortcomings

June 22, 2017

In a newly-released TEDMED talk, Sharon Terry of Genetic Alliance describes her journey to becoming a citizen scientist after her two children were diagnosed with the genetic condition pseudoxanthoma elasticum (PXE), which causes the symptoms of premature aging. She quickly learned “that there was no systematic effort to understand PXE . . . researchers competed with each other because the ecosystem is designed to reward competition rather than alleviate suffering.” In response, Terry and her husband, Patrick, educated themselves on the disease and gathered thousands of similarly affected people to initiate studies and clinical trials. The message of her talk is that “citizen scientists, activists using do-it-yourself science, and crowdsourcing are all changing the game.” Ms. Terry is a member of the working group for the LawSeq project, which is laying the policy groundwork to translate genomic medicine into clinical application; the principle investigators are Consortium Chair Susan M. Wolf, JD; Ellen Wright Clayton, MD, JD of Vanderbilt University; and Frances Lawrenz, PhD of the University of Minnesota. Last December, the Consortium sponsored an event on patient-led medicine and citizen science; video can be viewed here.

News

Doctor with patients and tablet computer

Research Ethics Videos Available

April 19, 2017

Videos are now available for sessions held during the two research ethics conferences presented at the University of Minnesota on March 8 and 9, 2017. At these events, researchers, policymakers, bioethicists, patient advocates and other stakeholders explored best practices for research with human participants. The conferences are The Future of Informed Consent: A Century of Law, Ethics & Innovation (March 8) and The Challenges of Informed Consent in Research with Children, Adolescents & Adults (March 9). The videos are posted at z.umn.edu/researchethicsvideos for free public access.

News

NIH logo

National Institutes of Health Announces Adoption of SMART IRB

March 24, 2017

Multisite clinical trials are a powerful way to drive the discovery of new therapies, but ensuring that the health and privacy of study participants are protected across multiple Institutional Review Boards (IRBs) has slowed things down in the past. Today, the NIH reached a major milestone: all of their Clinical and Translational Science Awards program sites have agreed to a new authorization agreement, SMART IRB. The agreement – whose acronym stands for Streamlined, Multisite, Accelerated Resources for Trials IRB – "will enable all participating study sites to rely on the ethics review of one IRB for each study, making it possible to initiate multisite studies within weeks instead of months. For patients waiting to enroll in a study, this could make a life-saving difference." Among the signatories on the agreement is Consortium member the Clinical and Translational Science Institute (CTSI).

Publication

O'Daniel JM , McLaughlin HM , Amendola LM , Bale SJ , Berg JS , Bick D , Bowling KM , Chao EC , Chung WK , Conlin LK , Cooper GM , Das S , Deignan JL , Dorschner MO , Evans JP , Ghazani AA , Goddard KA , Gornick M , Farwell Hagman KD , Hambuch T , Hegde M , Hindorff LA , Holm IA , Jarvik GP , Knight Johnson A , Mighion L , Morra M , Plon SE , Punj S , Richards CS , Santani A , Shirts BH , Spinner NB , Tang S , Weck KE , Wolf SM , Yang Y , Rehm HL . A Survey of Current Practices for Genomic Sequencing Test Interpretation and Reporting Processes in US Laboratories. Genetics in Medicine 2016;18(11). Download PDF (638.01 KB)

News

Nanobot in bloodstream

Chemistry Nobel Prize Goes to Nanotech Scientists

October 5, 2016

Three scientists have been awarded the 2016 Nobel Prize in Chemistry for their work on designing tiny machines "a thousand times thinner than a strand of hair," according to the BBCJean-Pierre Sauvage (Strasbourg University), Fraser Stoddart (Northwestern University) and Bernard Feringa (University of Groningen) will share the prize, which is worth approximately $930,000. Nanotechnology – "the creation of structures on the scale of a nanometer, or a billionth of a meter," as described in the New York Times – could be used to precisely deliver pharmaceuticals within the human body and may lead to entirely new therapeutic approaches. Consortium Chair Susan M. Wolf has led significant efforts, funded by the National Science Foundation and National Institutes of Health, to determine the best way to protect human beings who participate in nanotechnology research. Two major symposia evaluated oversight models using a historical and comparative approach and produced the first systematic, comprehensive recommendations on how to protect human participants in nanotech research. To see all the Consortium's work on nanotechnology, click here

News

Prof. Suma Jacob

DNA-based Autism Research May Support Early Interventions

September 28, 2016

A growing amount of evidence points to the importance of early behavioral interventions in the treatment of autism. However, health professionals typically won't diagnose the disorder sooner than 18 months, and often much later. To help close this gap, a major, national research study is being led in Minnesota by Prof. Suma Jacob, MD, PhD, of Consortium member the Center for Neurobehavioral Development. The study, SPARK, has the goal of collecting DNA and other data from 50,000 people with autism and their family members. An article in MinnPost quotes Prof. Jacob as saying "There have been studies that have shown that there are strong heritable components in autism. . . . What’s exciting about [SPARK] is we know that we need to gather a large number of families with autism to find as many potential connections as possible. We are in the process of collecting that large sample." She cautions, however, "The disorder is different in each individual. . . . Generalizations just don’t fit."

News

Prof. Jerrold Vitek

Major Grant Will Focus on Promising Parkinson's Treatment

September 26, 2016

The University of Minnesota has been awarded a five-year, $9 million grant for Parkinson’s research. Jerrold Vitek, MD, PhD, will lead the study, which establishes a prestigious Udall Center of Excellence in Parkinson's Disease Research on campus, one of only nine such centers in the nation. An article about the grant in Twin Cities Business Magazine quotes Brian Herman, PhD, the U's Vice President for Research: "I think it puts this university in a very elite class of other major U.S. and international research universities that have recognized expertise." Prof. Vitek's work focuses on the therapeutic effect of deep brain stimulation (DBS), which he likens to "a pacemaker for the brain." Watch a video about the grant and DBS here

News

Liquid being transferred from test tube to beaker

New Rules: Researchers Must Publish Results of All Clinical Trials

September 19, 2016

In a step toward greater transparency, the US Department of Health and Human Services (HHS) has announced that, going forward, researchers funded by the National Institutes of Health (NIH) must report the design and results of all clinical trials, whether successful or not. An article in Nature describes the new rules, which are designed to reduce the number of clinical trials that aren't reported; a 2013 study found that approximately half the time, trial results aren't published if they have unfavorable results. The article notes, "Under the new rule, trials must be registered on ClinicalTrials.gov within 21 days of enrolling their first patient — researchers can no longer wait for the results of their trails to report their data." For the first time, those who don't adhere to the guidelines can be penalized by having their funding withdrawn. Bioethicist Jennifer Miller of New York University’s Langone Medical Center praises the change, noting "You need a clear law so you can have clear compliance." 

News

Cancer Moonshot logo

Cancer Moonshot Announces Next Steps for Transformative Research

September 8, 2016

In his 2016 State of the Union address, President Barack Obama announced the Cancer Moonshot, an intensive research collaborative to accelerate immunotherapy as an effective treatment for cancer patients. Led by Vice President Joe Biden, doctors, patients, researchers, pharmaceutical companies, insurance companies, and technologists are striving to managing cancer as a chronic condition, like diabetes, rather than a devastating and life-threatening illness. Last June, Consortium member center Masonic Cancer Center hosted a local event that was part of the national launch of the Moonshot, which featured remarks by VP Biden and US Senators Amy Klobuchar and Al Franken. Yesterday, a Blue Ribbon Panel released its roadmap for the effort, recommending 10 transformative research approaches for achieving major strides in cancer prevention, diagnosis and treatment by 2020. The report can be read here.

Lecture

News

Scan of brain with Parkinson's Disease

Feud Over Parkinson's Drug Trial Reveals Power of Gatekeepers

August 31, 2016

A dispute between Dr. Charbel Moussa, a scientist at Georgetown University, and the Michael J. Fox Foundation (MJFF) has exposed some challenges of conducting independent drug research in collaboration with a major advocacy group. An article in STAT outlines the conflict, noting that the MJFF-funded Phase 2 trial based on Moussa's previous research "was supposed to begin in October, but Fox and the Georgetown team had a bitter falling out, and it’s unclear whether Georgetown will be able to obtain the medicine from its manufacturer so that the study can proceed. [MJFF], meanwhile, is moving forward on its own, and has established a separate group to study the same drug." The article provides a glimpse into unusually public discord within the world of high-stakes research. In a statement, MJFF states the conflict with Dr. Moussa and his team is a result of differences of opinion regarding the design and execution of the study, citing "the limitations of open-label clinical trials in Parkinson's, which have been shown to be disproportionately affected by placebo response." Leigh Turner, a professor at Consortium member the Center for Bioethics, is quoted in the STAT article, saying funders and journal editors should "be above reproach in their handling of insider information. [Turner cautions] 'You have these gatekeeper moments, where it is possible to take advantage of positions like that, to gobble up as much information as possible, and repurpose it for your own advantage.'"

News

Model of crystal structure for CRISPR-Cas9

There's More Than One Way to Edit a Gene

August 15, 2016

The gene-editing technique known as CRISPR has generated immense excitement for enabling scientists to alter genomes "with unprecedented precision, efficiency and flexibility," in hopes of accelerating cures for genetic diseases. However, while CRISPR has been receiving the lion's share of attention in the media, a new article in Scientific American outlines its limitations: as researchers have used CRISPR, particularly the Cas9 tool, they've been reminded "how fragile every new technology is," according to George Church of the Harvard Medical School. The article describes several alternatives that researchers hope will offer more precise pathways for rewriting DNA, which plant scientist Daniel Voytas of the University of Minnesota notes is essential to the advancement of the field: “Everyone says the future is editing many genes at a time, and I think: ‘We can’t even do one now with reasonable efficiency.'" Each month seems to bring new advances in the field: read the article, which provides an up-to-date overview of what's happening, here

News

Poo lookalike ice cream sundae from Beijing

Consortium Lecturers Discuss Fecal Transplant Pill

August 2, 2016

An article by Ed Yong in The Atlantic describes attempts to create effective stool substitutes to replace fecal transplants used to treat Clostridium difficile, which kills almost 30,000 Americans each year. One such substitute, from Seres Therapeutics, recently failed during a phase 2 drug trial. Yong quotes Dianne Hoffmann, who spoke last spring during the Consortium's Microbiome Therapeutics lecture series: she notes that "whole stool" is clearly more effective than bacterial pills. "Something in there is working. We just don’t know what it is, and it might be hard to deconstruct.” Another speaker in the lecture series, Alexander Khoruts, adds "The full spectrum of microbes harvested from donors has been designed by nature, and has a proven safety track record in the original host. That’s a very hard benchmark to improve upon with any kind of synthetic." You can view Diane Hoffmann's lecture on regulating microbiome therapeutics here; Alex Khoruts' talk, The Evolving Human Microbiome, is here.

News

National Academies of Science and Medicine logo

National Academies Call for Halt to Common Rule Update

June 29, 2016

The National Academies of Sciences, Engineering, and Medicine (NASEM) have issued a call for the withdrawal of the Notice of Proposed Rulemaking (NPRM), which is intended to update the federal guidelines for research with human participants, also known as the Common Rule. In the view of the National Academies, the NPRM "is marred by omissions, the absence of essential elements, and a lack of clarity," an opinion shared by many scientific groups. In its place, NASEM suggests a national commission be appointed to issue a "course correction" that will update the rules without increasing regulatory burdens or diminishing the effectiveness of human subjects research. An article in Science describes the debate and the initial response by a Department of Health and Human Services spokesperson, who notes they've "received more than 2,100 public comments" on the NPRM and will "take all public comments seriously" as they develop the final rule.

Research

LawSeq logo corrected Dec 2016

LawSeqSM: Building a Sound Legal Foundation for Translating Genomics into Clinical Application

This innovative 3-year project, based cooperatively at the University of Minnesota and Vanderbilt University, has convened a national Working Group of top legal and scientific experts to analyze current federal and state law and regulation on translational genomics and to generate consensus guidance on what the law should be. The project team will build a searchable online database of relevant law and an annotated bibliography for free public access, systematically collect and analyze a range of stakeholder inputs, convene a national public conference, and publish analyses and recommendations to help shape the law to support genomic medicine.