OMICS - BLOG | Journal for Translational Medicine

Translational Medicine

Jul 10

ANN ARBOR, Mich., July 9, 2012 /PRNewswire-USNewswire/  The inaugural $100,000 Taubman Prize for Excellence in Translational Medical Science has been awarded to Harry Dietz, M.D., of the Johns Hopkins University, the University of Michigan’s A. Alfred Taubman Medical Research Institute announced today.

Dietz, a cardiologist and genetics researcher, has made groundbreaking progress in the understanding of aortic aneurysm and related tissue disorders. His lab is the first to determine that some defects in the human body’s connective tissue, long thought to be unchangeable, can be modified with medication.  The discovery has overturned decades of conventional wisdom and has tremendous implications for the treatment of genetic connective tissue disorders.

Dietz will receive the $100,000 grant to further his research and will appear as keynote speaker Oct. 11, 2012 at the Taubman Institute’s annual symposium in Ann Arbor.

“Dr. Dietz epitomizes translational research,” said David Ginsburg, M.D., a Taubman Scholar and member of the Taubman Prize selection committee.  “He initiated a line of research into the molecular basis for an important class of human diseases at a time when the cause was completely unknown.

“His landmark body of research, beginning at the basic laboratory bench, identified the responsible gene and developed a mouse model, leading to remarkable new insight into molecular pathogenesis. Based on these laboratory findings, Dr. Dietz then led a clinical trial that is likely to change the standard approach to treatment for these diseases.  His accomplishments are a perfect example of exactly what we wish to honor with the Taubman Prize.”

The Taubman Prize was established to annually recognize the clinician-researcher who has done the most to transform laboratory discoveries into clinical applications for patients suffering from disease.

“Dr. Dietz exemplifies the passion and persistence of physician-scientists everywhere,” said Alfred Taubman, founder and chair of the A. Alfred Taubman Medical Research Institute.  “We established the Taubman Prize to recognize and encourage these dedicated researchers, whose drive to find effective treatments and cures is fueled by their interaction with the patients suffering from life-altering diseases.

“In honoring Dr. Dietz, an eminent scientist whose landmark findings may improve quality of life for tens of thousands of people, I am confident that we have accomplished our goal.”

Eva Feldman, M.D., Ph.D., director of the Taubman Institute and the Russell N. DeJong Professor of Neurology at the U-M Medical School, said the nominees for the prize included many of the most talented and accomplished clinician-scientists at the world’s top research institutions.

“It’s a tribute to the extraordinary nature of Dr. Dietz’s work that our selection committee unanimously agreed to award him the Taubman Prize,” said Feldman.  “The Taubman Prize is meant to reward translational research and to help remedy the long-standing disconnect between research in the laboratory and the introduction of new treatments for patients. Researchers like Dr. Dietz are truly scientists who create cures.”

Dietz, a professor of pediatric cardiology and genetics expert at the Johns Hopkins University School of Medicine and a Howard Hughes Medical Institute investigator, began his research career studying Marfan syndrome. This potentially fatal connective tissue disease enlarges the aorta, the largest blood vessel in the body and the main highway for blood as it leaves the heart. The syndrome makes the aorta more inclined to tear or burst, an often fatal event. About one in 5,000 people have Marfan syndrome, which is thought to have afflicted historical figures such as Julius Caesar, Abraham Lincoln and Charles de Gaulle. Olympic volleyball player Flo Hyman died in 1986 from an aneurysm that was the result of Marfan syndrome.

Working with his Johns Hopkins colleague, Bart Loeys, M.D., Ph.D., Dietz also was the first to observe and describe a related genetic disorder that bears their names, Loeys-Dietz syndrome.

In studying Marfan syndrome and related disorders at the molecular level, Dietz and his team have found that the lack of a certain connective-tissue protein adversely affects the development of various body tissues. They also made the key discovery that this condition is responsive to medication. Up until then, physicians had believed that manifestations of these disorders would be difficult or impossible to modify.

Dietz’s research has led to a clinical trial using the blood pressure drug losartan in Marfan syndrome patients at risk for aortic aneurysm, which currently is taking place at 20 sites in the United States, Canada and Belgium. Preliminary testing on mice in Dietz’s lab and in a smaller group of human subjects indicated that the drug dramatically slows the enlargement of the aorta, though Dietz has cautioned that full results of the broader trial will be needed to assess losartan’s efficacy.

Using this new knowledge as a foundation,  Dietz’s team and other scientists around the world are positioned to find new treatments for not only Marfan patients, but also for disorders such as aortic aneurysm, pulmonary emphysema, fibrosis, heart valve disease and skeletal muscle myopathy, to name a few.

Dietz, who is the Victor A. McKusick Professor of Genetics and Medicine at Johns Hopkins, as well as assistant director of the school’s medical genetics training program, said the prize will spotlight the importance of translational medical research.

“I am deeply honored to receive the Taubman Prize and equally thrilled by the prospect that this high-profile mechanism to recognize excellence in translational science will stimulate both interest and progress in this important field,” he said.

A national selection committee reviewed more than 30 nominations for the 2012 Taubman Prize.  Members of the committee include:

  • Nancy Andrews, M.D., Ph.D.
    Dean, Duke University School of Medicine
  • David Ginsburg, M.D.
    James V. Neel Distinguished Professor of Internal Medicine and Human Genetics, University of Michigan Medical School
    Howard Hughes Medical Institute Investigator
  • William N. Kelley, M.D.
    Professor of Medicine, University of Pennsylvania School of Medicine
  • Gary Koretzky,  M.D., Ph.D
    Professor, Pathology and Laboratory Medicine
    Vice Chair for Research and Chief Scientific Officer, Department of Medicine, University of Pennsylvania
  • Juanita Merchant, M.D.
    Professor of Internal Medicine and Professor of Molecular and Integrative Physiology, University of Michigan Medical School
  • Elizabeth G. Nabel, M.D.
    President, Brigham and Women’s/Faulkner Hospitals
  • David G. Nathan, M.D.
    Professor of Medicine, Harvard Medical School
  • David C. Page, M.D.
    Director, Whitehead Institute
    Professor of Biology, Massachusetts Institute of Technology
    Howard Hughes Medical Institute Investigator
  • Barbara Vickrey, M.D., M.P.H.
    President and Vice Chair, Department of Neurology,
    David Geffen School of Medicine,
    University of California-Los Angeles
  • Tachi Yamada, M.D.
    Executive Vice President, Chief Medical and Scientific Officer,
    Takeda  Pharmaceuticals

About the Taubman Prize
Since 2007, A. Alfred Taubman has donated $100 million to U-M to establish the Taubman Institute, which currently is supporting the research of 18 doctor-scientists at U-M in diseases such as cancer, stroke and cardiovascular disease, ALS, diabetes and obesity, among others.  Part of his gift also supports the Taubman Prize.

Nominations for the 2013 Taubman Prize are being accepted now on the Taubman Institute website at www.taubmaninstitute.org .  Self-nomination is permitted, and the prize is open to all clinician-scientists worldwide with the exception of University of Michigan researchers, who are eligible for other Taubman Institute funding.  In keeping with the Taubman Institute mission to support high-risk, high-reward work by physician-scientists, clinical practice is an important component of the judging criteria, and all nominees must possess an M.D.

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Translational Medicine

Jul 10

Diabetic peripheral neuropathy, or nerve damage, causes pain or loss of feeling in the toes, feet, legs, hands, and arms among diabetic patients. About 70 percent of diabetes suffer from neuropathy. Current treatments for severe diabetic peripheral neuropathy include tricyclic antidepressants, anticonvulsants, opioids, serotonin and norepinephrine reuptake inhibitors, and topical agents.

“However, none of these therapies directly targets the inflammation, most have negative side effects and less than 30 percent of patients experience adequate pain relief. Clearly, new therapies are needed,” explains Dr. Ruth Waterman of the Ochsner Clinic Foundation in New Orleans and lead author of the study

However, a new preparation of stem cell treatment may target the inflammation that is thought to be the main cause of neuropathy.

Dr. Waterman recently collaborated with other researchers including Dr. Aline Betancourt from Tulane University’s Center for Stem Cell Research and Regenerative Medicine to develop a new stem cell treatment that controls the inflammation that causes diabetic peripheral neuropathy.

Previous studies have indicated that mesenchymal stem cells (MSCs) are safe and effective in treating inflammatory disease such as diabetic peripheral neuropathy. Unfortunately, as Dr. Betancourt explains:

“The problem is that the current methods for preparing these cells yield a mixed pool of undefined cells that aren’t consistently effective in the clinic. Our laboratory developed a new method that results in a consistent, uniform MSC population and optimizes their anti-inflammatory effects. We call these cells MSC2s.”

This new preparation of stem cell treatment had produced significant improvements in inflammation and other symptoms. Mice given the new stem cell treatment preparation saw a 40 percent improvement. Mice given the original preparation saw only a 14 percent improvement.

As Dr. Waterman concludes:

“Moreover the responses did not differ from what we saw in the healthy control group of mice. These findings suggest that the use of anti-inflammatory MSC2 is a promising new therapeutic strategy for diabetic peripheral neuropathy that should be further investigated.”

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Translational Medicine

Jul 09

On June 19 and 20, Clinical Research & Translational Medicine Workshop between Sun Yat-sen University and Johns Hopkins Medicine was held on South Campus and North Campus respectively. President Xu Ningsheng welcomed the delegation from Johns Hopkins Medicine (JHM) and expressed expectations of SYSU to establish a long-term strategic partnership with JHM. Vice president Li Mengfeng, Vice President Wei Minghai and nearly 150 representatives from various medical units of SYSU participated in the workshop.

Professor Adrian Dobs, Professor David Friedman, Professor Youseph Yazdi, Associate Professor Christoph Lehmann, Associate Professor Zhiping Li and Assistant Professor Rex Yung of the JHM delegation, Professor Li Mengfeng, Vice president of SYSU, Professor Yu Xueqing, Deputy President of The First Affiliated Hospital of SYSU, Professor Ma Jun, Deputy President of SYSU Cancer Center, Professor Weng Jianping, Deputy President of The Third Affiliated Hospital of SYSU, Professor Zhang Li, Deputy Director of Lung Cancer Research Center and Deputy Director of Internal Medicine of SYSU Cancer Center, Professor Shao Jianyong and Associate Professor Sun Jian of SYSU Cancer Center, held discussions on the status quo, problems and challenges of clinical research and translational medicine in SYSU. The JHM delegation also visited Zhongshan School of Medicine, The First Affiliated Hospital of SYSU, SYSU Cancer Center, and Zhongshan Ophthalmic Center.

During the workshop, representatives from SYSU and JHM held two internal sessions to discuss issues related to establishing joint research unit in the field of clinical research, jointly cultivating talents and undertaking research projects, and exchange views on the modes of cooperation. The success of the workshop is a prelude to collaboration and the establishment of strategic partnership between SYSU and JHM.

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