*‡Leptin: From Bench to Clinical Applications

Christos Mantzoros, M.D., DSc, FACP, FACE, Chair
Harvard Medical School

Abstract:
Leptin, the prototype adipocyte secreted hormone, was discovered in 1994. Since then, intensive research efforts have altered significantly not only our view of the biology of obesity but have also changed our view of the adipocyte as a new endocrine organ. The first presenter will discuss lessons from basic research, the second presenter will discuss lessons learned from translational research which have repositioned the role of leptin in human biology and the third speaker will discuss emerging clinical uses of leptin in humans.

Presenters/Discussants
Christian Bjorbaek, PhD, Harvard Medical School and Beth Israel Deaconess Medical Center
"Leptin - a Thin Signal Without Weight?"

Christos Mantzoros, MD, Harvard Medical School
"Leptin: separating fact from fiction through translational research"

Christian Weyer, MD, Amylin Pharmaceuticals, Inc.
"Therapeutic potential of leptin in general obesity: Integrated neurohormonal approach"

*Fragile X-Associated Tremor/Ataxia Syndrome: Genotype, Animal Models, Phenotype and Intervention

Paul Hagerman, MD PhD, Chair
University of California, Davis, School of Medicine
Dept. Biochemistry and Molecular Medicine

Abstract:
Fragile X-associated Tremor/Ataxia Syndrome (FXTAS) is a recently discovered neurodegenerative disorder that may be one of the most common single-gene, late-onset forms of tremor and ataxia, and of dementia. Because the pathogenic trigger (RNA toxicity) is known, and can be recapitulated in animal and cell models, FXTAS represents a paradigm for studying the processes associated with neurodegeneration, and may therefore provide insights into downstream pathways in common with other degenerative disorders, such as Parkinson and Alzheimer diseases.

A newly-funded NIH Interdisciplinary Research Consortium at UC Davis and four satellite institutions, designated the NeuroTherapeutics Research Institute (NTRI), has been established to further define the pathogenic basis of FXTAS, to create appropriate animal models of the disorder, to develop targeted therapeutic agents for its treatment, and to engage in clinical trials for both existing neuroprotective agents and for future therapeutics that specifically target the underlying pathogenic mechanisms. NTRI also encompasses a postdoctoral training component that involves dual-mentorship of trainees to enhance the translational component of their training. NTRI encompasses four fundamental research components, which will be presented in the broader context of research in neurodegeneration: Molecular Pathogenesis (Dr. Paul Hagerman), Animal Models (Dr. Robert Berman), Clinical phenotype, and Interventions (Dr. Randi Hagerman, Dr. Maureen Leehey).

Presenters/Discussants
Paul Hagerman, MD, PhD, University of California – Davis
"Molecular pathogenesis of Fragile X-associated Tremor/Ataxia Syndrome"

Robert Berman, PhD, University of California – Davis
"FXTAS – Finding molecular targets through animal models"

Randi Hagerman, MD, University of California – Davis
"FXTAS – Phenotype and clinical presentation"

Maureen Leehey, MD, University of Colorado Health Sciences Center
"FXTAS - Development of novel intervention strategies"

*Workshop: Translating Your Ideas: Drug Development, Intellectual Property and the State of Academic-University Relations

Deborah Zucker, MD, PhD, Chair
AFMR and Tufts Medical Center

Abstract:
There are many steps along the path as you look to translate your novel scientific ideas and discoveries into beneficial treatments for patients. This Translational Medicine Development Workshop will provide researchers with an overview of the drug development process, beginning with approaches to innovation and the selection of promising ideas for development, through to the steps in drug assessment and clinical trials. It will look at issues of technology transfer for individual researchers, academic institutions, government funding sources and commercial entities. Attendees will learn more about the processes, potential funding options, and the promises and risks of translating scientific research into therapies. The Workshop will also address issues of public trust and conflicts of interest in the current changing landscape of Academic-Industry collaborations.

Presenters/Discussants
Mason Freeman, MD, Massachusetts General Hospital
"The Drug Development Process and Academic/Industry/Government Relationships"

Charles D. Smith, PhD, Medical University of South Carolina
"Your Idea and Your University: Issues in Academic Technology Transfer"

Indrani Mukharji, PhD, Northwestern University
"Patent & License Pearls and Pitfalls for Taking an Idea to the Marketplace"

Deborah Zucker, MD, PhD, AFMR and Tufts Medical Center
"Ethics & Academic Technology Transfer: Patients, Products and Public Trust"

Program Agenda
3:15	The Drug Development Process and Academic/Industry/Government Relationships Mason Freeman, MD, Massachusetts General Hospital
4:00	Your Idea and Your University: Issues in Academic Technology Transfer Charles D. Smith, PhD, Medical University of South Carolina
4:30	Patent & License Pearls and Pitfalls for Taking an Idea to the Marketplace Indrani Mukharji, PhD, Northwestern University
5:00	Ethics & Academic Technology Transfer: Patients, Products and Public Trust Deborah Zucker, MD, PhD, Tufts Medical Center & AFMR
5:20	Panel and Audience Discussion / Networking & Light Refreshments

*Adhesion Complex Related to Proteins in Myocardial Rhythm and Function

Robert S. Ross, M.D., Chair
University of California – San Diego
VA Healthcare San Diego

Abstract:
Cardiac hypertrophy and cardiomyopathy, with associated heart failure, are ever more important causes of morbidity and mortality. Identification of specific molecular pathways that are critical for contractile, electrophysiological, and structural abnormalities, as well as cardiomyocyte death and survival responses, of the hypertrophied and failing heart are necessary to identify new biologically targeted therapies directed at various stages of the disease process. Recently the importance of protein complexes involved in cell adhesion and ones localized in the intercalated disc, have been linked to cardiomyopathy in man. This session will focus on proteins in these complexes, and how they exert a major influence on hypertrophy, cardiomyopathy and arrhythmia by disrupting signal transduction, electromechanical function, and structural integrity of the myocardium. Future directions for study and relevance to potential therapies will be considered.

Presenters/Discussants
Robert S. Ross, MD, University of California – San Diego
"Introduction to the Session and then, Costameric proteins in Cardiac Dysfunction"

Ju Chen, PhD, University of California – San Diego
"Plakoglobin and Arrhythmogenic Right Ventricular Dysplasia/Cardiomyopathy."

Kirk U. Knowlton, MD, University of California – San Diego
"The role of the Coxsackie Adenovirus receptor (CAR) and CAR-interacting proteins in cardiac hypertrophy and failure."

Jeffrey E. Saffitz, MD, PhD, Harvard Medical School and Beth Israel Deaconess Medical Center
"Connexins in Cardiac Electrical and Contractile Function"

*Systems Biology Investigations of Glucocorticoid Efficacy in Tissue Remodeling

Robert J. Freishtat, MD, MPH and Eric P. Hoffman, PhD, Chairs
Children’s National Medical Center, Washington, DC

Abstract:
Synthetic glucocorticoids, such as prednisone and dexamethasone, are among the most widely prescribed drugs worldwide and are used to treat many inflammatory conditions. Glucocorticoids are very old drugs, yet they remain the standard of care for treatment of a variety of diseases including asthma, muscular dystrophy, autoimmune disorders, and arthritis. Other newer and more potent or targeted immunosuppressants have been tried in many of these disorders, but they have not shown the same efficacy as prednisone. A current paradigm is that glucocorticoids are anti-inflammatory in these disorders which leads to decreased tissue remodeling. However, an alternative model has emerged where the opposite is true; namely that glucocorticoids directly regulate pathological tissue remodeling which in turn reduces inflammation. This symposium will review this emerging model of glucocorticoid function in three disease states from a systems biology perspective: 1) Muscular Dystrophy, 2) Autoimmunity, and 3) Asthma.

Presenters/Discussants
Eric P. Hoffman, PhD, Children’s Natl. Med. Ctr., Washington, DC
"A model for efficacy of prednisone: re-synchronization of the tissue repair process."

Kanneboyina Nagaraju, MD, Children’s Natl. Med. Ctr., Washington, DC
"Functional and molecular effects of glucocorticoids on dystrophic skeletal and cardiac muscle: Role of novel glucocorticoid analogues."

William J. Jusko, MD, State University of New York at Buffalo, Buffalo, New York
"Systems pharmacology of anti-inflammatory effects of corticosteroids."

Robert J. Freishtat, MD, MPH, Children’s Natl. Med. Ctr., Washington, DC
"Protease/anti-protease balance and circadian influences on remodeling in lung."

*These symposia are supported by a grant from the National Center for Research Resources (NCRR), a component of the National Institutes of Health (NIH) and its contents are solely the responsibility of the authors and do not necessarily represent the official view of NCRR or NIH. ‡This program is funded in part by an educational grant from Amylin Pharmaceuticals, Inc. which had no control over its content. No personally identifiable information regarding you is provided to any grant supporter.”

Guidelines for Manuscripts Deriving From AFMR-sponsored Symposia at the EB Meetings >> Download Guidelines Manuscripts should conform to the general format for manuscripts published in the Journal of Investigative Medicine. These guidelines are available at http://edmgr.ovid.com/jim/accounts/ifauth.htm By design, the topics and scope of the symposia are designed to address important and timely topics that are relevant to the areas of clinical and translational research. Although the manuscripts submitted should reflect the content of the talks presented, the authors are encouraged to provide sufficient background material to provide context for the general readership of the Journal of Investigative Medicine. The use of summary schematics and figures is encouraged. Ideally, the structure of manuscripts derived from a symposium will reflect the organization of the symposium. In general, this will include one manuscript from each of the symposium talks. The submitted manuscript should reflect the information presented in each speaker's presentation. This would likely include both previously published as well as previously unpublished material. The talks will be introduced by a brief introduction written by the symposium organizer. Any different formats should be discussed with the Journal of Investigative Medicine editor. Authors should be careful to cite and obtain the requisite permissions in instances where they intend to use figures that have been published previously. All manuscripts should note the support provided by the NCRR by including the following notation with each manuscript in the acknowledgments: “This symposium was supported in part by a grant from the National Center for Research Resources (R13 RR023236)”.