Clinical Translational Seminar Series

 

The Center for Rare and Neglected Diseases welcomes the public to attend its Clinical Translational Seminar Series.

 

Please visit our Photohistory of the CTSS Seminar Series pages to see more about our past speakers (2009 - current).

 

 

 

The Fall 2013 CTSS Seminar Series

November 15, 2013 -Friday. DeBartolo Hall, RM. 138. 11 a.m. - 12 Noon

Michael Federle, M.D. Assistant Professor, University of Illinois at Chicago

My research focuses on discovering and understanding how bacteria communicate among themselves as a means for organizing group behaviors, especially behaviors facilitating the initiation and progression of disease in humans. Cell-to-cell communication in bacteria, termed Quorum Sensing, relies on a language of small, secreted signaling molecules called autoinducers.  Bacteria detect and respond to autoinducers through various types of receptor proteins sitting atop gene regulatory networks.  It is my goal to identify and describe the production and structure of new autoinducers and their cognate signal-transduction networks that contribute to the pathogenic state of the microorganism.  Our lab will use classic bacterial genetic and molecular biology techniques combined with conventional genomic, proteomic and metabolomic analyses to identify components and targets of these signaling systems.  Structural analysis of autoinducers and receptors, as well as screening for inhibitory compounds, will also be a focus of our work.  I am concentrating my efforts on Gram-positive pathogens, as these organisms pose the most current threat in developing resistance to multiple antibiotic treatments.  It is my hope that our research will lead to the development of new therapies that exploit and confuse communication systems bacteria use to organize attacks on the body.

 

The Fall 2013 CTSS Seminar Series

November 19, 2013 - Tuesday. LaFortune Student Center RM. 107. 11 a.m. - 12 Noon

Jeremy Burrows, M.D. Head of Discovery, Research & Development. Medicines for Malaria Venture

Dr. Jeremy Burrows leads the MMV Discovery team and supports groups across the portfolio with medicinal chemistry input.  Jeremy, a medicinal chemist, joins MMV’s Drug Discovery team from AstraZeneca, where he was an Associate Director in both Lead Generation and Lead Optimization Chemistry.  He has broad experience in drug discovery, from early target identification to lead optimization, and has led teams of chemists in the optimization of small molecules as future drugs for Alzheimer’s and Parkinson’s disease, rheumatoid and osteoarthritis, diabetes and the treatment of pain.

Jeremy has worked in the UK and Sweden. He holds a PhD in Chemistry from Oxford University and has published a significant number of scientific papers and patent applications.

 

The Fall 2012 CTSS Seminar Series Schedule & Guest Lectures

September 7, 2012 - Friday. Geddes Hall RM. B001. 11 a.m. - 12 Noon

Patrick E. Duffy, M.D. Chief, Laboratory of Malaria Immunology and Vaccinology, NIAID/NIH.

Dr. Duffy is the Chief of the Laboratory of Malaria Immunology and Vaccinology at NIAID/NIH with responsibility for NIAID's intramural program to develop and test malaria vaccines. Dr. Duffy's basic research is focused on understanding the pathogenesis and immunology of malaria in humans. Currently, he leads several groups, including the Pregnancy Malaria Initiative to develop a malaria vaccine for pregnant women, a Grand Challenges in Global Health consortium project to study immunity to severe malaria (SM) in African children, and a consortium of laboratories seeking to identify novel vaccine targets against liver stage malaria parasites. Dr. Duffy established the Seattle Malaria Clinical Trials Center, which is one of only four centers in the world with the mission of safely testing experimental malaria treatments and vaccines by the "malaria human challenge model." He also leads a training program for young East African scientists based in Tanzania.

Dr. Duffy received his medical degree from Duke University and trained in internal medicine training at Walter Reed. His postdoctoral training in molecular vaccine development was at NIH. Dr. Duffy is a graduate of West Point.

Seminar: Translating Malaria Immunity into Malaria Vaccines

 

September 21, 2012 - Friday. Jordan Hall RM. 310. 3 p.m. - 4 p.m.

Jose A. Bufill, M.D., F.A.C.P. Director, Cancer Genetics Program, St. Joseph Regional Medical Center, South Bend. Clinical Investigator, Northern Indiana Cancer Research Consortium. Private practice in Hematology-Oncology, Elkhart & South Bend, In.

Thrombotic thrombocytopenic purpura (TTP) is a rare and serious disorder, which can be life-threatening if untreated. According to the National Heart, Lung, and Blood Institute, "In TTP, blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop." TTP is caused by dysfunction of the normal metabolism of Von Willbrand factor (VWF) and may be either a congenital or inherited condition. The disorder is prone to relapse as the standard therapy of plasma exchange does not remedy the underlying cause of the disease. Recently, N-acetylcysteine (NAC) has shown promise in animal models as a potential therapy and a patient in South Bend with refractory, relapsing TTP who received NAC showed a temporary benefit as well and without significant side effects. Dr. Bufill will discuss this disorder and the further laboratory and clinical studies projected for NAC related compounds.

Dr. Bufill completed his medical degree at the University of Navarre in Pamplona, Spain and a medical residency and fellowship in medical oncology and hematology
at the Medical College of Wisconsin.

Seminar: Preliminary Observations using N-acetylcysteine for refractory TTP:

 

October 5, 2012 - Friday. Geddes Hall RM B001. 11 a.m. - 12 Noon

Carole Long, Ph.D. Chief, Laboratory of Malaria and Vector Research - Malaria Immunology Section, NIAID/NIH and Director, PATH Malaria Vaccine Initiative Growth Inhibition Assay-Reference Center.

The Laboratory of Malaria and Vector Research focuses on studies of malaria and insect vectors of infectious diseases with the aim of fostering collaborative research among the disciplines of the life sciences. Dr. Long's section investigates host-parasite interactions, both innate and adaptive, in rodent models, as well as children and adults in areas where the disease is endemic. Dr. Long is the director of the PATH Malaria Vaccine Initiative (MVI), an international nonprofit working to develop malaria vaccines and make them available across the globe, particularly in developing nations.

Dr. Long received her Ph.D. in microbiology and immunology from the University of Pennsylvania, where she also did her postdoctoral training.

Seminar: Progress toward development of a malaria vaccine

 

October 12, 2012 - Friday. Geddes Hall RM B001. 11 a.m. - 12 Noon

Dara Frank, Ph.D. Professor of Microbiology and Molecular Genetics, Medical College of Wisconsin and Director, Center for Infectious Disease Research.

Dr. Frank's research is focused on the interaction between hosts and bacterial factors involved in infections with P. aeruginosa and Francisella tularensis. Her exploration of the genetic regulation of exotoxin synthesis examines the relationship between the expression of toxins (e.g., toxins injected into eukaryotic cells that compromise the innate immunity in hosts), the pathogenesis of the bacterium, and host responses. Dr. Frank's lab played a leading role in the discovery and characterization of the P. aeruginosa type III system, which mediates the delivery of toxins. The Frank lab continues to explore enzyme inhibitors, antibody reagents, and vaccines, which neutralize these damaging toxins. Dr. Frank's work on the pathogenesis of F. tularensis has been part of a collaborative national biodefense program and seeks to identify virulence factors for drug and vaccine development.

Dr. Frank received her Ph.D. from the University of Texas at Austin.

Seminar: Molecular and Cellular Analyses of the Pseudomonas aeruginosa Cytotoxin, ExoU

 

October 26, 2012 - Friday. Geddes Hall RM B001. 11 a.m. - 12 Noon

George Dimopoulos, Ph.D., M.B.A. Professor, Department of Molecular Microbiology and Immunology, Director of the Parasitology Core Facility, and Deputy Director of the Malaria Research Institute, Bloomberg School of Public Health, Johns Hopkins University.

Dr. Dimopoulos investigates vector control for malaria and dengue fever, examining the mechanisms through which the mosquito's immune system and its intestinal microflora block pathogens. The aim of the Dimopoulos group is to develop human pathogen resistant mosquitoes. The group has produced genetically modified "super-immune mosquitoes" that are resistant to malarial parasites. Additionally, the group has identified intestinal bacteria in mosquitoes that block the malaria parasite and dengue virus.

Dr. Dimopoulos completed his Ph.D. in Biology at the University of Crete and the Institute of Molecular Biology and Biotechnology in Greece. His postdoctoral work was done at the European Molecular Biology Laboratory in Heidelberg, Germany.

Seminar: Basic science and translational aspects of human pathogen elimination in mosquito vectors

 

November 2, 2012 - Friday. Jordan Hall - Reading Room. 11 a.m. - 12 Noon

Timothy A. Grese, Ph.D. Chief Scientific Officer, Lead Generation and External Innovation Discovery Chemistry Research and Technology, Eli Lilly & Company.

In 2010, Dr. Grese accepted his current role as Chief Scientific Officer for Lead Generation and External Innovation at Eli Lilly & Company. His independent research contributions have focused on drug discovery in the areas of nuclear hormone receptors and therapeutics for osteoporosis and diabetes. Dr. Grese has been an innovator in the design and development of strategies and technologies advancing small molecule lead generation, including knowledge-based approaches to lead generation and structure-based design methodologies. To date, Dr. Grese's oversight of Lilly's Open Innovation Drug Discovery program has resulted in the establishment of novel academic-industrial drug discovery partnerships with Notre Dame, UC-Irvine, the NIH and others and he is continuing to explore new public-private partnership in early-stage drug discovery.

Dr. Grese holds a Ph.D. from Stanford University. His NIH postdoctoral fellowship was at UC-Irvine.

Seminar: Open Innovation Drug Discovery (OIDD): A New Platform for Academic-Industrial Collaboration

 

November 16, 2012 - Friday. Geddes Hall RM B001. 11 a.m. - 12 Noon

Vern B. Carruthers, Ph.D. Professor, Department of Microbiology and Immunology, University of Michigan Medical School.

Dr. Carruthers studies the pathogenesis of toxoplasmosis, which is caused by the protozoan parasite Toxoplasma gondii. Dr. Carruther's team has identified parasite virulence factors that function in host cell invasion, immune evasion, nutrient acquisition and lytic egress from host cells causing tissue damage and inflammation. Additionally Carruther's team has demonstrated the efficacy of novel experimental compounds for treating the chronic phase of infection and identified a panel of biomarkers that reveal extensive neuroinflammation during chronic infection.

Dr. Carruthers completed his Ph.D. in Microbiology at the University of Western Ontario and postdoctoral studies at The Rockefeller University and Washington University in St. Louis.

Seminar: Infection strategies of the highly successful neurotrophic parasite Toxoplasma gondii

 

November 30, 2012 - Friday. Geddes Hall RM B001. 11 a.m. - 12 Noon

Padmini Salgame, Ph.D. Professor and Director, Graduate Medical Research Program, University of Medicine & Dentistry of New Jersey. .

Dr. Salgame investigates host immunity and pathogenic mechanisms in infectious diseases, particularly Th1 immunity in tuberculosis, interleukin-12, T cell polarization, and dendritic cells. Utilizing transgenic models, Dr. Salgame's lab is clarifying the mechanism underlying Th1 initiation in tuberculosis, identifying the relationships of dendritic cells, macrophages, and T cells. The aim of Dr. Salgame's research is to discover new modalities for vaccine development and therapeutics based on immune response.

Dr. Salgame completed her Ph.D. in Immunology at Bombay University, India

Seminar: Intrinsic and Extrinsic Factors Regulating Host Anti-tuberculosis Defense

 

 

The Spring 2012 CTSS Seminar Series Schedule & Guest Lectures

 

March 2, 2012 - RM 138 Debartolo, 1-2 pm. on Friday

Marc C. Patterson, M.D. Chair - Division of Child and Adolescent Neurology, Professor of Neurology, Pediatrics and Medical Genetics, Director - Child Neurology Training Program Mayo Clinic. Dr. Patterson is board certified in psychiatry and neurology (with special qualification in child neurology and in neurodevelopmentaql disabilities.

Dr. Patterson investigates Niemann-Pick disease, type C (NPC) and other lysosomal diseases, and congenital disorders of glycosylation. His research experience includes laboratory studies at the University of Queensland (Neuropharmacology) and National Institutes of Health (NIH) (lysosomal diseases -LSDs) as well as participation in clinical studies, therapeutic trials and natural history studies at NIH, Columbia University and Mayo Clinic. Dr. Patterson's work explores the development of novel small molecule therapies for LSDs. He was principal investigator of a prospective trial of miglustat in NPC and of a retrospective study of the use of miglustat in NPC in clinical practice. Currently, Dr. Patterson is participating in the development of therapeutic protocols for metachromatic dystrophy and globoid cell leukodystrophy. He is a member of the Human Glycosylation Disorders Network, and a member of the Lysosomal Disease Network Steering Committee. In addition to many other awards, Dr. Patterson was honored in 2011 with the Compassionate Doctor Recognition and the Patients' Choice Award.

Dr. Patterson graduated from the University of Queensland (Australia) medical school and his internship and earlier residency training (Pediatrics, Internal Medicine, Neurology) was at the Royal Brisbane and Royal Children's Hospitals in Queensland. Subsequent residency training in Child Neurology and Pediatric/Adolescent Medicine was at the Mayo School of Graduate Medicine in Rochester, MN. His fellowship was at the Neurometabolic Disease, Developmental and Metabolic Neurology Branch, NIH, Bethesda, MD. His Clinical and Senior Clinical Fellowships in Pediatric Neurology were at the Mayo Clinic. Dr. Patterson is on the Scientific Advisory Board for the National Niemann-Pick Disease Foundation.

Seminar: An introduction to neurometabolic disease

 

March 9, 2012 - RM 138 Debartolo, 1-2 pm. on Friday

Mary Dunkle. Ms. Dunckle is the Vice-President for Communications for the National Organization for Rare Diseases (NORD)

Ms. Dunkle has oversight for all of NORD's website, publications, educational activities, and research programs. She has more than 2 decades of experience in journalism and public relations in the arena of medical research and communications. Ms.Dunkle moved to her position at NORD over 12 years ago. NORD is a national nonprofit organization comprising a federation of voluntary health organizations which serve the rare disease communities. NORD's mission is aimed at the identification, treatment, and cure of rare diseases through education, advocacy, research, and service. Ms. Dunkle describes the NORD website as "the central communications hub for patients, support organizations, medical professionals, policymakers and industry on topics related to rare diseases and orphan products." NORD's publications include: disease-specific booklets for physicians, electronic and print newsletters, and educational materials for patients, their families, medical professionals and the public.

Prior to undertaking her current post at NORD, Ms. Dunkle was the Manager of Penn State’s award-winning News Bureau and as Assistant Director of Public Relations for Penn State’s 22 campuses.

Seminar: The Role of the Rare Disease Patient Community

 

April 13, 2012 - RM 138 Debartolo, 1-2 pm. on Friday

Heiko Runz, M.D. Dr. Runz is a principal investigator and medical geneticist. He is a faculty member of the Molecular Medicine Partnership Unit (MMPU) at the University of Heidelberg and EMBL and a Junior Group Leader Molecular Metabolic Disease Unit, Institute of Human Genetics, University of Heidelberg

Dr. Runz completed his medical training at the University of Heidelberg (Germany), Harvard University (Boston), and Université Louis Pasteur (University of Strasbourg, France). Dr. Runz special interests include: cell biology of cholesterol metabolism and associated diseases; functional analysis of lysosomal storage disorders by advanced light-microscopy technology; identification of modifier genes for monogenetic lipid metabolic disorders.

The Molecular Medicine Partnership Unit (MMPU) is a collaboration between the European Molecular Biology Laboratory (EMBL) and the Medical Faculty of the University of Heidelberg for the purpose of discovering the molecular basis of disease and advancing personalized medicine. Dr. Runz currently co-leads the fifth of MMPU's 5 international teams, which is aimed at researching cell biology and disorders of cholesterol homeostasis.

Seminar: Niemann-Pick type C disease - from bedside to bench
and back


April 20, 2012 - RM 138 Debartolo, 1-2 pm. on Friday

Jacqueline Imrie, MSc. Clinical Nurse Specialist (Paediatrics). Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, UK

Ms. Imrie specializes in the treatment of patients with Niemann-Pick disease and metabolic disorders. She has co-authored of a number of studies on Niemann-Pick disease in the United Kingdom. Ms. Imrie brings a wealth of insight to rare diseases as both a practicing clinician of 30 years and a research investigator. Ms. Imrie has worked in many areas as a staff nurse, including general paediatrics, oncology, bone marrow transplant, cystic fibrosis, and neonatal intensive care. In 1999, Ms. Imrie became the first support nurse for families with Niemann-Pick disease throughout the UK and established a national database and help line.

Ms. Imrie holds an Honors degree from the Open University in Biochemistry and Genetics and a Master of Science degree in Genetic Counseling.

Seminar: Care and support for children and young adults with NPC in the UK

 

April 27, 2012 - RM 138 Debartolo, 1-2 pm. on Friday

Robert Erickson, M.D. - Holsclaw Endowed Professor in Genetics and Inherited Diseases (with tenure), Departments of Pediatrics and Molecular and Cellular Biology, University of Arizona COM, Tucson, Arizona. Board certifications in Pediatrics, Medical Genetics, Clinical Genetics, and Molecular Genetics.

Dr. Erickson's research interests include orofacial anomalies, lymphedema, and Native American diseases, mouse models of genetic disease, orofacial defects, and Niemann-Pick. His medical practice with the Genetics Section at U Arizona COM includes caring for many children and young adults with birth defects from a variety of causes, often genetic and sometimes resulting from substances. Recently, Dr. Erickson played a major part in the discovery of the gene for a rare form of facial clefting. Dr. Erickson is exploring the function of NP-C protein with an eye to finding medications to treat this condition.

Dr. Erickson is a leader in his field and his service is distinguished at state, national and international levels. He has served on the editorial boards of many publications, including current appointments to the Journal of Applied Genetics and Reviews in Mutation Research. He is the author of 4 books and over 400 scholarly articles, reviews, and other publications. After graduating his medical degree from Stanford's School of Medicine, Dr. Erickson completed residencies at Albert Einstein-Jacobi Hospital and NIH's Laboratory of Chemical Biology. His fellowships were at the National Institute of Medical Research Mill Hill London and the University of California - San Francisco. Dr. Erickson also served several years in the US Public Health Service.

Seminar: Niemann-Pick C1 disease: an introduction and the problem of lung involvement

 

 

The Fall 2011 CTSS Seminar Series Schedule & Guest Lectures

 

September 30, 2011

Mrs. Eva Luise Koehler and Horst Koehler, Ph.D. Mrs Kohler is the former First Lady of the Federal Republic of Germany, Patroness of the German Alliance for Rare Diseases (ACHSE), and previously a school teacher with experience with special needs students. Dr. Horst Koehler, former President of the Federa Republic of Germany, Managing Director and Chairman of the Executive Board of the International Monetary Fund.

Special Class Lecture: Q & A with the CRND Class and Facutly on International Rare Disease Collaborations

October 7, 2011 - Jordon Hall of Science Reading Room, 2-3 pm. on Friday

Karen Quandt, R.N., M.N. and Ty Quandt. Mrs. Quandt, RN, MN is the chair of the National Niemann-Pick Disease Foundation Board (NNPDF). Mrs. Quandt and her son Ty have first hand knowledge of living with NP-C and the vital work of the NNPDF with families, patients, researchers, educators and the public. The Quandts visited the CRND to teach a class in the Developing Health Networks in Rare and Neglected Diseases course.

Seminar: Niemann-Pick type C disease- Ty's story - told by Karen and Ty Quandt

 

October 28, 2011 - Jordon Hall Classroom 310

David Rhodes, Ph.D. Professor David Rhodes, Director of the Center for Plant Environmental Stress Physiology (CFPESP), Department of Horticulture, Purdue University. Dr. Rhodes is Board Member and Chief Scientific Advisor to PSC Partners Seeking A Cure. His lecture exploreed what is known and unknown about this rare disease.

Special Class Lecture: Primary Sclerosing Cholangitis (PSC) & PSC Partners Seeking A Cure

 

November 18, 2011 - Jordon Hall Classroom 310

Michael Pfrender, Ph.D. Faculty Director of the Genomics Core Facility and Associate Professor of Biological Sciences, and Faculty of the Eck Institute for Global Health, University of Notre Dame. Dr. Prender will share his expertise on advances in genomics and technology related to discovery in rare diseases.

Special Class Lecture: Genomic Medicine and Rare Diseases

 

December 2, 2011 - Jordon Hall Classroom 310

Mr. Michael McLaughlin. Senior Director - Regional Development at the University of Notre Dame.

Special Class Lecture: Blepharophimosis: A Family's Story

 

 

The Spring 2011 CTSS Seminar Series Schedule & Guest Lectures

 

March 4, 2011 - Nieuwland Hall (Rm 123), 1-2 pm

Steven U. Walkley, D.V.M., Ph.D. Director, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center. Head, Sidney Weisner Laboratory of Genetic Neurological Disease – Professor, Departments of Neuroscience, Pathology and Neurology, Albert Einstein College of Medicine in New York.

Dr. Walkley investigates the pathobiology and treatment of genetic neurological diseases, cerebral cortical development and ganglioside function in neurons. Dr. Walkley's research centers on the analysis of pathogenic cascades and the development of therapeutic strategies for genetic disorders of the endosomal-lysosomal system (Tay-Sachs, Hurler, Sanfilippo, Niemann-Pick, Batten), which cause progressive neurological dysfunction and premature death. Dr. Walkley's studies include animal models of storage diseases. Studies explore the link between the primary protein defect, the abnormal accumulation of substrate (gangliosides, glycosaminoglycans, cholesterol), and subsequent changes in trafficking and signaling events within affected neurons. Therapeutic strategies addressed in Dr. Walkley's work include replacement of missing proteins through cell-mediated approaches (e.g., bone marrow transplantation) and substrate reduction therapy.

Dr. Walkley joined the faculty of the Albert Einstein College of Medicine in 1982 after completing graduate and postgraduate studies at the University of Alabama in Birmingham, the University of Edinburgh, Auckland University in New Zealand and the Albert Einstein College of Medicine. Dr. Walkley's early training in veterinary and comparative medicine enabled his work in the identification and development of diseases in domesticated and laboratory animals. Dr. Walkley is on the Scientific Advisory Board for the National Niemann-Pick Disease Foundation. In 2007, he joined several other scientists in the formation of the NPC-SOAR project, the goal of which is the accelerated development of a therapy for Niemann-Pick type C disease.

Seminar: The Art of the Soluble: Cyclodextrin and Niemann-Pick C Disease

 

March 25, 2011 - Nieuwland Hall (Rm 123), 1-2 pm

Forbes Dennison Porter, M.D. Ph. D. - Senior Investigator, Chief: Section on Molecular Dysmorphology Program in Developmental Endocrinology and Genetics, National Institute of Child Health Development (NIHCD), National Institutes of Health.

Dr. Porter is on the Scientific Advisory Board for the National Niemann-Pick Disease Foundation.

Seminar: Development of Biomarkers for Niemann-Pick Disease, type C

 

April 1, 2011 - Nieuwland Hall (Rm 123), 1-2 pm

Daniel S. Ory, M.D. Professor of Medicine, Cell Biology and Physiology, Cardiovascular Division, Washington University School of Medicine, St. Louis, MO

Dr. Ory's work focuses on the mechanisms regulating cholesterol homeostatic pathways and the development of biomarkers for prevention and treatment of related human diseases. His approach is multidisciplinary, including biophysical, cell biology and steroid chemistry methods and his studies employing unbiased genetic screens have led to new discovery of small RNAs that regulate cellular cholesterol homeostasis. Candidate molecules identified by his approach are being examined in mouse models of atherosclerosis. Dr. Ory is also investigating the function and regulation of the Niemann-Pick C1 (NPC1) protein in cell-based and animal models. His studies use high throughput screens to identify small molecule “chaperones” that correct the protein-folding defect responsible for NPC1 disease and candidate compounds are being investigated in vivo in a mouse model of NPC1 disease. Using mass spectrometry-based lipidomic, Dr. Ory's team has identified candidate lipid metabolites that are being validated in clinical studies as biomarkers in human disorders associated with oxidative stress, including diabetes and Niemann-Pick C disease.

 Dr. Ory's followed his undergraduate training at Harvard College and attained his medical degree from Harvard Medical School in 1986. His internship, residency in medicine, clinical fellowship and research fellowship were at Brigham & Women's Hospital and Harvard Medical School. In the early nineties, Dr. Ory was a visiting scientist at the Whitehead Institute (MA) and Instructor in Medicine at the Harvard Medical School. In 1995, he moved to Washington University School of Medicine where he rose to Professor of Medicine (Cardiology) and Cell Biology & Physiology. In 2007, Dr. Ory became the Co-Director of the Diabetic Cardiovascular Disease Center at Washington University School of Medicine. Dr. Ory is the Faculty Director of Admissions at WU and a fellow of AAAS. Dr. Ory is on the Scientific Advisory Board for the National Niemann-Pick Disease Foundation.

Seminar: Niemann-Pick C Disease: Strategies & Challenges for Discovery of New Therapies

 

April 15, 2011 - Nieuwland Hall (Rm 123), 1-2 pm

Marc C. Patterson, M.D. Chair - Division of Child and Adolescent Neurology, Professor of Neurology, Pediatrics and Medical Genetics, Director - Child Neurology Training Program Mayo Clinic.

Dr. Patterson investigates Niemann-Pick disease, type C (NPC) and other lysosomal diseases, and congenital disorders of glycosylation. His research experience includes laboratory studies at the University of Queensland (Neuropharmacology) and National Institutes of Health (NIH) (lysosomal diseases -LSDs) as well as participation in clinical studies, therapeutic trials and natural history studies at NIH, Columbia University and Mayo Clinic. Dr. Patterson's work explores the development of novel small molecule therapies for LSDs. He was principal investigator of a prospective trial of miglustat in NPC and of a retrospective study of the use of miglustat in NPC in clinical practice. Currently, Dr. Patterson is participating in the development of therapeutic protocols for metachromatic dystrophy and globoid cell leukodystrophy. He is a member of the Human Glycosylation Disorders Network, and a member of the Lysosomal Disease Network Steering Committee.

Dr. Patterson graduated from the University of Queensland (Australia) medical school and his internship and earlier residency training (Pediatrics, Internal Medicine, Neurology) was at the Royal Brisbane and Royal Children's Hospitals in Queensland. Subsequent residency training in Child Neurology and Pediatric/Adolescent Medicine was at the Mayo School of Graduate Medicine in Rochester, MN. His fellowship was at the Neurometabolic Disease, Developmental and Metabolic Neurology Branch, NIH, Bethesda, MD. His Clinical and Senior Clinical Fellowships in Pediatric Neurology were at the Mayo Clinic. Dr. Patterson's certifications include: Neurology (Royal Australasian College of Physicians), Neurology with special qualification in Child Neurology (American Board of Psychiatry and Neurology), and Neurodevelopmental disabilities (American Board of Psychiatry and Neurology). Dr. Patterson is on the Scientific Advisory Board for the National Niemann-Pick Disease Foundation.

Seminar: Niemann-Pick disease, type C - challenges and opportunities in a rare disease

 

April 18, 2011 - Nieuwland Hall (Rm 118), 3-4 pm

Christopher McClendon, Ph.D. Candidate, University of California -San Francisco

Christopher McClendon will present novel methods to identify statistically significant correlated motions and population shifts from equilibrium molecular dynamics simulations, using a program he developed called "MutInf" Demonstrating the application of these methods to a number of proteins to provide insights into allosteric mechanisms and to identify novel allosteric pathways in any protein of interest, McClendon shows new experimental results on caspase-1, where he uses MutInf to guide mutational analysis and assist in elucidating the mechanism of cooperativity.

Seminar: Quantifying Correlated Motions, Altering Allosteric Networks

 

The Fall 2010 CTSS Seminar Series Schedule & Guest Lectures

 

September 3, 2010 - Jordon Hall ( RM 105), 1-2 pm

Robert Murphy, M.D.  John Phillip Phair Professor of Infectious Diseases, Feinberg School of Medicine, Division of Infectious Diseases, Northwestern University Medical School;   Director, Global Health Center

Dr. Murphy is an Attending Physician in infectious diseases at Northwestern Memorial Hospital.  In the early 90's, Dr. Murphy supported AIDS prevention training efforts for U.S. Peace Corps physicians caring for volunteers in Western Africa. In 1996, he founded the International AIDS Education Project and serves as its director. In 2003, Dr. Murphy was appointed Nigeria Country Director for the President's Emergency Plan for AIDS Relief (PEPFAR). In 2008, Dr. Murphy received two Fogarty International Research Collaboration Awards to develop international research partnerships between NIH-supported U.S. scientists and collaborators in Mali, Nigeria, and other countries in the developing world.

Seminar: Impact of scaling up HIV therapies in resource-limited settings: morbidity, mortality and cost

 

September 10, 2010 - Jordon Hall ( RM 105), 1-2 pm

RADM Sandy Kweder, M.D.  United States Food and Drug Administration, Deputy Director, CDER Office of New Drugs (OND);  Associate Professor of Medicine at the Uniformed Services University of the Health Sciences.

RADM Dr. Sandra Kweder is involved in all OND drug oversight activities, including over 700 scientific professionals. She oversees the Pediatric and Maternal Health group, which implements the Best Pharmaceuticals for Children Act and product labeling. She also oversees the Study Endpoints and Labeling Development team, which supports the science of endpoint assessment in clinical trials and coordination of drug labeling activities for CDER.  Dr. Kweder joined the FDA in 1988 as a medical reviewer in the Division of Antiviral Drugs, and she has served in the leadership of the Division of Postmarketing Surveillance and Epidemiology and of the Office of Antimicrobial Products. RADM Kweder continues to practice clinical medicine and teaches at the National Naval Medical Center.

Seminar: Drugs and FDA: Demystifying the interface of science and public policy

 

October 1, 2010 - Jordon Hall ( RM 105), 1-2 pm

Olaf Schneewind, M.D., Ph.D. Louis Block Professor and Chair of the Department of Microbiology University of Chicago;  Director, Great Lakes Regional Center of Excellence for Biodefense and Emerging Infectious Diseases Research (RCE)

Dr. Schneewind is an infectious disease expert who has developed a variety of techniques to improve the study of protein targeting in pathogenic bacteria. He has been involved in translational research, consulting with many pharmaceutical companies. As the Director of the Great Lakes Regional Center of Excellence (RCE) for biodefense and emerging infectious disease research, Dr. Schneewind supports close collaboration between 20 area medical research centers to prevent biological terrorism and emerging diseases such as SARS, anthrax and West Nile Fever. Dr. Schneewind played a key role in organizing the Regional Biocontainment Laboratory (Biosafety Level 3 facility).

Seminar: On the pathogenesis and prevention of Staphylococcus aureus infections

 

October 8, 2010 - Jordon Hall ( RM 105), 1-2 pm

Philip E. Coyne Jr., M.D., M.S.P.H. CAPT, US Public Health Service, Department of Preventive Medicine and Biometrics; Assistant Professor of Tropical Public Health, F. Edward Herbert School of Medicine, Uniformed Services University of the Health Sciences

Dr. Coyne is an alumnus of the University of Notre Dame and the Peace Corps. He is soon to join the USAID Neglected Tropical Diseases program. Previously, Dr. Coyne served as: a Program Officer for Antiparasitic Drug Development at NIH;  the Associate Director for Regulatory Affairs, Division of Experimental Therapeutics at the Walter Reed Army Institute of Research; Consultant for Tropical Medicine, Onchocerciasis Coordination Unit Africa Region, The World Bank; and Medical Review Officer, Antiparasitic Drugs Division of Special Pathogens & Immunologic Drugs, CDER, FDA.

Seminar: Drugs for Neglected Diseases Initiative and Product Development Partnerships

 

October 29, 2010 - Jordon Hall ( RM 105), 1-2 pm

Anne Rowley, M.D. Professor of Pediatrics and of Microbiology-Immunology, Feinberg School of Medicine, Northwestern University

Dr. Rowley is also currently an Attending Physician in the Infectious Diseases Division of Children's Memorial Hospital. Her laboratory focuses on the pathogenesis of Kawasaki Disease, an acute, potentially fatal vasculitis of young childhood. Kawasaki Disease has replaced acute rheumatic fever as the most common cause of acquired heart disease in children in the US and other developed nations.

Seminar: Synthetic Kawasaki Disease antibodies identify cytoplasmic inclusion bodies in Kawasaki Disease tissues

 

November 12, 2010 - Jordon Hall ( RM 105),1-2 pm

Michael Gottlieb, Ph. D.  Associate Director for Science, Foundation for the National Institutes of Health (FNIH); Deputy Director for Grand Challenges in Global Health (Bill & Melinda Gates Foundation)

Dr. Gottlieb is the Co-Principal Investigator for the Malnutrition and Enteric Diseases Project (Mal-Ed). Mal-Ed is funded by the Fogarty International Center (FIC), part of the National Institutes of Health, and the Bill & Melinda Gates Foundation.  Mal-Ed will establish sites in the developing world for researchers to identify the risk factors for malnutrition, intestinal diseases and health consequences (e.g., developmental impairment, in children). One in every five children in the developing world is malnourished and half of all child deaths worldwide result from poor nutrition. Mal-Ed aims to develop models to estimate the distribution and burden of malnutrition and intestinal infections and identify beneficial interventions. FNIH was established by Congress to support the mission of the National Institutes of Health – improving health through scientific discovery.  The foundation identifies and develops opportunities for innovative public-private partnerships involving industry, academia, and the philanthropic community.

Seminar: The MAL-ED Project: Towards a better understanding of the relationship between malnutrition and enteric diseases

 

December 3, 2010 - Jordon Hall ( RM 105), 1-2 pm

Peter Hotez, M.D., Ph. D.  Distinguished Research Professor, George Washington University; Walter G. Ross Professor & Chair, Department of Microbiology, Immunology, & Tropical Medicine, George Washington University Medical Center; President, Sabin Vaccine Institute; Editor-in-Chief, PLoS Neglected Tropical Diseases

Dr. Hotez has particular expertise in 1) vaccine development for hookworm and other human helminth infections and 2) global policies and advocacy for the control and elimination of the human helminth infections and other neglected tropical diseases. His research focuses on the discovery, development and evaluation of technologies for treatment and eradication of neglected tropical diseases, specifically vaccines, diagnostics and drugs. Currently, he serves as the Principal Scientist of the Human Hookworm Vaccine Initiative (HHVI), a public private partnership sponsored by the Sabin Vaccine Institute with major funding from the Bill and Melinda Gates Foundation. Dr. Hotez is a leading advocate and educator in the area of NTDs, particularly in his work for PLoS.

Seminar: Innovation in the control of the neglected tropical diseases

 

 

Spring 2010 CTSS Seminar Series Schedule & Guest Lectures

 

January 22, 2010

Patrick Lammie, Ph.D. - Senior Scientist with the Division of Parasitic Diseases at the Center for Disease Control and Prevention.

Seminar: Elimination of Lymphatic Filariasis in Haiti: Where do we go from here?

 

February 5, 2010

Dr. Marie-Denise Milord, M.D. - Postdoctoral Researcher in The University of Notre Dame Haiti Program

Special Class Lecture: Control of Malaria and Lymphatic Filariasis in Haiti


February 26, 2010

Terrie Taylor, D. O. - University Distinguished Professor of internal medicine in the College of Osteopathic Medicine, Michigan State University. Director, Pediatric Malaria Research, Queen Elizabeth Hospital, Blantyre Malawi

Special Class Lecture: Severe Malaria in African Children: Clinical Features and Pathogenesis

March 26, 2010

Nicole Yanjanin , R.N., M.S.N. - Program in Developmental Endocrinology and Genetics, NICHD, NIH. N. Yanjanin, Nurse & Investigator

Special Class Lecture: Clinical Progression in Niemann-Pick Disease Type C

 

April 9, 2010

Forbes Dennison Porter, M.D. Ph. D. - Senior Investigator, Chief: Section on Molecular Dysmorphology Program in Developmental Endocrinology and Genetics, National Institute of Child Health Development (NIHCD), National Institutes of Health.

Special Class Lecture: Biomarker development and clinical aspects of Niemann-Pick Disease, type

 

April 16, 2010

Cate Walsh Vockley, M.S., C.G.C.- Genetic Counselor, Coordinator of Education, Referral and Advocacy for the National Niemann-Pick Disease Foundation (NNPDF).

Special Class Lecture: Rare Diseases: A Family-Focused Perspective

 

 

The Fall 2009 CTSS Seminar Series Schedule & Guest Lectures


Sept 4, 2009

Cindy Parseghian, MBA, Ph. D. - President, Ara Parseghian Medical Research Foundation.

Seminar: Parseghian Family Journey

 

Sept 11, 2009

Kasturi Haldar, Ph.D. - Course instructor. Julius Nieuwland Chair of Biological Sciences and Director of the Center for Rare and Neglected Diseases, University of Notre Dame.

Lecture: Journal Club

 

Sept 25, 2009

Forbes Dennison Porter, M.D. Ph. D. - Senior Investigator, Chief: Section on Molecular Dysmorphology Program in Developmental Endocrinology and Genetics, National Institute of Child Health Development (NIHCD), National Institutes of Health.

Seminar: Smith-Lemli-Opitz Syndrome and Niemann-Pick Disease type C: Bench to Bedside and Bedside to Bench.

October 2, 2009

Nicole Yanjanin , R.N., M.S.N. - Program in Developmental Endocrinology and Genetics, NICHD, NIH. N. Yanjanin, Nurse & Investigator

Special Class Lecture: Linear clinical progression, independent of age of onset in Niemann- Pick Disease type C

 

October 9, 2009

Crislyn D’Souza-Schorey, Ph. D. - Walther Cancer Institute Associate Professor, Department of Biological Sciences, Center for Rare and Neglected Diseases, The University of Notre Dame

Seminar: Understanding the cellular basis of Niemann Pick Type C pathogenesis

 

October 16, 2009

Thomas Quinn, M.D., M.S. - Director, Johns Hopkins Center for Global Health, Associate Director for International Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health

Seminar: Challenges and Opportunities in Global Health: Different Approaches to Two Infectious Diseases Epidemics

 

October 30, 2009

Herman Vandenburg, Ph. D. - Professor of Pathology and Laboratory Medicine (Research), Brown University, Founder and Scientific Advisor, Myomics, Inc.

Seminar: Bioengineering Contractile Tissues for High Content Drug Screening Applications.

 

November 6, 2009

Christopher P. Austin, M.D. - Director, NIH Chemical Genomics Center, National Human Genome Research Institute (NHGRI), National Institutes of Health.

Seminar: Therapeutics for Rare and Neglected Diseases Program

 

November 13, 2009

Terrie Taylor, D. O. - University Distinguished Professor of internal medicine in the College of Osteopathic Medicine, Michigan State University. Director, Pediatric Malaria Research, Queen Elizabeth Hospital, Blantyre Malawi

Karl Seydel , M.D., Ph.D. works with Dr. Taylor in Malawi and is an Assistant Professor at Michigan State University 's Department of Internal Medicine, College of Osteopathic Medicine.

Seminar: Malaria: cerebral disease and fever

 

 

 

Of Interest...