BIOS 60570: A Course in Developing Health Networks in Rare & Neglected Diseases
Developing Health Networks in Rare & Neglected Diseases (BIOS60570) is an interdisciplinary course, which explores the development of health networks through the incubation of clinical research and service projects for rare diseases, such as Niemann-Pick Disease type C (NPC), and neglected diseases, such as malaria and lymphatic filariasis. Among other projects, students in the course will contribute to the development of clinical scores for NPC and medical notebooks, which will assist patients and families in communicating with health specialists. Students also participate in the CRND's Clinical-Translational Seminar Series (CTS), which offers them the opportunity to interact with some of the nation's foremost investigators and experts in clinical and translational medicine for rare and neglected diseases.
The Inaugural Course
January, 2009
A poster, "Developing Health Networks in Rare and Neglected Diseases: contributing to a clinical score for Niemann-Pick Disease, type C" was developed by participants in the inaugural seminar based on their work with Dr. F.D. Porter and Nicole Yanjanin, R.N., M.S.N. from the National Institutes of Health Program on Developmental Endocrinology and Genetics (NICHD, NIH, DHHS). The poster was presented at the Ara Parseghian Medical Research Foundation 2009 Scientific Conference on Niemann-Pick Disease type C (May 28-30, 2009 in Tucson, Arizona) by Jenny Shin and Daniel Castellanos. The course was created by Dr. Kasturi Haldar and Jenny. Shin, Program Coordinator.
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Participants in the inaugural seminar meeting in the winter of 2009. From left front, Sam Rund, Erin Brennan, Edwin Siu, Jenny Shin, Kinzie Kiser, Kasturi Haldar, and Daniel Castellanos. (Photo: Cheryl Kelly, 2009) |
Course II & The Clinical-Translational Seminar Series
Fall 2009
| September 4, 2009 - The Parseghian Family Journey & Foundation |
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Dr. Cindy Parseghian shares the story of her family's experience with Niemann-Pick Disease type C (NPC) and her work as the President of the Ara Parseghian Medical Research Foundation. Students also presented their research on other families who have children with NPC. The class explored the formation of relationships and networks between families, healthcare providers, public and private researchers, academic institutions, the National Institutes of Health, foundations, community organizations, advocacy groups, pharmaceutical companies, and other stakeholders. The Parseghian family's story was a focal point of the Fighting For The Lives of Children program, which aired on NBC at game half-time on September 5, 2009. (Photo: Will Wall, 2009) |
| September 11, 2009 - Journal Club with Professor Haldar |
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Dr. Haldar and the class enact a journal club discussion of a foundational paper by
Yanjanin et. al : "Linear clinical progression, independent of age of onset in Niemann- Pick Disease type C". Students focused on diagnostics, research tools, current therapeutic interventions, disease scores, and terminology as they practiced a first round of scoring a single clinical visit. The role of the literature in clinical-translational medicine comes to life as students gain hands-on experience with the broad cycle of knowledge. (Photo: Will Wall, 2009) |
| September 25, 2009 - Dr. F.D. Porter, NIH Investigator & Physician | |
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Forbes Dennison Porter, M.D., Ph.D., is a Senior Investigator and Chief for the Molecular Dysmorphology Section for the Program in Developmental Endocrinology and Genetics at the National Institute of Child Health Development. Dr. Porter's lecture on Smith-Lemli-Opitz Syndrome and Niemann-Pick Disease type C explored clinical-translational medicine in the context of his program at NIH. The Physician-Investigator's sensitive discussion of his clinical work with children and families at NIH helped UND students understand the profound connection between "the bench and the bedside". (Photos: Will Wall) |
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| October 2, 2009 - Nicole Yanjanin, Clinical Nursing-Research Perspective |
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| Nicole Yanjanin , R.N., M.S.N. works with children and families affected by Niemann-Pick Disease at the the Program in Developmental Endocrinology and Genetics, NICHD, NIH . She is the first author of the paper, "Linear clinical progression, independent of age of onset in Niemann- Pick Disease type C," which has been pivotal to students' understanding of NPC progression and the development of the NPC medical notebook project for this course. Ms. Yanjanin shared her insights as a Clinical Nurse-Researcher, helping students understand the multitude of considerations affecting data gathering, interpretation, and clinical scoring in the context of team-based patient care and family support . (Below) Jenny Shin, course co facilitator and Nicole Yanjanin demonstrating elements of the medical notebook. (Photo: Will Wall, 2009) |
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| October 9, 2009 - Dr. D'Souza-Schorey, Cell Biology & Therapeutics |
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Dr. Crislyn D'Souza-Schorey discussed her research on ADP -ribosylation factor 6 (ARF6) , which promotes the invasion and migration of cells in cancer. Dr. D'Souza-Schorey discussed the use of in vitro cells, biological and chemical assays, as well as mouse models, which provide an avenue for understanding the activity of the ARF6 cell signaling protein in humans. The class used the journal club format to review Dr. D'Souza-Schorey's paper, "Neurodegeneration in Niemann-Pick Type C Disease and Huntington’s Disease: Impact of Defects in Membrane Trafficking" (Schweitzer JK, Krivda JP, D'Souza-Schorey C., 2009). Teams of students presented figures from this paper, linking images to data and discussing issues with interpretation and levels of evidence. This seminar offered insight into the development of therapeutics in light of differentiated responses among individuals. (Photo: Will Wall, 2009) |
| October 16, 2009 - Dr. Tom Quinn, Research & Health Delivery in LCDs |
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Dr. Thomas Quinn is the Director of Johns Hopkins Center for Global Health. Dr. Thomas Quinn holds a B.S. and M.Sc. from UND and a medical degree from Northwestern University. Dr. Quinn discussed the development of health networks from a variety of perspectives, including: building research infrastructure and conducting field operations at overseas sites; organizing integrated systems to deliver health services, pharmaceuticals, and social education in less developed countries (LDCs); and recognizing opportunities for professional development and service throughout a long career. Dr. Quinn's lecture, "Challenges and Opportunities in Global Health: Different Approaches to Two Infectious Diseases Epidemics" helped students identify the complex issues and diversity associated with global health networks. (Photo: Will Wall, 2009) |
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| October 30, 2009 - Dr. Vandenburgh, High Content Drug Screening |
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| Dr. Herman Vandenburgh, Professor of Pathology and Laboratory Medicine at Brown University and founder of Myomics, Inc. discussed Myomics Inc.'s approach to drug screening, using a bioartificial muscle platform to screen well-studied FDA approved drugs. This screening identifies compounds, which can improve contractility or reduce muscle fatigue in patients with muscular disorders (e.g., Duschenne's muscular dystrophy). Myomic's strategy reduces the time and costs associated with typical drug development in which molecular target identification is followed by drug compound library screens to identify selective inhibitors of the target. Bypassing the steps of target identification, Dr. Vandenburgh screens compounds for a desired functional effect. By screening drugs that are already FDA approved and in clinical use, Dr. Vandenburgh hopes to avoid the need for expensive and extremely time-consuming animal and human studies to determine the toxicity of new compounds. (Photo: Will Wall) |
| November 6, 2009 - Dr. Austin, NIH's Molecular Libraries Probe Production |
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| Christopher Austin, M.D., Director of the NIH Chemical Genomics Center (NCGC) at the National Human Genome Research Institute (NHGRI), NIH. Dr. Christopher Austin discussed his work at NCGC, particularly his involvement with the Molecular Libraries Probe Production Centers Network (MLPCN). MLPCN is a network of small molecule screening centers, which perform High Throughput Screening for public sector researchers against over 300,000 chemically diverse small molecules. MLPCN's activities, including information about small molecules, which can be used as “chemical probes” to study genes and signaling pathways, will be made available through PubChem to public and private sectors. Bringing NIH's capacity for large-scale screening to the early stages of identifying chemicals, which modulate biological functions and assisting researchers with drug target validation, promises to make drug discovery for rare and neglected diseases economically viable. Dr. Austin's lecture, "Therapeutics for Rare and Neglected Diseases Program" discussed the evolution of small molecule screening at NIH. Above: Dr. Austin and Dr. Haldar in class. (Photos: Will Wall, 2009) |
