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| October 30, 2009. Students in UND's course, Developing Health Networks in Rare and Neglected Diseases (BIOS60570) attended class with Dr. Herman Vandenburgh, Professor of Pathology and Laboratory Medicine at Brown University and founder of Myomics, Inc. Dr. Vandenburgh 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 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 animal and human studies to determine the toxicity of new compounds. Dr. Vandenburgh's lecture, "Bioengineering Contractile Tissues for High Content Drug Screening Applications," was the fifth lecture in the CRND's Clinical Translational Seminar Series (CTS). (Photo: Will Wall) |
The Center for Rare and Neglected Diseases
Science for Society
Around the globe, people are suffering and dying from diseases that have generally been ignored by major pharmaceutical companies because the potential financial rewards for developing new drugs and vaccines to treat them are insignificant. These are the rare and neglected diseases of the world. There are tens of thousands of rare diseases, typically defined as those, which afflict less than 200,000. Among them are cystic fibrosis, thalassemia, Niemann-Pick C disease, and several of the rare forms of cancer. Neglected diseases, by contrast, can affect billions, but like their rare counterparts, have been ignored by researchers. This is especially prevalent in the developing world and in pockets of grinding poverty in developed nations, where the populations suffering from illnesses such as tuberculosis, malaria, and lymphatic filariasis do not have the money to buy drugs and vaccines, giving pharmaceutical companies little incentive to develop treatments that cost far more than could ever be recouped through reasonable prescription prices. The result is a gaping hole in healthcare—one which Notre Dame is uniquely positioned to fill.
Rare Diseases: those which afflict less than 200,000. Examples are cystic fibrosis, thalassemia, Niemann-Pick C Disease, adrenolekodistrophy and several forms of cancer.
Neglected Diseases: diseases of poverty, lymphatic filariasis disease pathologies of TB and Malaria.
Where We Are:
University of Notre Dame- College of Science
Center for Rare and Neglected Diseases
Galvin Life Science Building
Notre Dame, IN 46556
Email at: crnd@nd.edu
Phone (574) 631-3372
Fax (574) 631-9788
