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| November 6, 2009. Students in UND's course, Developing Health Networks in Rare and Neglected Diseases (BIOS60570) attended class with 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" was the sixth lecture in the CRND's Clinical Translational Seminar Series (CTS). Above: Dr. Austin and Dr. Haldar in class. (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
