Cindy and Ara Parseghian NPC Run Walk April 29 2009Rare disease research has a long history at Notre Dame, going back a few decades to the pioneering efforts of a handful of faculty in chemistry, biochemistry, and biology. These various lines of research were first brought together in 2001, when the initial charter for a rare disease research center was submitted for University approval.

In 2010, the University of Notre Dame and the Ara Parseghian Medical Research Foundation formed a partnership dedicated to finding a treatment for Niemann-Pick Type C (NPC). Through this partnership the University and the Foundation jointly fundraise for NPC research and support the administration of grants, internally and externally, to researchers around the globe.

Now, the University is poised to launch the Ara Parseghian Medical Research Fund that will take on the roles and responsibilities of the Foundation and drive the Foundation’s fundraising and research efforts to find a cure. The University is also pleased to announce Sean Kassen, PhD, as the new director of the Ara Parseghian Medical Research Fund.

Our joint success: cos_3883_4835312070_o

Researchers at the University of Notre Dame have targeted NPC since 2003. Research to fight NPC has rapidly developed through the Boler-Parseghian Center for Rare and Neglected Diseases led by Kasturi Haldar, the founding director and Rev. Julius A. Nieuwland professor of biologic
al sciences. Now over ten Notre Dame faculty are involved in NPC research including Crislyn D’Souza-Schorey, Holly Goodson, Kevin VaughanPaul HelquistOlaf WiestMalcolm Fraser, Jesus Izaguirre, Rich Taylor, Matt Champion, and Mary Beckman. Their work covers a wide range of approaches, computational and synthetic strategies in chemistry, cellular pathways, protein function and detection, engineering, clinical outreach as well as, induced pluripotent stem cells for NPC therapies.

As a result of the initial partnership in 2010, Notre Dame researchers established the William K. Warren Center for Drug Discovery and Development, a resource any member of the worldwide NPC research community may utilize for the design and synthesis of potential drug candidates. The Center enjoys multidisciplinary collaborations with Cornell, Columbia, Tufts, Washington University, and Texas Southwestern medical centers along with researchers at Purdue and Scripps Research Institute. Compounds produced in the Center are now under active investigation in all of these collaborators’ labs.

With partnerships through the Boler-Parseghian Center for Rare and Neglected Diseases, there have been multiple studies funded to identify the molecular mechanisms behind NPC disease, develop new NPC disease models, identify novel techniques to better understand the disease, and support new young investigators to perform research on NPC disease.

We are proud to acknowledge that the University of Notre Dame and their collaborators have made tremendous progress against NPC.  Of recent, they have developed a promising treatment discovered by medicinal chemistry Professor Paul Helquist and his collaborators. The treatment is currently undergoing FDA clinical trials.

With the ultimate goal of saving lives, Notre Dame will now take on this bold goal of finding a cure for NPC disease by creating the Ara Parseghian Medical Research Fund that will raise funds to sustain an energetic program of NPC research at Notre Dame and with collaborators worldwide.

We believe that this alliance will serve as a new and effective model for research on rare and neglected diseases.


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