Rare 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.
In 2016, the University launched the Ara Parseghian Medical Research Fund that took on the roles and responsibilities of the Foundation and is driving the Foundation’s fundraising and research efforts to find a cure. Sean Kassen, PhD, is the director of the Ara Parseghian Medical Research Fund.
Our joint success:
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 biological sciences.
Now over eight Notre Dame faculty are involved in NPC research including Holly Goodson, Kevin Vaughan, Paul Helquist, Olaf Wiest, Brian Blagg, Rich Taylor, John Koren, and Matt Champion. 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. Below are 3 grants that were recently funded by the APMRF at Notre Dame:
- Brian Blagg, Professor, Director of the Warren Family Research Center for Drug Discovery and Development, University of Notre Dame – Title: Investigations of NPC Disease and Lysosomal Storage Disorders through Computational and Medicinal Chemistry Studies
- Paul Helquist, Professor, University of Notre Dame – Title: Actively Targeted BBB-Crossing Cyclodextrin Derivatives for Treatment of NPC
- Kevin Vaughan, Associate Professor, Notre Dame – Stimulation of StARD9 as a Novel Mechanism to Overcome NPC Disease.
With the ultimate goal of saving lives, Notre Dame has taken on this bold goal of finding a cure for NPC disease by creating the Ara Parseghian Medical Research Fund that raises funds to sustain an energetic program of NPC research at Notre Dame and with collaborators worldwide.
This alliance serves as a new and effective model for research on rare and neglected diseases.
- Learn more about the Ara Parseghian Medical Research Foundation
Learn more about the Boler-Parseghian Center for Rare and Neglected Diseases.
Learn more about the William K. Warren Center for Drug Discovery and Development
- 2010 http://niemannpick.nd.edu/nd-parseghian-partnership/partnership-announcement
- 2016 http://news.nd.edu/news/66927-ara-parseghian-medical-research-fund-launches-at-notre-dame
- 2017 https://parseghianfund.nd.edu/research-supported-by-the-ara-parseghian-medical-research-fund-reaches-important-milestone/