Notre Dame researchers and Niemann-Pick Type C

Program: WVPE Features

Reporter: Clara Ritger

Airdate: 04/07/2011

While the name "Ara Parseghian" usually brings to mind Notre Dame football, it's increasingly become associated with the race to find a cure for Niemann-Pick Type C. University of Notre Dame researchers have announced progress in treatment for the fatal brain disorder suffered by three of the former Irish head football coach's grandchildren. 

Notre Dame researchers are hoping to make changes in the lives of children with Niemann-Pick Type C, a fatal brain disorder, as the Notre Dame community announces a discovery in treatment for the disease.


Notre Dame professors Paul Helquist and Olaf Wiest co-authored the statement with other Notre Dame and Cornell University scientists.


Helquist: "Niemann-Pick Type C disease normally afflicts young children when they're about two or three years old; and it's 100 percent fatal. Typically the kids decline because of deterioration of their central nervous system over ten years and then they pass away; and there is no effective treatment up to this point in time" 

Children with the disease cannot properly process and metabolize cholesterol, which starts the process of cell degeneration in the brain.


Helquist: "What our studies show is that we can force the Niemann-Pick patient's cells to produce much larger quantities of the protein; and then in turn, in the cell studies that we've done, it completely corrects the flaw that we see in the disease."


Helquist says that they hope to see clinical trials of the treatment in humans within the year. If what they see in the cells in the labs translates to what they see in humans, this treatment would completely correct the disease.


Three of the four grandchildren, of former Notre Dame head football coach Ara Parseghian died of NP-C, and the University has been involved in research on the disorder for years. Last year, the university formally united with the Parseghian Foundation, and the Desert to Dome bike ride served as a way to raise money and awareness for research.


Dean of the College of Science Greg Crawford says he was intrigued by the passion of the Parseghian Foundation and the rest of the Notre Dame community in their search for a cure.


Crawford: "I decided to ride my bike, since I'm a physicist and not a biologist or a biochemist, that I would ride my bike from Tucson to South Bend, Indiana, with my wife, Renata, as to symbolically accept this gift and accept this challenge to move forward and try to fund researchers so that we can get to the bottom of this disease."


One of the biggest hurdles to finding the cure is raising enough awareness to get others to care about it. Last month the Notre Dame Cycling Team hosted the Midwest Collegiate Cycling Conference race in downtown South Bend to benefit the foundation.


Notre Dame graduate student Douglas Ansel is a contender for Rider of the Year and helped organize the race.


Ansel: "We can help the foundation in two ways. The first is to raise publicity, because this isn't a disease that is well known and unfortunately it's debilitating and people don't really realize how prevalent or how bad it is. The second is to raise funds that will help fund more research."


More about the Parseghian Foundation and NP-C research

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