The Newsletter of the
Ara Parseghian Medical Research Foundation
During my years of coaching, I tried my best to put the right players in the right positions at the right times. It usually worked out, but not always. It wasn’t any easy task.
Now, I am humbled by the volunteers and supporters like you who step onto the field to offer time, talent and backing in the quest to find a treatment and cure for Niemann Pick Type C. You are the right players in the right positions and you are helping out at the right time…and I thank you so much for this.
With your generous support, we are now funding research at 24 labs around the world. You are ultimately responsible for research that isolated the gene that causes NP-C and in the process discovered a previously unknown cholesterol pathway. Implications are far-reaching, not only for those suffering from NP-C, but also Alzheimer’s, Tay-Sachs, Fabry, Gauchers, Batten, and Sandhoff. We have one drug in trial and hopefully a second drug will begin soon.
Key to this research effort are visionary sponsors of our upcoming 12th annual “One More Victory, Ara!” Celebrity Golf and Gala Weekend Presented by University Medical Center. In addition to UMC, our title sponsor, our other event sponsors include Precision Toyota, Jim Click Automotive Team, Bon Voyage Travel, Wells Fargo, La Encantada and The Westin La Paloma Resort & Spa. The Foundation’s signature fund-raising event has also enjoyed ongoing support from celebrities and sports legends, and I pay tribute to their dedication in our fight for life.
Coaches don’t just win games…players do. During the game, you have the feeling that you are part of something bigger than yourself; but it is not until the game is over that you realize you have made history. We are in the middle of the game with NP-C, but I recognize this feeling: it is history in the making and each of you is part of it. We will carry the ball…the cure…over the goal line together.
NP-C Natural History Study
The Parseghian Foundation in collaboration with the National Institutes of Health (NIH) has launched an important project called the NP-C Natural History Study.
For years NP-C researchers and clinicians have expressed concern over the lack of clinical information about NP-C patients. When the NIH stepped forward to conduct this study, the APMRF set right to work in recruiting families to participate.
There are three main reasons why this study is so important:
- The consistent clinical data will be useful to all physicians with NP-C patients.
- Data is urgently needed by researchers to develop therapies.
- Hopefully new biomarkers, indicators used during a drug trial to see if the drug is working or not, will be discovered. Presently there are only 1-2 biomarkers for NP-C. Better ones are needed in order for the FDA to approve drugs.
According to Dr. Forbes “Denny” Porter who heads the study, the enrollment rate has been much higher than anticipated. This is good news.
In total 15 patients of the potential 25 have enrolled in the study as of March 2007. They represent 6 females and 9 males, ages 3 to 32 and travel to the NIH in Bethesda, MD every six months. Over a period of 3-4 days the NP-C patient participates in a number of clinical tests. We are very grateful to these families and patients for committing to this important study.
Four patients in the study have now returned for their second visit. The comparison of the same tests over this six-month period will give a great deal of clinical information. Although interesting indications have been observed that could be meaningful if they continue with the second visit of all the patients, it’s too early to make definitive statements about certain outcomes.
The families who have taken part have been very complimentary of Dr. Porter, of the study nurse Nicole Yanjanin, as well as the specialists who are conducting the numerous tests. Although it is a big commitment for these families, they are reassured to know their child is being seen by clinicians who are also following 15-20 other NP-C patients. And, it’s one more way to contribute to the battle against NP-C.
The following was taken from an email message sent to the APMRF from Laurie Friedl, an NP-C mother who enrolled her daughter in the study: “We’ve returned home from our trip to NIH...Dr. Porter and his nurse are wonderful! It was a long week, but we’re very glad we went. Ashton actually cried when we were leaving, because she wanted to live at the Children’s Inn. It’s amazing how quickly kids forget about the bad things and focus on the good!
We wanted to thank you again for informing us about the study. If there are other parents who are considering becoming involved, we’d be happy to talk to anyone.” Laurie may be contacted at: firstname.lastname@example.org
If any NP-C family would like more information about enrolling their child in the study, please contact Nicole Yanjanin at: email@example.com or Glen Shepherd at firstname.lastname@example.org.
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The Smith Family...A Common Bond
The Smith family of West Lafayette, Indiana and the Parseghian family of Tucson share common bonds. Each was blessed with four children. Although days were hectic caring for and loving four little ones, life was good. Until, sadly, the three youngest children in each family were diagnosed with Niemann-Pick Type C disease.
Another common bond is through Notre Dame sports. Grandpa Ara was the legendary football coach at Notre Dame. Julie Smith’s cousin, David Schrage, is the head baseball coach for the Fighting Irish.
Chandlar Smith (14), the eldest child of Trent and Julie Smith, is NPC-free. Chandlar’s brother Braden died from complications of NP-C in October 2006 at the age of 10. He is missed dearly by his family and friends. Riley, age 8, and Keaton, age 6, show some symptoms although are doing fairly well.
Chandlar is a cheerleader, on Student Council, and is in the school and church choir. Riley is a Brownie Girl Scout, loves to swim and play softball. Last summer she won the league-wide Sportsmanship Award. Keaton is in Kindergarten and enjoys playing many sports and spending time with dad on the golf course. Julie is a stay-at-home mother and community volunteer. Trent works for Eli Lilly and Company as a pharmaceutical team production leader.
“We cherish every smile and every word they say,” says Julie. “We take life a bit slower, just to appreciate the little things.” The Smiths, like many NP-C families, are determined to give the children as much happiness as they can for as long as they can.
Trent and Julie are known to their neighbors and friends as humble, grateful people. One neighbor said, “Julie handles anything, she goes with the flow...she tries to keep family activities going and that is what keeps her going.” The Smiths have tried hard to raise Chandlar as a normal girl, and her father says she is “a big help to us” in caring for the other children.
The Smiths and friends have hosted a golf tournament to benefit NP-C research and are doing all that they can to keep apprised of the latest developments in NP-C therapy research. Keaton is enrolled in the Zavesca clinical drug trial for NP-C, and they are considering enrolling Riley in the NP-C Natural History Study at the NIH.
Above all, the most common bond shared between the Smiths and the Parseghians is the deep love they feel for their children…and the shared passion to find a treatment and cure for Niemann-Pick Type C disease as quickly as possible.
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In his letter to the APMRF Steve Mulvey, President of Quail Valley Charities, wrote, “We are proud to present to you our check in the amount of $25,000. We hope that these proceeds from our 2007 Quail Valley Golf Club Charity Cup will help find a cure for Niemann-Pick Type C Disease.”
Each year these heartfelt folks from Vero Beach, FL raise more than $250,000 which is distributed to local children’s and educational charities, and to the APMRF on a national level. This annual golf tournament has been so successful that they have won a national philanthropic award for their work.
For two years the owners and Club members have warmly welcomed Ara and Katie, and Cindy and Mike Parseghian to Vero Beach for a wonderful weekend of golf and festivities…all in support of worthy causes.
“Hearts & Hands Together” is a most fitting logo for IBM’s Employee Charitable Contribution Campaign (ECCC) Since the inception of the APMRF many thoughtful IBM employees and retirees across the country have been supporting the fight against NP-C disease.
In 2006 they pledged more than $5,000 to the APMRF through the ECCC.
IBM is one of many companies across the US that offers employees the opportunity through payroll or pension deductions to give to the their favorite not-for-profit IRS 501(c)(3) health and human services agencies.
Our sincere thanks to the IBM family for their generous support of the Parseghian Foundation.
Jessica Bolhack, Alex Ricker, Anne Chiorazzi, Perri Blazer and John Bennett--all classmates and friends of Marcia Parseghian--have spent many hours raising money to fund NP-C research to help save the life of their good friend. Marcia is gone, but their dedication lives on. It has been seven years since this wonderful group of young adults graduated from Sunrise Drive Elementary School in Tucson, but in many ways their hearts are still there.
This past fall, under the leadership of Jessica Bolhack, this group returned to Sunrise Drive School to share with students the important role they too can play in helping fight the disease that took the lives of the three Parseghian children.
Pennies are adding up in the decorated collection buckets that have been placed in all of the classrooms. In just a few short months over $650 has been collected.
As a result of the initial Sunrise Penny Drive that began 12 years ago, many of the district schools also collect pennies and to date over $80,000 has been raised.
Verna Burrows, grandmother to 10 year-old Ty who has NP-C, did another super job of organizing, promoting and hosting the 2nd Annual Niemann-Pick Benefit Concert and Silent Auction.
Held in Las Vegas, this event has now raised more than $20,000 for NP-C research. Verna and her committee again rallied the support of individuals, businesses and organizations in the Las Vegas area to provide the community with an evening of entertainment, food and a fabulous auction. The highlight of the evening featured performances given by children, revealing the sweet magic of children helping children.
No sooner had the curtain closed on this year’s event, Verna began work on next year’s event. She hopes it will be even more successful. Our sincere thanks to Verna, her committee and the Las Vegas community for the support you have all given to every family living with NP-C.
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Those Who Care...Karen Sarikas - Volunteer Extraordinaire
One of the first persons Karen Sarikas met when she moved to Tucson in 1982 was Cindy Parseghian. Little did she know this would be the start of a 25-year relationship.
Friendship has always been at the heart of this relationship along with working together as young professionals for four years at a Tucson business, sharing the births of each others’ children, celebrating holidays and special occasions as families, and lending a hand and a lot of heart during the passing of Michael, Marcia and Christa.
As a volunteer, Karen helped launch the Ara Parseghian Medical Research Foundation in 1994 and since its inception has served as our volunteer accountant. In this role Karen oversees the bookkeeping, accounting and banking aspects of the Foundation.
Her upbeat, caring, thoughtful personality is always a beacon of light in the Foundation office. As a CPA, Karen commits hundreds of volunteer hours a year overseeing the fiduciary responsibilities of the APMRF. She is dedicated to ensuring that every dollar given by our generous donors is impeccably handled and utilized in the pursuit of a cure for NP-C disease.
Karen Sarikas is truly a volunteer extraordinaire.
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