More Than $1.8 Million Raised at Seventh Annual Natalie’s Wish Event

June 03, 2008

The seventh annual Natalie’s Wish fundraiser in Newport Beach garnered $1.811 million from well-wishers who were entertained by Ronan Tynan, the acclaimed Irish tenor, and his Olympian story of triumph over personal tragedy.

Since 2003, the annual event for the Cystinosis Research Foundation has raised more than $8.3 million, all of which is being used to fund research to develop better treatments and a cure for the rare, genetic and slowly fatal metabolic disorder that afflicts about 2,000 people, mostly children, worldwide. The May 30 fundraiser at the Balboa Bay Club drew 350 persons.

“This year’s event was extraordinary because of Ronan’s magnificent performance, his inspiring story and the generosity and support of our community and friends. To them, we are exceedingly grateful,” said Nancy Stack, a trustee and co-founder of the CRF. Her daughter Natalie, 17, was diagnosed with cystinosis as an infant.

In patients with cystinosis the amino acid cystine accumulates in the tissue due to the inability of the body to transport cystine out of the cell. Over a period of years, the cystine damages various organs including the kidneys, liver, muscles, white blood cells, eyes and central nervous system. Other complications occur that include muscle wasting and difficulty swallowing. As the cystine accumulates in the cells, the various organs slowly deteriorate. Currently the CRF has 17 ongoing studies with 30 different researchers in five countries – The Netherlands, France, Germany, Italy and the United States.

Amazing Progress In Research Is Reported

Stack also announced at the event that CRF-funded researchers at The Scripps Research Institute in La Jolla, Calif., are reporting encouraging progress with gene therapy and bone-marrow stem-cell therapy studies that someday may lead to a cure. But it will take five more years of laboratory tests before the investigation moves into clinical trials, she said.

“We have talked about a cure, we have dreamed about a cure and now our researchers have an actual plan to test for a cure for cystinosis in the next five years,” she said.

Additionally, trials are scheduled next year to test an improved pharmaceutical treatment developed by CRF-funded researchers at the University of California at San Diego.

This year’s Natalie’s Wish event also was attended by 12 cystinosis sufferers and their families. An estimated 500 persons in North America are afflicted with the disease. Due to the small number affected, little public attention has been given to cystinosis.Cystinosis is among the 6,000 rare or “orphan” diseases in the United States that affect 25 million Americans.

The CRF is the world’s largest provider of cystinosis research. The Irvine-based foundation recently organized the first international research symposium on the disease, which drew 43 researchers and scientists from around the world to the Arnold and Mabel Beckman Center of the National Academies of Science and Engineering, adjacent to the University of California at Irvine.

Vocalist Tynan Overcame His Own Physical Handicap

Tynan, who recently performed at the White House and Carnegie Hall, was born with deformities in his legs, which he lost following a car accident at 20 years old. Despite his handicap, he won 18 gold medals at the Paralympics and earned a medical degree from Trinity University in Dublin. He became a specialist in orthopedic sports medicine.

After becoming a doctor, he began singing at age 33. He won vocal competitions and joined the renowned Irish Tenors. As a soloist he has performed worldwide and made several recordings for Decca Records. Tynan sang at the televised funeral for President Ronald Reagan at National Cathedral in Washington, D.C., a performance heard by 35 million people worldwide. He recently performed at a St. Patrick’s Day reception at the White House and at Carnegie Hall with the New York Pops.

The Irish-born Tynan also is known for his charismatic storytelling and motivational messages. He treats his lifelong physical handicap with lighthearted humor and focuses on the importance of achievement and helping others.

“My father told me, ‘If you make a difference to one person, you’ve done your work on earth,’” he said.

Following his performance, Tynan offered a tribute to cystinosis sufferers:

"One can only applaud and have the greatest admiration for these children and their parents, who have been so challenged by cystinosis. Natalie Stack is one of the finest examples to all young people who live with this burden because she lives her life to the absolute fullest, inspiring younger children to lead a normal life while dealing with the disease and the demands it makes on their lives.

"When you meet Natalie for the first time, you are totally unaware of her struggles. That is something she can feel so very proud of. She is not looking for sympathy or pity. She is eager to enjoy and engage with people. She is a class act, a young lady with great grace and poise.

"All the children with cystinosis, and their parents, should receive a standing ovation for their love, devotion and dedication in helping each other and others less fortunate. Bravo to all. My life has been enriched by meeting you."

Natalie’s Wish: ‘To Have My Disease Go Away Forever’

Natalie’s father, Jeff Stack is a managing director of the SARES●REGIS Group of Irvine. He is prominent in the Southern California real estate industry. He also is chairman of the Urban Land Institute’s Multi Family Silver Residential Council and is past chairman of the National Multi Housing Council.

Jeff and Nancy Stack established the Cystinosis Research Foundation of Irvine in 2003 after learning on Natalie’s 12th birthday that she had a secret wish: “To have my disease go away forever.” Natalie’s plea motivated her parents to launch a campaign to aggressively fund research aimed at treating and curing the disease.

There are three clinical forms of cystinosis. Infantile (nephropathic) cystinosis; late-onset cystinosis; and benign cystinosis. Infantile cystinosis is the most severe and the most common type of cystinosis. Children with nephropathic cystinosis appear normal at birth but by 9 to 10 months of age they develop symptoms that include excessive thirst and urination and failure to thrive. Children often appear pale and thin and have short stature. The abnormally high loss of phosphorous in the urine leads to rickets.

After one year of age, cystine crystals appear in the cornea and causes severe sensitivity to light (photophobia). Children with cystinosis also have an increased level of cystine in their white blood cells. In time, patients can develop problems such as hypothyroidism, severe muscle weakness and central nervous system complications. These children have normal intelligence, but have an unusual defect in short-term visual memory.

Other than $210,000 in auction proceeds, all the money raised came in cash contributions from corporate and individual donors. Additionally, $200,000 was contributed from the Jenna and Patrick Foundation of Hope, a Northern California non profit that was started by Kevin and Teresa Partington, whose 3-year-old twins have cystinosis. The non-profit foundation partners with the CRF.

Major sponsors and contributors to the event included: Michael K. and Lynette Hayde, Merry and John Hagestad, Chris and Bill Thormahlen, David and Suzu Neithercut, Time Warner Cable, R.D. Olson Development, David Pyle, Daryl and Renee Carter, Gerson and Barbara Bakar, the Building Owners and Managers of Orange County, The Dialynas Family, Ralph Eidem, Allan Hunter Family, Peter and Valerie Kompaniez, Joe and Lori McKay, Augie and Lynne Nieto, Rob Wagner and Mark Kroll of Regis Homes of Northern California, Bob and Betty Stine and Mary Anne King and Alex Rose, Kevin and Teresa Partington, Kris and Linda Elftmann, Allen Matkins Leck Gamble Mallory & Natsis, Dianne and Richard Azar, Paul and Sue Ellen O’Connor, Bob and Joni Alter, Automatic Fire Sprinklers Inc., Bank of America, Robert and Susanne Barth, Phil and Shelley Belling, Mike Bissell and Pamela Swartz, Budge Collins, Don and Sonja Grant, Heidi and James Harper, Hoelscher Bell Elliott Foundation, Clyde and Rena Holland, Holland & Hart LLP, Lincoln Property Company, Janet Keller and Bernie Schneider, Karin Krogius and Scott Mason, John and Janice Markley, David and RL Peters, Milan Puskar, RealtyCom Partners LLC, Sandy Sewell, The Summit Fund of Washington and Jill and Larry Tucker, Steve and Pam Quinn, Max and Artyn Gardner, Mark and Denice Flerchinger, Wylie and Bette Aitken.

For more information about Natalie’s Wish and the Cystinosis Research Foundation, call Zoe Solsby at (949) 223-7610 or visit www.natalieswish.org.