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news.The Cystinosis Research Foundation (CRF) raised $2.3m at its annual fundraiser for new research to improve cystinosis treatments and continue progress to cure the rare metabolic disease.
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CRF trustee and president Nancy Stack noted that the foundation is on the brink of new possibilities and treatments.
Stack spoke of CRF-funded stem cell and gene therapy that has reversed the disease in mice and how first-ever trials using nanotechnology as a treatment delivery system to relieve the symptoms of corneal cystinosis are planned within a year.
About 450 people at the CRF’s Natalie’s Wish event 5 April 2014 at the Balboa Bay Club in Newport Beach, California, were treated to a sumptuous dinner and world-class entertainment that included The Tenors, a quartet of Canadians who performed songs from their latest album, "Lead With Your Heart."
An auction that included several lavish vacations, concert tickets and dinners in addition to hand-picked cases of fine wines raised a record $245,000.
Among the guests were cystinosis patients and their families from throughout US and Canada, whose lives have been improved by the achievements funded by the CRF.
Cystinosis is the abnormal accumulation of the amino acid cystine in cells. The disease gradually destroys the major organs, including kidneys, liver, eyes, muscles, bone marrow, thyroid and brain. Other complications include muscle wasting and difficulty swallowing. Currently, there is no cure.
CRF-funded doctors discovered a delayed release form of the life-saving medication. Approved by the FDA last year, the medication is the first advancement in decades. The CRF is the largest fund provider of cystinosis research, having raised $23m and funded 114 studies and fellowships since 2003. Eleven grants totaling $2.1m were awarded in 2013.
A total of 12 cystinosis family foundations contributed $631,300 from their community fundraising efforts. Cash contributions and money raised at auction totaled $479,603. Contributions received before and after the event totaled $1,189,600. All contributions go entirely to research. The CRF’s costs are privately underwritten.
The event is named for Natalie Stack, the 23-year-old daughter of Nancy and Geoffrey Stack. Natalie was diagnosed as an infant and on her 11th birthday wished for her disease to go away forever.
n addition to the newly approved treatment – a delayed-release form of cysteamine, which cuts dosing frequency in half and with reduced side effects – CRF is making progress on other promising fronts.
It was the 12th annual Natalie’s Wish celebration. Live auction donors were Michael Browne of Kosta Browne Winery, Richard Eddy of Pines Loge Development, Johnna and Don Grant, Alan Greeley of The Golden Truffle Catering/Restaurant, Inspirato Luxury Destination Club, Paula and Doug Neff, Kokkari Restaurant, Pacific Hospitality Group – Meritage Collection, Anna Simons, Nancy and Jeff Stack and Traditional Jewelers.