Second Annual Fore A Cure Golf Tournament Announced

September 03, 2008

The Cystinosis Research Foundation is holding its second annual Fore a Cure, Natalie’s Wish golf tournament Monday, Nov. 10, at the Coto de Caza Golf & Racquet Club. All event proceeds will fund research to find better treatments and a cure for cystinosis, a rare metabolic disorder that afflicts 500 children and young adults in the United States.

The event is open to 144 golfers. The entry fee is $2,500 per foursome. Sponsorship opportunities and benefits still are available. Last year’s event by the Irvine-based foundation raised $240,000.

Sponsors to date include: Time Warner Cable, SARES●REGIS Group, JM Realty, Bristol Group, Bank of the West, Manly & Stewart, Eagle Construction, Gramercy Gifts, First Regional Bank, Quiksilver, Noelle Marketing Group, Dzida, Carey & Steinman, Regency Group and Traditional Jewelers.

“Cystinosis is a metabolic disease that eventually and slowly destroys every organ in the body, including the liver, kidneys, eyes, muscles, thyroid and brain. There is a medicine that prolongs the children’s lives, but there is no cure. Almost all sufferers succumb before 40 years old,” said event Chairman Vince Ciavarella.

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. This causes development of crystals, resulting in early cell death. Cystinosis is a rare, incurable metabolic disease that afflicts 500 children and young adults in the United States and approximately 2000 worldwide. There are more than 6,000 rare or “orphan” diseases in the United States. An orphan disease as defined by the Orphan Disease Act affects less than 200,000 people. Collectively however, these diseases affect more than 25 million Americans. While there are only a small number of patients who suffer from any given orphan disease, knowledge discovered by studying one disease may lead to advancements in other rare diseases and more prevalent and well-known disorders. There is no cure for cystinosis, but there is hope.

About The Foundation

The CRF was formed in 2003 and has raised $8.3 million, all of which is committed for medical research. Currently, the CRF has 26 ongoing studies with researchers in the United States and throughout Europe.

The CRF recently announced it has funded or made commitments of $1,316,361 for nine new scientific research studies in the United States and Europe aimed at finding better treatments and a cure for cystinosis. A second global call for research proposals and fellowship grants will go out this fall.

Geoffrey Stack, an owner of the diversified Irvine-based real estate development, construction and management company SARES●REGIS Group, and his wife, Nancy, formed the Cystinosis Research Foundation in 2003. Their daughter Natalie, 17, suffers from cystinosis. The non-profit foundation has raised more than $8.3 million, all of which has been spent or is earmarked to fund cystinosis scientific studies in the United States and Europe. The annual Natalie’s Wish banquet on May 31 in Newport Beach raised $1.8 million and featured a performance and keynote address by acclaimed Irish tenor Ronan Tynan.

The Cystinosis Research Foundation’s mission is two-fold and focused: to find better treatments and a cure for cystinosis.Funding quality research studies remains a priority and is an ongoing process.The CRFannounces two globalcalls for research proposals in the spring and fall of each year. After evaluation by the foundation’s Scientific Review Board, research grants are issued.In 2006, the CRF established the first Cystinosis Research Fellowship Program designed to support scientists and new researchers who have an interest in cystinosis research.

Changing The Course Of Research

To date, the CRF has funded and committed more than $6.5 million to cystinosis research and has funded more than 49research studies andfellowships.Today, with the support of CRF’s friends and community, the foundation has significantly changed the course of cystinosis research. CRF’s funding efforts have allowed talented doctors and researchers in the area of cystinosis to initiate novel research studies and to advance their research efforts.

This past year has been a year of new research developments and significant advancements in the treatment of cystinosis. The research funded at the University of California, San Diego (UCSD) has resulted in the development of a slow-release form of cysteamine. Late last year, Raptor Pharmaceuticals Corp.’s subsidiary, Bennu Pharmaceuticals, Inc., acquired the worldwide license for the slow-release form of cysteamine from UCSD. They are committed to improving and advancing the slow-release drug and applying to the FDA for approval of the new drug in 2009. The slow-release medication will be a reality for all of our children in the near future.

The Cystinosis Research Foundation’s primary focus is to find a cure for cystinosis. In April, CRF-funded scientists from Scripps Institute in La Jolla, who presented a plan that will lead to clinical trials in children with cystinosis. The clinical trials will test new therapies that they believe and hope will lead to a cure for cystinosis. Although the possible clinical trials to test for a cure in patients with cystinosis are at least five years away, their plan holds the first real promise that a cure for cystinosis is a possibility.

The CRF’s first International Cystinosis Research Symposium was recently held at the prestigious Arnold and Mabel Beckman Center of the National Academies of Engineering and Science on the UCI campus.The event was attended by more than 60 researchers from the United States and Europe who have been working to achieve better treatments and a cure for cystinosis.Twenty-four researchers funded by CRF presented their current bench and clinical studies on cystinosis.

“The symposium was very important for cystinosis because many of the participants began sharing information on their research efforts – information that generally isn’t shared until it’s published, which often takes months or years. This can only help accelerate new medical and scientific advancements we’ve been working to achieve for so many years,” said Dr. Jerry Schneider, co-chairman of the symposium.The Second CRF International Cystinosis Research symposium is scheduled for April 8-9, 2010.

For more information about sponsorships, the tournament and cystinosis research, contact Zoe Solsby at 949-223-7610 or go to www.natalieswish.org.