SOURCE: Cystinosis Research Foundation

Cystinosis Research Foundation

September 14, 2010 09:00 ET

Cystinosis Research Foundation Issues Global Call for Proposals to Treat and Cure Fatal, Genetic Disease

IRVINE, CA--(Marketwire - September 14, 2010) -  The Cystinosis Research Foundation -- the largest privately supported source of research funds for the study of cystinosis, a fatal genetic disease -- today issued its Autumn 2010 Global Call for Research Proposals and Fellowships. The organization has allocated $750,000 for research in this round of funding.

"Funding by the CRF has opened up exciting new fields of study, leading to promising new treatments and a cure for cystinosis. We expect a strong response from scientists, many of whom are in our worldwide network of cystinosis researchers. Since 2003, the CRF has funded $11.8 million in research," said Nancy Stack, President of the CRF.

Grant and fellowship proposal guidelines are available on\research. The proposal deadline is Oct. 20, 2010. Awards will be announced at the end of the year.

Cystinosis is a metabolic disease that 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. Most cystinosis sufferers succumb to the disease or its complications by age 40. Cystinosis afflicts about 2,000 children and young adults worldwide.

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.

"Getting funds directly into the labs of the world's leading cystinosis researchers and scientists has changed the course of cystinosis research. CRF funding in the past has led to the final Phase 3 clinical trial of delayed-release cysteamine which is under way at Emory University, Atlanta, Ga.; Stanford University, Palo Alto, Calif.; and Children's Memorial Hospital, Chicago, Ill.; and will soon be at several other sites in the U.S. and Europe," Stack said.

Additionally, a group of CRF-funded scientists this year formed the CRF Cystinosis Gene Therapy Consortium whose goal is to advance progress on the most promising current findings, including moving novel therapeutic modalities into human patients as quickly as possible. Work is now underway at The Scripps Research Institute in La Jolla, Calif., to develop the necessary preclinical animal model data and translate these results into an FDA-approved clinical trial.

"We hope, if all goes well, there will be a clinical trial for a cure within the next two to three years," Stack said.

The CRF also has launched the Cure Cystinosis International Registry (CCIR), the first cystinosis registry. Its purpose is to consolidate information about cystinosis patients into a single data repository which will help advance research and clinical trials leading to future treatments and cures.

Nancy Stack and her husband, Geoffrey, a managing director of the SARES•REGIS Group, an Irvine real estate company, have a daughter, Natalie, 19, with cystinosis.

Every dollar raised by the CRF is committed for medical research. Administrative costs are privately underwritten. All grants are awarded based on evaluations by the CRF's Scientific Review Board, which is comprised of leading doctors and research scientists worldwide.

Last spring, the CRF awarded $988,759 in grants to seven specialized research studies in the United States, France, Belgium and Ireland. For more information, visit or call (949) 223-7610.

Contact Information

    Cystinosis Research Foundation
    Zoe Solsby
    (949) 223-7610
    Art Barrett
    (714) 602-6021