Research Progress

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 CRF announces two global calls for research proposals in the spring and fall of each year.  After evaluation by our 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. 

To date, the CRF has funded and committed more than $10.8 million to cystinosis research and has funded 51 research studies and fellowships.  In 2009, the CRF funded $3.3 million in new grants. Today, with the support of our friends and community we have significantly changed the course of cystinosis research. Our 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 when Phase 3 trials are completed.  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, we met with a team of CRF-funded scientists from Scripps Institute in La Jolla, who presented us with 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.  Their plan holds the first real promise that a cure for cystinosis is a possibility.

Our first CRF International Cystinosis Research Symposium was held in April 2008 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 and April 9, 2010.

» Click Here to view graphs of our progress