Research Progress

The Cystinosis Research Foundation’s mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis.  Funding quality research studies remains a priority and is an ongoing process.  The CRF announces global calls for research proposals bi-annually in the spring and fall of each year.  After review and recommendations 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 $39 million to cystinosis research and has awarded 159 research studies and fellowships in 12 countries.  Today, with the support of our partners, 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.

We have reached important CRF milestones in research developments and significant advancements in the treatment of cystinosis.  The CRF research funded at the University of California, San Diego (UCSD) has resulted in the development of a slow-release form of cysteamine.  On April 30, 2013 the FDA approved the new drug Procysbi™.

The Cystinosis Research Foundation’s primary focus is to find a cure for cystinosis.  A group of leading researchers in stem call and gene therapies, molecular biology and clinical pathophysiology have formed the Cystinosis Research Foundation (CRF) Gene Therapy Consortium, with the goal to advance progress on the most promising current findings, including moving novel therapeutic modalities into human patients as quickly as possible. The CRF Gene Therapy Consortium holds the first real promise that a cure for cystinosis is a possibility.

The Fourth CRF International Cystinosis Research Symposium was held March 6 and March 7, 2014 at the prestigious Arnold and Mabel Beckman Center of the National Academies of Engineering and Science on the UCI campus. Hosted by the Cystinosis Research Foundation with sponsorships from Raptor Pharmaceuticals and Sigma-Tau, the symposium brought together more than 60 researchers and scientists from all over the globe – each with a special expertise in some aspect of the care and treatment of cystinosis.

In 2018, the Cystinosis Research Foundation will be hosting the Fifth CRF International Research Symposium at the Arnold and Mabel Beckman Center of the National Academies of Engineering and Science on the UCI Campus.
The symposium, to be held March 1 and March 2, 2018, is by invitation only and attended by CRF-Funded researchers from around the world who are invited to discuss their research progress and to share opportunities for the advancement of cystinosis research.