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 cystinosis 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 Cystinosis Research Foundation is excited and pleased to announce that we are one step closer to the cure. Dr. Stéphanie Cherqui, who has been funded by CRF since 2007, recently formed the Cystinosis Stem Cell and Gene Therapy Consortium. The consortium includes experts in the fields of nephrology, neurology, endocrinology gastroenterology, ophthalmology, bone marrow transplantation and gene therapy, and members of the cystinosis community. Collectively, the panel will contribute to the design and methodology of a clinical trial in cystinotic patients that tests the safety of autologous transplantation with hematopoietic stem cells that have been gene modified ex-vivo with a lentiviral vector to express a functional CTNS gene.
CRF is also funding the work of Dr. Ghanashyam Acharya at the Baylor College of Medicine and Dr. Jennifer Simpson at Universtiy of California, Irvine, for the Nanowafer Project for Corneal Cystinosis. CRF is working closely with them on developing a clinical trial to test a novel treatment for corneal cystinosis. We have begun working with the FDA to comply with their requirements and move towards a clinical trial in early 2018.
The CRF research funded at the University of California, San Diego (UCSD) has resulted in the development of a slow-release form of cysteamine, and on April 30, 2013 the FDA approved the new drug Procysbi™.
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. The next cystinosis research symposium will be held in 2016.