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 $54 million to cystinosis research and has awarded 190 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 dedicated to finding better treatments for corneal cystinosis. Corneal cystinosis is the build-up of cystine crystals in the eyes that causes photophobia (extreme sensitivity to light) severe eye pain, and sometimes, blindness. There is an existing treatment but it is rigorous and requires hourly dosing of medicated eye drops.