Cystinosis Research Foundation Awards $2 Million in 2011 for Grants to Find a Cure for Deadly, Genetic Disease
(firmenpresse) - IRVINE, CA -- (Marketwire) -- 01/05/12 -- Five grants totaling $776,693 were issued to cystinosis researchers worldwide investigating better treatments and a cure for the disease by the Cystinosis Research Foundation in its second round of proposals in 2011. It brings the CRF's 2011 total to more than $2 million in funded grants. Included is funding for additional research on stem cell and gene therapy, which holds the greatest promise to cure the rare, deadly metabolic and genetic disease. Cystinosis afflicts about 500 children and young adults in the United States and 2,000 worldwide.
"A cure is within sight. These studies will provide important data to realize our goals which will be life changing for the cystinosis community. Our hope is to begin transplanting healthy stem cells into human patients soon. The CRF is funding other important cystinosis research that could offer hope to those diagnosed with Parkinson's and Huntington's disease," said Nancy Stack, CRF Trustee and President.
The CRF's autumn grants were awarded to five research programs at hospitals and universities in the United States, Italy, Belgium, Ireland and Australia. In the last 10 years the CRF has raised more than $17.5 million and has funded 90 studies and fellowships in nine countries. The CRF is the leading funding source for bench and clinical investigations of cystinosis worldwide.
"We are guided by the extraordinary members of the CRF Scientific Review Board who evaluate and analyze every research proposal we receive during our twice-a-year announcement for research and fellowship applications," Stack said. The scientific panel is headed by Dr. Corinne Antignac, a Paris researcher who first identified the CTNS gene in 1998.
Dr. Stephanie Cherqui, assistant professor at Scripps' Department of Molecular and Experimental Medicine, was on Dr. Antignac's team that discovered the cystinosis gene and she chairs the CRF Cystinosis Gene Therapy Consortium. Dr. Cherqui's work, in 2009 reversed the disease and halted tissue damage in mice.
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.
The CRF's mission to find a better treatment for cystinosis has been realized with completion of RP103 Phase 3 clinical trials of delayed-release cysteamine. Raptor Pharmaceuticals, which has licensed the CRF-funded research and is conducting the clinical trials and anticipates FDA approval this year. In addition, the cysteamine and its delayed-release formula is being used in clinical trials for Huntington's disease and NASH, a progressive liver disease.
The CRF also has launched the Cure Cystinosis International Registry (CCIR), whose 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.
Stack and her husband, Geoffrey, a managing director of the SARES-REGIS Group, an Irvine real estate company, have a daughter, Natalie, 20, with cystinosis.
Every dollar raised by the CRF is committed for medical research. Administrative costs are privately underwritten. For more information, go to .
Dept. of Neurosciences, University of California San Diego
"Patient-Reported Outcome and Health-Related Quality of Life in Adults with Cystinosis: A Study Utilizing the NIH 'PROMIS'"
$51,260 - 1 year grant
The Scripps Research Institute, La Jolla, California
"Stem and Gene Therapy for Cystinosis"
$72,503 - 1 year grant
Bambino Gesú Children's Hospital and Research Institute
Telethon Institute of Genetics and Medicine
"Drug Library Screening to Improve the Outcome of Nephropathic Cystinosis"
$323,150 - 2 year grant
Department of Pediatric Nephrology, University Hospitals Leuven
Telethon Institute of Genetics and Medicine
"The Role of Altered Calcium and mTOR Signaling in the Pathogenesis of Cystinosis"
$258,500 - 1 year grant
University College Dublin
Biomedical Sciences, Curtin University
"Role of Nitric Oxide in the Kidney Proximal Tubular Dysfunction Associated with the Fanconi Syndrome in Cystinosis"
$71,280 - 1 year grant
University of California, San Diego
"Tandem Mass Spectrometer Support"
$121,130 - 1 year grant
Gennaro Napolitano, PhD, Research Fellow
The Scripps Research Institute, La Jolla, California
"Small Molecule Regulators of Vesicular Trafficking to Enhance Lysosomal Exocytosis in Cystinosis."
$129,050 - 2 year grant
The Scripps Research Institute, La Jolla, California
"Mechanism of Bone Marrow Stem Cell-Mediated Therapy in the Mouse Model of Cystinosis."
$401,574 - 2 year grant
De Duve Institute, Brussels, Belgium
"Cellular and Tissular Mechanisms for Stem Cell Therapy of Epithelial Cells in a Mouse Model of Cystinosis Coping with Tissue Heterogeneity."
$126,618 - 1 year grant
University of California, San Diego
"Two Year Funding Proposal for Betty Cabrera, BS, MPH."
$194,250 - 2 year grant
University of California, San Diego
"A Study to Evaluate Enteric-Coated Cysteamine Therapy in Patients with Cystinosis"
$52,767 - 2 year grant
Seattle Children's Research Institute, Washington
"Elucidating the Role of Cystinosin-Deficient Macrophages in Nephropathic Cystinosis."
$218,966 - 2 year grant
Zoe Solsby
(949) 223-7610
Art Barrett
(714) 602-6021
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Datum: 05.01.2012 - 21:12 Uhr
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