IRVINE, Calif.--(BUSINESS WIRE)--The Cystinosis Research Foundation has announced the first round of 2015 cystinosis grants. The studies were awarded to research institutions to enable scientists to investigate improved treatments and a cure for cystinosis, a rare metabolic and genetic disease that afflicts about 500 children and young adults in the United States and 2,000 worldwide. The five research grants total $1,022,879.
Click to Tweet: CRF Awards $1 Million In First Round Of 2015 Cystinosis Research Grants For Improved Treatments And The Cure
Several proposals that received a CRF semi-annual grant were for new areas of research. Ghanashyam Acharya, Ph.D., at Baylor College of Medicine received a grant for the development of a potential novel treatment for cystinosis; a cysteamine-infused transdermal patch. Sihoun Hahn, Ph.D., at Seattle Children’s Hospital received a grant for newborn screening for cystinosis.
“The more we learn about cystinosis the faster we advance research aimed at better treatments and a cure. CRF funds the best ideas in cystinosis research with the goal of reaching clinical trials. We place strong emphasis on translational and clinical research to ensure the development of new therapies for those with cystinosis. Dr. Acharya’s cysteamine patch study could lead to a superior delivery system for the life-saving medication our children depend on,” said Nancy Stack, CRF Trustee and President.
“We are ecstatic to fund the first newborn screening study for cystinosis which we know will result in saving lives. Early detection is crucial for children because once diagnosed, treatment can begin,” said Stack.
The CRF’s 2015 Spring Research Grants include continued support for Stéphanie Cherqui, Ph.D., and her cutting edge bone marrow stem cell-mediated therapy at the University of California, San Diego. Her extraordinary work holds great promise for a cure. Also funded was a research fellowship grant to Liang Feng, PhD and Xue Guo, PhD at Stanford University, Palo Alto, California, that will aim to understand the molecular mechanism of cystinosis in an effort to understand the cause and cure for cystinosis. Internationally, Anna Taranta, PhD at Bambino Gesù Children’s Hospital in Rome was awarded a grant to study the factors that contribute to bone remodeling defects in cystinosis.
“This is an exciting time for the cystinosis community. We are on the brink of new treatments. Since 2003, the CRF has raised more than $29 million, all of which is committed to research. The CRF has funded 130 studies and fellowships in 12 countries. We are proud to say that our global research strategy ensures that every minute of the day, a CRF researcher is working to find better treatments and a cure,” Stack said.
Cystinosis slowly destroys every organ in the body, including the liver, kidneys, eyes, muscles, thyroid and brain. There is a medicine, cysteamine, that prolongs the patients’ lives, but there is no cure.
The CRF is guided by a Scientific Review Board comprised of renowned cystinosis researchers and scientists who evaluate the research applications. Their expertise and leadership guarantees that CRF funds only the most promising research studies aimed at solving the mysteries of the disease. The scientific panel is headed by Dr. Corinne Antignac, the Paris researcher who first identified the CTNS gene in 1998.
One of CRF’s missions, to find a better treatment for cystinosis, was realized in 2013 with FDA approval of a delayed-release form of cysteamine developed by CRF-funded researchers at University of California at San Diego. In addition, a delayed-release cysteamine formula is being used in clinical trials for Huntington’s disease and NASH, a progressive liver disease.
Stack and her husband, Geoffrey, a managing director of the SARES•REGIS Group, an Irvine-based real estate company, have a daughter, Natalie, 24, with cystinosis. Every dollar raised by the CRF is committed for medical research. Administrative costs are privately underwritten. For more information, go to www.cystinosisresearch.org.
2015 SPRING CRF RESEARCH GRANTS FUNDED TOTAL: $1,022,879
Ghanashyam Acharya, PhD, Principal Investigator
Baylor
College of Medicine, Houston, Texas
“Development of Transdermal
Cysteamine Delivery System”
Award - $112,500
Stéphanie
Cherqui, PhD, Principal Investigator
University of California,
San Diego
“Mechanism of Bone Marrow Stem Cell-Mediated Therapy in
the Mouse Model of Cystinosis”
Award - $364,800
Liang Feng,
PhD, Mentor
Xue Guo, PhD, Fellow
Stanford
University, Palo Alto, California
“Molecular Mechanism of
Cystinosis”
Award - $225,000
Sihoun Hahn, MD, PhD,
Principal Investigator
Seattle Children’s Hospital, Seattle,
Washington
“Newborn Screening for Cystinosis”
Award - $153,929
Anna
Taranta, PhD, Principal Investigator
Andrea Del Fattore,
PhD, Co-Principal Investigator
Bambino Gesù Children’s
Hospital, Rome, Italy
“A Study on Bone Remodeling Defects in
Nephropathic Cystinosis”
Award - $166,650
