Cystinosis Research closes in on a Cure and leads to trials on diseases like Parkinson’s
Waterloo, ON – June 7, 2011 – Scientists are closing in on a gene and stem-cell therapy-based cure for the rare, metabolic and fatal disease, Cystinosis which afflicts about 2,000 persons, mostly children, worldwide, 50-70 children in Canada. Leading the charge, the Cystinosis Research Foundation (CRF) has awarded $11.8 million in research grants in eight countries and has funded 78 multi-year studies worldwide along with 13 research fellows. The Waterloo based Cystinosis Awareness and Research Effort (CARE) has contributed over $230 thousand in just 2 short years thanks to the loving families and friends of children suffering from this disease.
"CRF-funded research at The Scripps Research Institute has resulted in amazing progress moving us closer to a cure. Using cystinosis mouse models, researchers have been able to reverse cystinosis in mice using bone marrow stem-cell transplantation. After mice were transplanted, every single organ in the cystinosis mouse saw a reversal of cystinosis," said Nancy Stack, CRF Chairwoman.
Thanks must be given to the Stack family of Irvine, California who in 2003 began chasing their daughter Natalie’s wish “to have my disease go away forever”.
"The advancements made by the Cystinosis Research Foundation are astonishing," said Stack. "New treatment discoveries made by CRF-funded researchers are the closest thing we have to a cure. These same new treatments are now being tested in trials for Huntington's disease, Parkinson's disease and NASH, a progressive liver disease."
CARE the only organization raising funds for research Canada began in 2009 and has been receiving amazing support from families and communities who have experienced the suffering of cystinosis. After listening to scientists first hand May 18 there is a definite cause for those with other diseases to take note. “These techniques pioneered from research on cystinosis will lead the way in advancing cures for numerous other recessive gene disorders,” says Jody Strauss, mother of Gabbie, who has cystinosis and cofounder of CARE, now a member of the CRF board.
“After seeing the research results to date, I believe whole heartedly a cure is within reach and I want to thank every family who has donated, every friend who has supported and every community which has cared,” says Strauss “while we are closing in on the cure, there is still more research to be done and we need every family affected by cystinosis to rally and raise funds to expedite its arrival”
To learn more visit www.cystinosis.ca and if you are suffering from cystinosis be certain to register at www.cystinosisregistry.org so you are kept up to date on the latest advancements.
Waterloo, ON – June 7, 2011 – Scientists are closing in on a gene and stem-cell therapy-based cure for the rare, metabolic and fatal disease, Cystinosis which afflicts about 2,000 persons, mostly children, worldwide, 50-70 children in Canada. Leading the charge, the Cystinosis Research Foundation (CRF) has awarded $11.8 million in research grants in eight countries and has funded 78 multi-year studies worldwide along with 13 research fellows. The Waterloo based Cystinosis Awareness and Research Effort (CARE) has contributed over $230 thousand in just 2 short years thanks to the loving families and friends of children suffering from this disease.
"CRF-funded research at The Scripps Research Institute has resulted in amazing progress moving us closer to a cure. Using cystinosis mouse models, researchers have been able to reverse cystinosis in mice using bone marrow stem-cell transplantation. After mice were transplanted, every single organ in the cystinosis mouse saw a reversal of cystinosis," said Nancy Stack, CRF Chairwoman.
Thanks must be given to the Stack family of Irvine, California who in 2003 began chasing their daughter Natalie’s wish “to have my disease go away forever”.
"The advancements made by the Cystinosis Research Foundation are astonishing," said Stack. "New treatment discoveries made by CRF-funded researchers are the closest thing we have to a cure. These same new treatments are now being tested in trials for Huntington's disease, Parkinson's disease and NASH, a progressive liver disease."
CARE the only organization raising funds for research Canada began in 2009 and has been receiving amazing support from families and communities who have experienced the suffering of cystinosis. After listening to scientists first hand May 18 there is a definite cause for those with other diseases to take note. “These techniques pioneered from research on cystinosis will lead the way in advancing cures for numerous other recessive gene disorders,” says Jody Strauss, mother of Gabbie, who has cystinosis and cofounder of CARE, now a member of the CRF board.
“After seeing the research results to date, I believe whole heartedly a cure is within reach and I want to thank every family who has donated, every friend who has supported and every community which has cared,” says Strauss “while we are closing in on the cure, there is still more research to be done and we need every family affected by cystinosis to rally and raise funds to expedite its arrival”
To learn more visit www.cystinosis.ca and if you are suffering from cystinosis be certain to register at www.cystinosisregistry.org so you are kept up to date on the latest advancements.
