Open menu

Nanotechnology General News

The latest news from academia, regulators
research labs and other things of interest

Posted: May 06, 2014

Fundraiser generates $2.3m for nanotechnology research to improve cystinosis treatments

(Nanowerk News) The Cystinosis Research Foundation raised a record $2.3 million at its annual fundraiser for new research to improve cystinosis treatments and continue progress to cure the rare metabolic disease. Cystinosis destroys the body’s organs and afflicts about 2,000 people, mostly children, worldwide.
“We are on the brink of new possibilities and treatments,” said Nancy Stack, CRF Trustee and President. She spoke of CRF-funded stem cell and gene therapy that has reversed the disease in mice and how first-ever trials using nanotechnology as a treatment delivery system to relieve the symptoms of corneal cystinosis are planned within a year.
About 450 people at the CRF’s Natalie’s Wish event April 5 at the Balboa Bay Club in Newport Beach, Calif., were treated to a sumptuous dinner and world-class entertainment that included The Tenors, a quartet of Canadians who performed songs from their latest album, “Lead With Your Heart.” An auction that included several lavish vacations, concert tickets and dinners in addition to hand-picked cases of fine wines raised a record $245,000.
Among the guests were cystinosis patients and their families from throughout United States and Canada, whose lives have been improved by the achievements funded by the CRF.
Cystinosis is the abnormal accumulation of the amino acid cystine in cells. The disease gradually destroys the major organs, including kidneys, liver, eyes, muscles, bone marrow, thyroid and brain. Other complications include muscle wasting and difficulty swallowing. Currently, there is no cure.
CRF-funded doctors discovered a delayed release form of the life-saving medication. Approved by the FDA last year, the medication is the first advancement in decades. The CRF is the largest fund provider of cystinosis research, having raised $23 million and funded 114 studies and fellowships since 2003. Eleven grants totaling $2.1 million were awarded in 2013.

Twelve cystinosis family foundations contributed $631,300 from their community fundraising efforts. Cash contributions and money raised at auction totaled $479,603. Contributions received before and after the event totaled $1,189,600. All contributions go entirely to research. The CRF’s costs are privately underwritten.

The event is named for Natalie Stack, the 23-year-old daughter of Nancy and Geoffrey Stack. Natalie, who recently graduated from college, was diagnosed as an infant and on her 11th birthday wished for her “disease to go away forever.” Her mother says Natalie takes 36 pills a day, which totals 13,140 pills per year, plus she takes seven to eight eye drops per day to avoid going blind. She said on average that cystinosis patients take between eight to 12 different medications daily. The medications which cause painful side effects must be taken “every day just to stay alive.”

With Natalie home from college, Stack said, she again is a constant witness to her daughter’s life of daily discomfort, pain and forbearance. Stack described the “extreme fatigue” in cystinosis patients along with painful eye irritation caused by an accumulation of cystine crystals.

“On the outside our children look healthy but on the inside, cystinosis is destroying their bodies,” she said.
“Given how she feels physically, I cannot believe she functions as well as she does. She rarely complains. Although cystinosis is a relentless disease, it doesn’t mean a life of misery and hopelessness,” Stack said. Like all others who bravely struggle with cystinosis, she said, “Natalie lives with courage, grace and determination.”
In addition to the newly approved treatment – a delayed-release form of cysteamine, which cuts dosing frequency in half and with reduced side effects – CRF is making progress on other promising fronts.
Dr. Stephanie Cherqui at the University of California at San Diego is working with the federal Food and Drug Administration in anticipation of a clinical trial for an autologous stem cell and gene therapy treatment. “The treatment, if it works, will potentially cure cystinosis,” Stack said.
Additionally, Stack said, “astonishing” research is being done to treat corneal cystinosis, the painful eye condition that causes severe photophobia and often blindness. Dr. Jennifer Simpson, an ophthalmologist at the Gavin Herbert Eye Institute at UC Irvine, and Dr. Ghanshyam Acharya at the Baylor School of Medicine in Texas are developing a novel treatment for corneal cystinosis. The potential treatment involves nanotechnology. If the new treatment works, it will be revolutionary and it will eliminate the need for hourly drops, currently the only treatment for corneal cystinosis. “We are hopeful that a clinical trial will begin within one year, she said.
Source: Cystinosis Research Foundation
If you liked this article, please give it a quick review on reddit or StumbleUpon. Thanks!
Check out these other trending stories on Nanowerk:

Subscribe to a free copy of one of our daily
Nanowerk Newsletter Email Digests
with a compilation of all of the day's news.