SOURCE: Cystinosis Research Foundation

Cystinosis Research Foundation

January 24, 2011 20:02 ET

Cure Cystinosis International Registry Is Translated Into Spanish, Boosting the CCIR's Accessibility

IRVINE, CA--(Marketwire - January 24, 2011) - The recently launched Cure Cystinosis International Registry (www.cystinosisregistry.org) has been translated into Spanish, dramatically increasing the accessibility of the CCIR, which is the first global effort to link cystinosis patients with scientists and researchers who are developing new treatments. The rare metabolic and fatal disorder afflicts about 2,000 persons, mostly children, worldwide.

"We're working diligently to reach cystinosis patients worldwide so they can take advantage of the benefits of the CCIR and help advance efforts to develop a cure," said Nancy Stack, President and co-founder of the Cystinosis Research Foundation, one of the registry's key partners.

Plans are to translate the registry into French within the next two months. Cystinosis patients from 25 countries have enrolled in the CCIR, Stack said.

"Patients and families on six continents have registered in the four months since the CCIR was activated. We're very encouraged with the response. Top cystinosis researchers are encouraging cystinosis patients to register with CCIR which is the only international patient registry in the world dedicated to the cystinosis community," Stack said.

"Our goal is to have every cystinosis patient worldwide register with the CCIR. The benefits of CCIR enrollment will be lifesaving and life changing," Stack said.

The purpose of the CCIR is to identify people with cystinosis globally and collect their medical histories and information to share (anonymously) with the cystinosis research community. This information will allow clinicians, researchers and pharmaceutical companies to accelerate novel treatments and a cure for cystinosis.

Researchers Worldwide Enrolling With CCIR

Many researchers among the 48 teams of scientists currently funded by the Cystinosis Research Foundation have registered with the CCIR. Stack said this clearly demonstrates the importance of the patients' information, particularly to researchers doing advanced bench and clinical studies.

The CCIR is a collaboration of 12 cystinosis advocate foundations worldwide. The countries in which patients are enrolled, thus far, are: the United States, Canada, Belgium, England, Scotland, France, Italy, Portugal, Ireland, Germany, Norway, Sweden, Switzerland The Netherlands, Australia, Russia, Iran, South Africa, Argentina, Chile, Bolivia, Brazil, Costa Rica, St. Barthelemy and The Bahamas.

Patient Information Is Private, Protected

The CCIR is the only registry created specifically for individuals with cystinosis and will contain current information regarding cystinosis clinical trials and studies. All patient information is de-identified (anonymous) and held in a secure data base accessible only by the CCIR curator. Information that could identify participants and their family members will not be shared without their expressed written approval. Participants will also be able to view aggregate data allowing them to view how they fit within the larger cystinosis community.

CCIR officials say recruitment for clinical trials can be a lengthy process, especially for a rare disease like cystinosis. Participation in this registry will help speed up the recruitment process and facilitate and expedite clinical trials, officials say.

In patients with cystinosis, the amino acid cystine accumulates in the tissue due to the inability of the body to transport cystine out of one of the compartments of the cell. 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.

For more information about the Cure Cystinosis International Registry, contact Betty Cabrera at curator@cystinosisregistry.org.

Contact Information

  • Contact:

    Art Barrett
    714-602-6021

    Zoe Solsby
    949-809-2400