Using Stem Cells From Children With Rare Diseases To Find New Treatments

Using Stem Cells From Children With Rare Diseases To Find New Treatments



By [http://EzineArticles.com/expert/Richa_Verma/2314151]Richa Verma

Chris Waters is the founder of Rare Science; an organization that has taken up the mission of accelerating cures for kids with rare diseases. She defines her motivation best by saying, "The biggest gap in drug development is that we are not addressing the specific needs of children, especially those with rare diseases. We need to focus on kids. They are our future. If it takes 14 years and US$2 billion to get FDA approval for a new drug, how is that going to help the 35% of the 200 million children across the world that are dying before 5 years of age because they have a rare disease? That's why we created Rare Science. How do we help kids right now, how do we help the families? How do we make change?"

According to Chris, kids with rare diseases can go through a prolonged struggle before being accurately diagnosed. Post diagnosis, most of the time, treatment is not available for the same. "Even though we know that the affected individuals have the gene mutation, we have no idea how the gene causes the observable traits that are widely variable across the individuals we know. We need research tools to help us understand the biology of ADCY5 and other rare disease - it is not enough to just know the gene mutation. We always wanted to do a stem cell line that would help us get at these biological questions,"says Chris.

With limited resources in hand along with a number of intimidating roadblocks, Chris Waters initiated this mission. With every tissue donation, she required consent forms from the children and their parents in question so that the donors were aware of what was involved in giving the samples and how they were going to be used during research. These collected samples - some of them healthy, some suffering from diseases such as autism, Alzheimer's, heart, lung and liver disease and blindness, will be available to researchers globally.

The samples will be turned into pluripotent stem cells that have the ability to be turned into any other type of cell in the body - enabling researchers to investigate and carry our trials on future therapeutic usages through disease progression. With this program, Chris hopes to establish a service model for other rare diseases by creating stem cell lines from them to reduce the gap between discovery, research and clinical impact.

To know more about stem cell banking visit   [https://www.cordlifeindia.com/]https://www.cordlifeindia.com/.

Article Source: [http://EzineArticles.com/?Using-Stem-Cells-From-Children-With-Rare-Diseases-To-Find-New-Treatments&id=9559808] Using Stem Cells From Children With Rare Diseases To Find New Treatments

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