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Van Andel Gets Funding for Rare Disease Project

NEW YORK (GenomeWeb News) – The Van Andel Institute has received a $500,000 award to launch an effort that will use genomic data to develop new personalized treatment options for a rare genetic disorder.

The Pathway of Hope project will focus on tuberous sclerosis complex (TSC), which causes tumors in vital organs, including the brain, eyes, skin, heart, kidneys, and lungs, and has been found to cause epilepsy, autism, and developmental disabilities in some patients. Around 80 percent of individuals diagnosed with this disorder will have epilepsy at some point in their lives, Van Andel said in a statement yesterday.

Van Andel said that the initial funding, which came through a grant from the Michigan Strategic Fund, has been augmented by additional investments from Blue Cross Blue Shield of Michigan and Rockford Construction.

The three-year project will incorporate basic biology, translational research, and clinical approaches to develop personalized treatments for TSC and will involve a partnership with the Tuberous Sclerosis Alliance. It will include researchers from across the US, Van Andel said, and aim to identify drug targets and enroll patients in a feasibility trial in 2013. That trial will determine how to best develop treatment plans based on genomic analysis and also determine if characterization of skin tumors can guide therapies for other more invasive tumors.

"Since TSC involves tumors that grow slowly, we have an excellent opportunity to fully analyze genetic data to inform personalized treatment strategies," Giselle Sholler, co-investigator and pediatric oncology research program lead for Van Andel, said in a statement.