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Van Andel Institute begins three-year study to treat rare tumor disorder
Researchers at Van Andel Institute in Grand Rapids have begun a major multi-million dollar study of the tuberous sclerosis complex — a rare genetic disorder most often diagnosed during infancy or childhood.
The genetic disorder causes tumors in vital organs throughout the body, affecting the brain, eyes, skin, heart, kidney and lungs.
While diagnosed primarily in childhood, TSC is not solely a pediatric disorder, and can result in health complications throughout adolescence and adulthood.
Tumors resulting from TSC have been found to cause epilepsy, autism and developmental disabilities in some patients. Recent studies indicate that 80 percent of individuals diagnosed with TSC will have epilepsy at some point in their life.
The research initiative, called Pathway of Hope, “will employ our tumor biology expertise and personalized medicine, in concert with our collaborators and the Tuberous Sclerosis Alliance, in hopes of improving the lives of children and adults living with this incurable disorder,” said Jeffrey P. MacKeigan, principal investigator directing the research.
“Pathway of Hope is a true collaboration between Van Andel Institute, our research partners and organizations like the TS Alliance," said David Van Andel, the institute’s president and CEO. "This initiative gives people hope, and lets people living with TSC know that research experts are taking a serious look at better ways to treat this debilitating disorder."
The multi-million dollar project spans three years and involves TSC experts and investigators from across the United States working to identify potential drug therapies.
The research team will enroll patients in a feasibility trial in 2013, to determine how to best develop treatment plans based on genomic analysis, and if characterization of skin tumors can guide therapies for other more invasive tumors.
Participants for the feasibility trial will be enrolled in West Michigan at Helen DeVos Children’s Hospital.
“Since TSC involves tumors that grow slowly, we have an excellent opportunity to fully analyze genetic data to inform personalized treatment strategies,” said Dr. Giselle Sholler, co-investigator and Pediatric Oncology Research Program lead for VAI. Sholler is also a physician with Spectrum Health Medical Group.
First year funding for the project has been committed by the state's Michigan Strategic Fund through a $500,000 signed grant agreement. Private sources, including Blue Cross Blue Shield of Michigan Foundation and Rockford Construction, also have contributed for the first phase of the study.
Dr. Steven Roberds, chief scientific officer for TS Alliance, said the research “addresses significant gaps in tuberous sclerosis complex knowledge that are important for improving and creating therapeutic strategies for TSC.”