Grand Rapids, Mich. (February 18, 2013) – Researchers at Van Andel Institute (VAI) have embarked on a multi-faceted initiative to study tuberous sclerosis complex (TSC), 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% of individuals diagnosed with TSC will have epilepsy at some point in their life span.
The research initiative, Pathway of Hope, involves a combination of basic biology, as well as translational and clinical approaches, with the goal of developing new and effective treatments for individuals with TSC.
“We will employ our tumor biology expertise and personalized medicine, in concert with our collaborators and the Tuberous Sclerosis Alliance (TS Alliance), in hopes of improving the lives of children and adults living with this incurable disorder,” said Jeffrey P. MacKeigan, PhD, Principal Investigator directing the initiative.
The collaborative research initiative positions Van Andel Institute as a leading center for rare-tumor research and clinical trials.
“Pathway of Hope is a true collaboration between Van Andel Institute, our research partners and organizations like the TS Alliance. 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,” said David Van Andel, President and CEO, Van Andel Institute.
The multi-million dollar project spans three years and involves TSC experts and investigators from across the United States. The research team aims to identify novel drug targets and enroll patients in a feasibility trial in 2013. The trial will 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.
“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, Van Andel Institute; and Spectrum Health Medical Group physician.
Pathway of Hope has implications for the way TSC is treated in the future, and Dr. Steven DeRoos, co-investigator and Division Chief of Pediatric Neurology at Spectrum Health Medical Group and Helen DeVos Children’s Hospital physician, states, “This unique initiative between researchers and physicians will look to improve outcomes among TSC patients where surgical resections or current therapies do not provide long term efficacy.” Participants for the feasibility study will be enrolled in West Michigan at Helen DeVos Children’s Hospital.
Year one project funding has been committed by the Michigan Strategic Fund through a $500,000 signed grant agreement. Private sources, including Blue Cross Blue Shield of Michigan Foundation and Rockford Construction, have also contributed funding for the first phase of the study. Generous financial support from these public and private entities gives Pathway of Hope investigators the opportunity to achieve their research aims, and will ultimately play an important role in positively impacting treatments for TSC patients.
The TS Alliance, a national voluntary organization dedicated to finding a cure for TSC, is partnering with Van Andel Institute on the initiative, and is instrumental in raising awareness about this high-impact project within the national and international TSC community. The Maryland based non-profit also aims to connect the research team to both intellectual and intangible resources.
Dr. Steven Roberds, Chief Scientific Officer of the TS Alliance, states that the research “addresses significant gaps in tuberous sclerosis complex knowledge that are important for improving and creating therapeutic strategies for TSC.”
About Van Andel Institute – Established by Jay and Betty Van Andel in 1996, Van Andel Institute (VAI) is an independent research and educational organization based in Grand Rapids, Mich., dedicated to preserving, enhancing and expanding the frontiers of medical science, and to achieving excellence in education by probing fundamental issues of education and the learning process. Van Andel Research Institute (VARI), VAI’s research arm, is dedicated to studying the genetic, cellular and molecular origins of cancer, Parkinson’s and other diseases and working to translate those findings into effective therapies. This is accomplished through the work of more than 200 researchers in on-site laboratories and in collaborative partnerships that span the globe. Find out more about Van Andel Institute or donate by visiting www.vai.org. 100% to Research, Discovery & Hope℠.