VAI’s Pathway of Hope targets rare genetic disorder

Getting the right treatment to the right patient is the all-important aim of any research endeavor and has been at the heart of Van Andel Institute’s mission since its founding in 1996. Focusing our efforts on both basic, molecular science and clinical research moves research from discovery to practical use as quickly as possible.

Our scientists are at the forefront of research to repurpose FDA-approved drugs for non-traditional uses. We are finding more and more that existing drugs used singly or in combination with other drugs are having new and unexpected benefits beyond their originally intended use.

Van Andel Institute also has been a leader in the effort to develop targeted therapies tailored to the metabolic and molecular signature of each patient and his or her disease, and we have instituted wide networks of clinical partnerships to impact translational velocity.

A recently launched Van Andel Institute initiative incorporates these types of practices and values through efforts to help patients afflicted by a rare tumor disorder. 

Van Andel Institute’s Pathway of Hope initiative is a multi-faceted project to study tuberous sclerosis complex, a genetic disorder affecting nearly 1 million people worldwide. 

TSC 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 lives. 

Directed by Principal Investigator Jeffrey MacKeigan, Ph.D., Pathway of Hope seeks to develop more effective treatments for patients of TSC by combining basic biology with translational and clinical approaches.

Pathway of Hope employs the mTOR pathway expertise and personalized medicine approaches of Van Andel Institute’s Laboratory of Systems Biology. Research goals are to identify novel drug targets and enroll patients in feasibility trials. Trials will guide treatment plans based on genomic analysis, and determine if work on skin tumors can guide therapies for more invasive tumors.

The study currently involves clinical investigators from leading children’s hospitals and may have important implications for how TSC is treated in the future.

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. 

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 nonprofit also aims to connect the research team to both intellectual and intangible resources.  

Year one project funding has been committed by the Michigan Strategic Fund through a $500,000 grant. 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.

Van Andel Institute’s Pathway of Hope initiative is all about getting the right treatment to the right patient in an effort to fulfill our mission to improve human health and impact individual lives.

David Van Andel is the chairman and CEO of Van Andel Institute.

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