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2019 Highlights of Hope: Fall/Winter

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RESEARCH HOW VAN ANDEL

RESEARCH HOW VAN ANDEL INSTITUTE SUPPORTS CLINICAL TRIALS THROUGH COLLABORATION AND CONNECTION INSTITUTE SERVES AS A CONNECTOR THAT FOSTERS DISCOVERY OF POTENTIAL NEW TREATMENTS Clinical trials can be a bastion of hope for people living with terminal disease. For a person with cancer who may be at the end of what standard care offers, clinical trials investigating potential new treatments can quite literally be a lifeline. At the same time, clinical trials for diseases like Parkinson’s, where breakthroughs have historically been few and far between, give participants hope that they could be on the cutting edge of potential new therapies. Although Van Andel Institute does not treat patients or conduct clinical trials onsite, it is positioned to support the global efforts to find new and innovative approaches to treating — and perhaps curing — these diseases. VAI scientists lend their expertise and laboratories to the hunt for new therapies, and the Institute brings together the many groups — scientists, physicians, philanthropists and companies — required to carry out trials. Currently, VAI supports nearly two dozen clinical trials geared toward finding new, life-changing treatments for Parkinson’s and cancer through a pair of innovative collaborations: the Linked Clinical Trials initiative, a partnership with The Cure Parkinson’s Trust in the United Kingdom, and the Van Andel Research Institute–Stand Up To Cancer Epigenetics Dream Team, a coalition of leading scientists, physicians, philanthropies and industry partners with a shared goal of getting promising new cancer therapies to patients faster. “Diseases like cancer and Parkinson’s are too big of a problem for one person or group to take on alone — they require a concerted, collaborative effort.” — DR. PETER A. JONES “Diseases like cancer and Parkinson’s are too big of a problem for one person or group to take on alone — they require a concerted, collaborative effort,” said Dr. Peter A. Jones, VAI's chief scientific officer. “Together we are united with one goal: to provide groundbreaking new therapies and, ultimately, beat these diseases that steal away so many of our loved ones.” Clinical trials ongoing Among the clinical trials currently underway through the VARI– SU2C Epigenetics Dream Team are a slate of combination therapies that pair epigenetic drugs, which work by regulating when and to what extent the instructions encoded in our genes are carried out, with other medications such as those that boost the immune system. These potentially potent combinations are now being investigated in lung cancer, bladder cancer and blood cancers, among others, and may better fight these tough-to-treat diseases. “We’re trying to advance the standard of care by having new treatment options in the arsenal that an oncologist or a hematologist could have to fight patients’ cancers,” said Dan Rogers, the Institute’s clinical research manager. “From a patient perspective, clinical trials offer treatments that could possibly extend their life and give them good quality of life.” Progress can’t wait According to some estimates, it can take over a decade and more than a billion dollars to shepherd a new drug through the research, development and clinical trial phases before pharmaceutical companies can obtain federal approval to go to market. Still, despite the hard work poured into developing new therapies, more than 9 in 10 new drugs fail in development, while the diseases they are meant to treat continue claiming lives. That is where the Institute’s Linked Clinical Trials collaboration with The Cure Parkinson’s Trust helps by exploring whether drugs already on the market could be effective treatments for diseases they weren’t originally designed to treat. “Through LCT, scientists are looking at what drugs are already approved by the Food and Drug Administration to identify potential avenues for new treatments,” Rogers said. “This approach, coupled with work being done to discover new drugs, helps us have a broad impact in clinical research across the country and world.” For instance, clinical trials are ongoing to explore whether a number of drugs intended for diabetes treatment — including exenatide, lixisenatide and liraglutide — could also be treatments for people with Parkinson’s. That’s because the complex molecular mechanisms that give rise to both diseases overlap, meaning a drug designed to fix a problem in diabetes may also help fix a similar issue in Parkinson’s. A tailored approach A strong research portfolio and dedicated team of scientists positions the Institute to pursue the breakthroughs that benefit humanity. The Institute’s multipronged approach to clinical trials — both uncovering new drugs and seeking to repurpose old ones — reflects this. “By working with a collective of investigators and institutions, we leverage our scientific strength to have an impact,” Rogers said. “We work boldly to advance ideas that could change the standard of care for generations.” For more information on these clinical trials, visit vai.org/clinical-trials. DISEASES BEING TARGETED BY CLINICAL TRIALS Parkinson's disease Metastatic colorectal cancer Acute myeloid leukemia Myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) Small cell lung cancer Non-small cell lung cancer Bladder (urothelial) cancer Liver, pancreatic, bile duct and gallbladder cancers Breast cancers, triple negative or hormone resistant/ HER2-negative metastatic breast cancer 6 | VAN ANDEL INSTITUTE HIGHLIGHTS OF HOPE VAN ANDEL INSTITUTE HIGHLIGHTS OF HOPE | 7

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