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2016 Annual Report

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Battling Parkinson's -

Battling Parkinson's - Embracing Advocacy “He said, ‘You have Parkinson’s; don’t freak out,’” she recalled. “I decided I could curl up and be miserable, or I could do whatever it takes to make every day the best that I could.” December 18, 2013, is the day Alison Sheltrown’s world changed. For two years, the then 41-year-old had been plagued by stiffness and pain in her right shoulder that eventually began radiating down her arm, interfering with her DR. PATRIK BRUNDIN AND ALISON SHELTROWN. intensive martial arts training and everyday tasks like writing. At first, she chalked it up to normal wear and tear, but when surgery for a herniated disk didn’t fix it and new problems cropped up, including a small twitch in her right leg, her doctor referred her to a neurologist. Sheltrown was shocked. Sheltrown threw herself into helping others who are newly diagnosed with the disease through her social media presence and spreading the word about the benefits of exercise, which has been shown to help people with Parkinson’s maintain muscle control. She’s also a passionate advocate for research and frequently volunteers with Purple Community, Van Andel Institute’s (VAI) grassroots program. “If MSDC-0160 is as successful in the clinic as it was in lab models, it could be a game-changer for millions of people with Parkinson’s around the world...” Dr. Patrik Brundin “It’s important that we have a place like VAI that is actively researching to find cures, not just treatments,” she said. “Just knowing that it’s happening in my hometown makes me thankful that we have this kind of research here and that we have the opportunity to be a part of it.” 16 | VAN ANDEL INSTITUTE ANNUAL REPORT 2016

RESEARCH In many ways, Parkinson’s disease is an enigma. Its symptoms, age of onset and progression can vary widely from person to person, although it is typically diagnosed after age 60. With the exception of a small percentage of cases that may be traced genetically through families, there is no firmly established cause. And most problematic, there is no definitive test to diagnose it and no cure. Only a few treatments for symptoms exist, and none that slow or stop it. But that may soon change. MSDC-0160, a drug originally developed for type II diabetes, has shown exceptional promise in impeding Parkinson’s in laboratory models, preserving critical brain functions that are lost as the disease advances. It was created just down the road in Kalamazoo by Metabolic Development Solutions Company, which is working closely with the Institute and UK-based research charity The Cure Parkinson’s Trust to move it into human clinical trials. “If MSDC-0160 is as successful in the clinic as it was in lab models, it could be a game-changer for millions of people with Parkinson’s around the world,” said Dr. Patrik Brundin, head of the Institute’s Center for Neurodegenerative Science and the senior author of a 2016 paper describing the work. “We know more about Parkinson’s disease than ever before. Thanks largely to the stunning breadth of collaboration in the scientific, medical and patient communities, we have an unprecedented opportunity to have a real, life-changing impact.” MSDC-0160 isn’t alone. Another diabetes drug, exenatide, and a respiratory drug, ambroxol, also have shown promising results in the laboratory and in early human trials for slowing Parkinson’s progression and are already being studied in the clinic as part of the Linked Clinical Trials initiative, a joint effort between The Cure Parkinson’s Trust and the Institute. “To know that there’s somebody else that’s going to battle for me and that they found something that could potentially just knock this disease in the teeth really makes me proud and happy and excited and thrilled,” Sheltrown said. “It’s one of those crying-tears-of-joy moments, not just for me but for so many people I know.” Other breakthroughs are on the horizon, fueled by collaborations between scientists at the Institute and their colleagues, both in Grand Rapids and around the world. By teaming with experts in other fields, VARI scientists are making significant inroads in understanding what makes Parkinson’s tick—and how to definitively diagnose and treat it sooner and more effectively. These efforts are taking aim at all facets of the disease, from the underlying molecular cause to disease progression to therapeutic development. It’s these advances that Sheltrown shares with others, along with a message of strength and solidarity. What are clinical trials? Clinical trials are a critical step on the road from the laboratory to the clinic. These rigorously designed and managed studies help ensure new therapies are not only safe in humans but also effective. Although VAI does not host trials on-site or treat patients, many of its scientists and physician-scientists participate in trials at collaborating organizations. The Institute also “To know that there’s somebody else that’s going to battle for me and that they found something that could potentially just knock this disease in the teeth really makes me proud and happy and excited and thrilled...” Alison Sheltrown “If I have one thing to offer, it’s this—don’t give up hope,” she said. “You have to live your life, love people and love yourself, and stay hopeful.” is proud to support the development of potentially lifechanging therapies through the VARI–SU2C Epigenetics Dream Team and the Linked Clinical Trials initiative, multi-institutional collaborations that help move promising drugs into the trial process. For more information on clinical trials, please visit www.clinicaltrials.gov. VAN ANDEL INSTITUTE ANNUAL REPORT 2016 | 17

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