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2017 Fall Highlights of Hope

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RESEARCH MAKING PROGRESS

RESEARCH MAKING PROGRESS AGAINST PARKINSON'S 200 Years After the First Medical Description of the Disease, Hope for a Cure Grows DR. PATRIK BRUNDIN. In 1817, an English surgeon named James Parkinson published An Essay on the Shaking Palsy, the first medical description of a disease that would later come to bear his name. Since then, the tireless work of scientists, physicians, advocates and people with Parkinson’s have revolutionized what we know about Parkinson’s, opening new avenues for a major change in how it is diagnosed and treated. For one, Parkinson’s likely isn’t a single uniform disease but rather a constellation of related conditions held together by common threads such as progressive loss of movement brought on by damage to dopamine-producing cells in the brain. We also now know that it starts decades before the most notable symptoms appear, often with innocuous signs like trouble sleeping and a loss of a person’s ability to sense odors. This early phase—called prodromal Parkinson’s disease—is of great interest to scientists, because it offers an incredible opportunity to potentially head off disease onset. But here’s the challenge: currently, there is no cure for Parkinson’s and available treatments only help manage symptoms, not slow or stop its progress. There is hope, however, and it may come from somewhere that appears suprising at first glance. On the surface, Parkinson’s and diabetes don’t appear to have much in common. But a deeper look reveals important overlaps that indicate some diabetes drugs could also disrupt the course of Parkinson’s, which one day may give people more years with fewer symptoms. Called drug repurposing, this approach has the potential to save precious time and resources, getting much-needed therapies to patients faster. “In my 35 years studying Parkinson’s, I’ve never been more excited and more optimistic about a new drug therapy than I am now,” said Dr. Patrik Brundin, director of Van Andel Research Institute’s (VARI) Center for Neurodegenerative Science and chair of the Linked Clinical Trials committee, a joint effort between VARI and UK-based The Cure Parkinson’s Trust. “Repurposing offers not just hope but a realistic shot that we will find a treatment that moves us closer to slowing disease progression.” As part of the Linked Clinical Trials initiative, several approved diabetes drugs, such as exenatide and liraglutide, are undergoing clinical testing to see if they can slow disease progress. Others, such as a compound called MSDC-0160, are not yet on the market but have demonstrated promising results in Brundin’s laboratory. Scientists also are making headway on more definitive ways to diagnose the disease, such as a simple blood test or brain scan. If all of these pieces come together, they could give physicians a powerful set of tools to delay the onset of symptoms and improve the lives of millions of people with the disease worldwide. “In my 35 years studying Parkinson’s, I’ve never been more excited and more optimistic about a new drug therapy than I am now.” - Dr. Patrik Brundin “In a perfect world, we would catch and treat the disease with drugs that slow or stop progression years, if not decades, earlier than we can even detect it now,” Brundin said. “We’re not there yet, but we’re making crucial steps in that direction. Positive change is coming.” All treatment decisions should be made in close consultation with a physician. It is critical that potential therapies, even those that are approved to treat other conditions, are thoroughly vetted for safety and effect in people with Parkinson’s before use in the clinic. For more information on these trials and others supported by VARI, please visit vai.org/clinical-trials. Drug Repurposing Using a drug that was developed or approved to treat one disease as a treatment for another. 2 | VAN ANDEL INSTITUTE HIGHLIGHTS OF HOPE

COMING TOGETHER TO FIGHT CANCER When it comes to combating cancer, collaboration is one of the most powerful tools in our arsenal. That’s why we teamed up with Stand Up To Cancer, the American Association for Cancer Research and other leading organizations, scientists and physicians two years ago to form the Van Andel Research Institute-Stand Up To Cancer (VARI-SU2C) Epigenetics Dream Team—to see what we can do when our collective expertise and resources are combined. The results to date are four clinical trials at sites across the country and abroad, all aimed at fighting a variety of cancers, including: MEMBERS OF THE VARI-SU2C EPIGENETICS DREAM TEAM. 1 2 3 4 Metastatic colorectal cancer, the second leading cause of cancer death in men and women combined in the U.S. Acute myeloid leukemia (AML), an aggressive blood cancer that is notoriously difficult to treat and has poor long-term survival. Myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), two blood cancers that are incurable with current drugs and that may progress to AML, a much more aggressive cancer. MDS and AML, which also are the subject of a small pilot study that is investigating whether a simple tweak to the standard care regimen may improve the current therapy’s ability to impede cancer cell growth and destroy cancer cells. These trials are a critical step on the road from the laboratory to the clinic; they ensure the treatments being tested are safe and effective. If successful, the drug combinations being studied could help improve the lives of people suffering from these devastating diseases. Learn more at vai.org/clinical-trials. It is with a heavy heart that we mourn the loss of Tom Isaacs, co-founder of The Cure Parkinson's Trust, a passionate advocate for the Parkinson’s community and friend of the Institute; who passed away in May. Following his diagnosis more than 20 years ago, Tom made it his mission to find a cure for Parkinson’s by supporting innovative research, engaging the patient community and bringing scientists, physicians and advocates together in paradigm-shifting collaborations. He was a force of nature whose vibrant personality and dedicated efforts have had and will continue to have a major impact on the pursuit for a cure. TOM HOLDING THE OLYMPIC TORCH. COURTESY OF THE CURE PARKINSON'S TRUST. VAN ANDEL INSTITUTE HIGHLIGHTS OF HOPE | 3

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