Progress PKD. Growing Future Leaders in PKD Research. Page 5. READ MORE ONLINE! pkdcure.org/pkd-progress. Fall 2015

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1 A magazine to inform and educate PKD patients and families, Foundation supporters, health professionals and researchers. Progress A magazine to inform and educate PKD patients and families, PKD Foundation supporters, health professionals and researchers. Fall 2015 Growing Future Leaders in PKD Research Learn why fellowship recipient Whitney Besse, M.D. chose to focus her talent on PKD research. Page 5 INSIDE: Mentoring rising stars: The next generation of researchers Promising drug trial made possible through Foundation support Celebrating cilia: Chief Scientific Officer talks about the importance of conference funding Families pay it forward through estate planning READ MORE ONLINE! pkdcure.org/pkd-progress

2 LEADERSHIP Dr. Jared Grantham We are dedicated to ensuring that talented people, who have the desire and skills to do the work, but not the funding, have the support they need. Co-Founder supports emerging PKD researchers Many positive influences in my life contributed to who I am today a physician scientist who helped discover how the PKD genes cause cysts to form and misbehave; a caregiver for PKD patients; and co-founder of and enthusiastic spokesman for the PKD Foundation. Standing out along the path to get here are compassionate mentors and generous donors who rallied in support of my personal growth and aspirational goals. In my youngest days, Dr. Ross Fields, a small town doctor, took me under his wing and introduced me to the wonders of the biological world. When I started college, my intent was to become a physician just like him. I took a different turn though, when Dr. Paul Schloerb, another mentor, steered me into researching how kidneys work. My boyhood friend, Ronnie Wilkerson, was my inspiration to work on PKD, which eventually took his life. Once committed, colleagues and other mentors challenged me to push the PKD research agenda because people with PKD were counting on me. With the magnanimous help of Joseph Breuning, I co-founded the PKD Foundation in 1982 to attack the disease on a strategic level. Very little was known about the disease before then. Simply stated only a handful of scientists cared or knew anything about PKD. We decided early on to spike the system by offering startup grants to bright, successful scientists who were looking for new challenges. Support through the Foundation allowed new researchers around the world set to work to figure out how cysts form and enlarge. The genes responsible for AD and ARPKD were discovered by 2002, opening the intricate, hidden molecular machinery within cyst cells to detailed analysis of new targets for therapeutic drugs and diets. Today, more than ever, early career scientists like I was need funding to get and keep them on the path of investigating these potential treatments. Funding from sources such as the National Institutes of Health is declining, and competition for grants is fierce. This is where the PKD Foundation comes in. We are dedicated to ensuring that talented people, who have the desire and skills to do the work, but not the funding, have the support they need. In this issue you ll read about fellowships the Foundation recently granted, and hear from one of the awardees and her mentor. You ll also read about work that is supporting promising projects and trials that might not otherwise take place. I wouldn t be here without my mentors, and these discoveries couldn t have happened without funding through the PKD Foundation. Though I m now retired, I m proud to still be an activist for an organization that continues to support scientists when they need it most. Sincerely, Dr. Jared Grantham Now showing Founder s Focus: Chatting with Jared Grantham, M.D. In this video series, Dr. Grantham shares his thoughts about research, his special connection to PKD, and how the Foundation came to be. You can also read the full story about his dedication to PKD and creation of the Foundation in his book Why I think about Urine.and a Treatment for Polycystic Kidney Disease. Visit pkdcure.org/pkd-progress 2

3 Stefan Somlo, M.D., on the impact of funding and mentoring scientific talent Funding fellowships is an important part of the PKD Foundation s focus on research. By doing so, we recognize early-career scientists who represent the future of PKD research that will lead to treatments and a cure. The relationship of mentor and mentee is critical to a successful and productive fellowship. Stefan Somlo, M.D., is vice chair of the PKD Foundation s Scientific Advisory Committee and mentor to one of the Foundation s new fellowship recipients, Whitney Besse, M.D. (see profile on Dr. Besse p. 5) Dr. Somlo s lab at Yale studies the human polycystic diseases of the kidney and liver with the goal of understanding the basic mechanisms that will lead to development of specific treatments. He is considered one of the major intellectual forces in PKD research, and his findings and insights form the cornerstones of current understanding of normal and abnormal kidney function in this disease. His early career was propelled by fellowship and research funding from the PKD Foundation more than two decades ago. That funding made a lasting impression on me, said Dr. Somlo. Obtaining grant support is an absolute requirement for a career in biomedical research. One of the hurdles to pursuing a career in research is the uncertainty about whether one can successfully compete for grants. Receiving funding from the PKD Foundation broke the ice and provided me reassurance that I could perhaps continue in this direction of research and be funded to do it. The decision to zero in on PKD research was inspired by his mentor, renowned genetics researcher Stephen Reeders, M.D., during his fellowship training at Yale. Dr. Somlo views that experience as more than a chance to be part of cutting-edge science. It offered a unique opportunity to impact human disease directly, and this was immensely important to me as a physician scientist. Over the years, Dr. Somlo s lab has grown into one of the preeminent PKD research hubs in the world. We ve had the opportunity to contribute to PKD research through identification of several disease genes, including the PKD2 gene and more recently through functional studies based on mouse models that closely resemble human ADPKD. That reputation helps him to recruit some of the best researchers. We ve had the good fortune to engage a number of outstanding colleagues at Yale into the field of PKD research. It s really elevated the experience for all of us and has produced an outstanding environment for training future investigators, like Dr. Besse, in the fields of cilia biology and PKD research. n Stefan Somlo, M.D. Photo Courtesy of Yale University CAREER HIGHLIGHTS A.B., Harvard College, Biochemical Sciences M.D., College of Physicians and Surgeons, Columbia University PKD Foundation fellowship and research grant recipient (1989) PKD Foundation Scientific Advisory Committee member (Vice Chair) C. N. H. Long Professor of Medicine (Nephrology), Professor of Genetics, and Chief of the Section of Nephrology, Yale School of Medicine Homer W. Smith Award from the American Society of Nephrology Lillian Jean Kaplan International Prize for the Advancement in the Understanding of PKD (PKD Foundation) RESEARCH Read Dr. Somlo s full interview about his PKD experience at pkdcure.org/pkd-progress. 3

4 RESEARCH PKD Foundation awards fellowships to promising PKD researchers The Foundation has selected five outstanding researchers as recipients of the 2015 PKD Foundation Fellowships. The fellowships recognize early-career scientists whose achievements and potential identify them as rising stars the next generation of scientific leaders in PKD research. Each fellow receives $50,000 a year for two years, totaling a half-million dollars. The caliber of applicants was impressive, said Terry Watnick, M.D., PKD Foundation Scientific Advisory Committee Chair. This fellowship aims to attract promising trainees to obtain significant research experience as they initiate and we hope spend long and productive careers in PKD research. The PKD Foundation wants to foster research to further our understanding of the physiological, biochemical and genetic mechanisms of PKD, and this is a key way to accomplish that. Fellowship recipients are: Jeong-In Baek, Ph.D., Medical University of South Carolina Dr. Baek is a post-doctoral fellow in the Department of Medicine, Medical University of South Carolina in Charleston. She earned a bachelor s degree in biology, a master s degree in molecular biology, and a Ph.D. in human genetics from Kyungpook National University in South Korea. She has authored over 21 publications (nine as first author). Her research is under the mentorship of Joshua Lipschutz, M.D., internationally known expert in renal ciliogenesis and PKD. Whitney Besse, M.D., Yale School of Medicine Dr. Besse earned a bachelor s degree in biomedical engineering from Brown University, a medical degree from the University of Connecticut School of Medicine, and completed residencies in internal medicine and nephrology at the Yale School of Medicine. Her mentor, Stefan Somlo, M.D., is director of the Center for Polycystic Disease Research at Yale School of Medicine. Marcelo Cassini, M.D., Ph.D., Yale School of Medicine Dr. Cassini earned an M.S. degree in kidney transplantation and a Ph.D. in kidney injury from the University of Sao Paulo Brazil. He received his medical degree at Federal Fluminense University in Rio de Janeiro Brazil and completed residency training in surgery and urology at State University of Rio de Janeiro. His research is under the guidance of mentor Lloyd Cantley, M.D., at Yale School of Medicine. Read more about the 2015 Foundation Fellowship recipients, visit pkdcure.org/fellowships. 4

5 Jacqueline Peda, Ph.D., University of Kansas Medical Center Dr. Peda earned her B.S. in biology at Washburn University, and an M.S. and Ph.D. in pathology at the University of Kansas Medical Center. She is pursuing post-doctoral training under the mentorship of Dr. Xiaogang Li at the University of Kansas Medical Center. Irfana Soomro, MBBS, New York University Langone Medical Center Dr. Soomro earned her MBBS (bachelor of medicine/surgery) at Dow Medical College Karachi, Pakistan. She is a clinical instructor in the division of nephrology at New York University Langone Medical Center, Bellevue Hospital Medical Center. Her research is under the mentorship of Edward Skolnik, M.D., director of the Division of Nephrology. n Whitney Besse, M.D. Young Yale researcher guided by mentor, Stefan Somlo, M.D. For as long as she can remember, Whitney Besse, M.D., has been fascinated by genetics. But it was her connection with PKD patients during her clinical nephrology training in medical school that steered her toward a career in kidney disease research. Dr. Besse is one of the five scientists awarded fellowships in 2015 through the PKD Foundation (see p. 4). In lay terms, Dr. Besse hopes that studying the genetics of patients with isolated polycystic liver disease can identify genes that control cyst development (kidney and liver) knowledge which would shed light on pathways to target for new PKD treatments. Her early interest in genetics was a natural progression to nephrology and the study of PKD. One patient in particular she met during her first year of clinical nephrology training helped seal her interest in research. The 19-year-old man had severe PKD, chronic kidney failure, suffered repeated life-threatening cyst hemorrhages and a double nephrectomy, and ultimately died from a brain hemorrhage. It was the role of the nephrology fellow to answer all of his PKD-related questions, make medical decisions, and help him and his family, said Dr. Besse. That tragic experience of having no cure for this brave young man is a lasting memory that serves as a reminder of my passion to find explanations and treatments for this disease. That s what she hopes to discover in Dr. Somlo s lab. And in an interesting coincidence, the story comes full circle. Her mentor, Dr. Somlo, was one of the PKD Foundation s first fellowship grant recipients in Now he s in a position to help others advance their careers in PKD research. (see profile on Dr. Somlo p. 3). Whitney Besse, M.D. Dr. Somlo is the vice chair of the PKD Foundation Scientific Advisory Committee (SAC) and the C.N.H. Long Professor of Medicine (Nephrology) and Genetics and section chief in Nephrology at the Yale School of Medicine. Under his guidance, Dr. Besse has defined the next steps in her career. Whitney has the most critical ingredients for success in this path she has the intellectual ability, work ethic, focus and exceptional motivation that will ensure her success, said Dr. Somlo. I feel very fortunate to have an opportunity to mentor such a talented individual and genuinely support her career development. Dr. Besse is happily settled full-time in her research. Her goal is to transition to a faculty position at Yale. Funding from the PKD Foundation will help her accomplish her dreams. I am very grateful for this fellowship opportunity. n There s more! Read Dr. Besse s full story at pkdcure.org/pkd-progress 5

6 RESEARCH Researchers in Dr. Bonnie Blazer-Yost s lab at Indiana University School of Medicine (from left): Shehab Ahmed, Ellen Maue, Ayodamola Otun, and Bonnie Blazer-Yost, Ph.D., photo courtesy of Indiana University-Purdue University Indianapolis School of Science PKD Foundation helps fund early trial for promising repurposed drug Funding from the PKD Foundation is facilitating clinical research of a drug initially developed for use in Type 2 diabetes that holds promise for patients with ADPKD. As part of our multi-faceted approach to research, the Foundation supports work that investigates new uses for previously approved drugs, called drug repurposing.this takes advantage of work that s been done for a different therapeutic use, dramatically reducing development time and costs. For example, aspirin was initially developed for pain, but it can also be used at lower doses to reduce the risk of heart attacks and colon cancer. As public funding dollars becoming harder to come by, academic labs that generate important, sometimes overlooked research are turning more to private sources like the PKD Foundation to supplement and sustain their work and test their hypotheses in patients. An example of how research in one area opened the possibility for potential advancements in PKD therapies is a surprising discovery of a drug used to treat Type 2 diabetes by Bonnie Blazer-Yost, Ph.D., Indiana University Purdue University Indianapolis (IUPUI) and Indiana University School of Medicine (IUSM). While working with scientists at GlaxoSmithKline, Dr. Blazer-Yost was researching why their insulin sensitizing agent, rosiglitazone, was causing fluid retention (edema). She happened upon an intriguing finding: the drug inhibited a chloride channel called the cystic fibrosis transmembrane conductance regulator (CFTR) the same chloride channel believed to be involved in cyst growth in PKD. In PKD, the chloride channel allows chloride to move into the cysts; water follows and the cysts enlarge. Studies funded by IUPUI and IUSM showed that both rosiglitazone and pioglitazone, another drug in the same class (also approved for Type 2 diabetes), were effective in delaying cyst growth in rat models, at concentrations significantly lower than the dose used to treat diabetes. They discovered that Actos (pioglitazone), a drug developed by Takeda Pharmaceuticals for use in Type 2 diabetes, blocked the synthesis and activity of the chloride channel. The implication: Actos could slow the growth of ADPKD cysts. (Continued on next page) To read more and learn about clinical studies, visit pkdcure.org/clinical-studies 6

7 Next step testing effectiveness in clinical trials In October, Dr. Blazer-Yost and co-principal investigator Sharon Moe, M.D., began enrolling candidates into a randomized, controlled, double-blind clinical cross-over pilot study with a placebo (an inactive substance) and pioglitazone (the active compound). A $42,000 grant from the PKD Foundation is funding the cost of medications to ensure the trial takes place. Without funding like this, completion of the trial would be at risk. This research has been the high point of my career, said Dr. Blazer-Yost. The stars have all aligned, and thanks to support from the PKD Foundation, we re able to move forward. The FDA is also supporting this study through its orphan drug products division. Private/public partnerships like this are critical in bringing financially at-risk studies to life. Drug repurposing is a key initiative to test certain marketed compounds whose safety profiles are already well known as potential treatments for PKD patients, said David Baron, Ph.D., Chief Scientific Officer, PKD Foundation. Dr. Blazer-Yost and Dr. Moe s grant proposal provided a compelling argument based on promising preclinical data. We re pleased to support this research that could translate into a major clinical advance for PKD patients. n This research has been the high point of my career. The stars have all aligned, and thanks to support from the PKD Foundation, we re able to move forward. Listen to Dr. Blazer-Yost s discussion through the recorded webinar at pkdcure.org/webinar-wednesdays. The REPRISE clinical research study is investigating if tolvaptan can reduce cyst growth and delay kidney failure in adults with autosomal dominant polycystic kidney disease (ADPKD). Approximately 1,300 people with ADPKD will participate in the study. You may be able to join this study if you: Are between the ages of 18 and 55 years old; patients aged 56 to 65 may be able to participate in some circumstances Are diagnosed with ADPKD There are other criteria that you must meet to participate. The study staff can discuss these criteria with you in greater detail. If you are eligible for the study, your participation will last approximately 15 months. Participating in a study is one way to be active in developing ADPKD treatments. Consider learning more today! 7

8 RESEARCH Foundation to fund new research grants In September, the Foundation released a Request for Application (RFA) for research grants in PKD. These two-year grants will provide $160,000 for meritorious research projects. We hope to fund 15 quality grants, and announce awardees in early In 2014 the PKD Foundation awarded 15 research grants from 51 applications submitted during the previous call for applications. This funding is critical to researchers. With research funding so highly competitive, it is difficult for newly independent researchers to build their program and for established researchers to maintain their operations. Grants are directed to laboratory research aimed at increasing understanding of the genetic and pathological processes involved in PKD, and therapy development. The awardees represent top researchers and physician scientists throughout the U.S., as well as internationally. New criteria this year include special consideration for research on autosomal recessive PKD and a collaborative grant with the PKD Foundation of Canada to fund a Canada-based researcher. Applicants must have an M.D., Ph.D. or equivalent degree and hold a faculty appointment at the institution where the research will be conducted. Applicants need not be United States citizens. To learn more about the RFA and current grant projects visit pkdcure.org/grants. The Mrs. Pamela Whitcomb Larsen Award Family funds grant to honor loved one There s not a day that goes by that Pamela Whitcomb Larsen s loved ones don t feel the loss. Pamela died at the age of 55 from leukemia nine years ago, but her memory lives on in the PKD Foundation s Mrs. Pamela Whitcomb Larsen Award which funds Jordan Kreidberg, M.D., Ph.D. s research at Boston Children s Hospital. Dr. Kreidberg s lab is focused on Wnt a specific pathway thought to be exaggerated in cells that form cysts. Research hasn t been definitive, so Dr. Kreidberg is aiming to prove that it s involved in cyst formation. Since pharmaceutical companies are looking for ways to develop inhibitors of the Wnt pathway, there s potential that these inhibitors could be used to target PKD. Pamela didn t suffer from PKD, but her husband David and teenage son Glenn do. David watched his mother struggle with PKD until she died at a young age. So when the chance to fund research that could impact treatments and a cure came along, Pamela s sister Lisa Whitcomb Capra and David were both on board. We were impressed with Dr. Kreidberg s research, says Lisa. We believe Pamela would approve supporting it, because research like this represents hope for her son and husband and others struggling with PKD. Pamela loved her family dearly, and I know she d be humbled and honored that the research is funded in her name. She d be smiling from the heavens. Lisa Whitcomb Capra with Pamela Whitcomb Larsen s sons Andy and Glenn To learn how you can fund a grant, Jackie Hancock, jackieh@pkdcure.org or Michelle Davis Wingate michelled@pkdcure.org. 8

9 David Baron, Ph.D. Chief Scientific Officer It was inspiring to see the collaborations among the more than 150 attendees from disciplines and institutions across the globe, all with a passion for basic research into cilia-based diseases. Listen to Dr. Baron talk about cilia through the recorded webinar pkdcure.org/webinar-wednesdays Celebrating cilia In July, I attended the Federation of American Societies for Experimental Biology (FASEB) Conference on Cilia and Flagella. This is a preeminent scientific meeting that promotes dialogue among prominent scientists, academic clinicians, postdoctoral fellows and graduate students working out the crucial roles these tiny organelles play in human development, physiology and genetic disease, most notably PKD. The PKD Foundation was a major funder of the conference, one of our strategic initiatives in supporting basic research most likely to lead to effective therapies and ultimately a cure for PKD. This particular FASEB Conference focused on areas of profound medical importance to the PKD community mutations in genes that code for the ciliary proteins polycystin 1 and polycystin 2. The meeting gathered some of the best minds leading a growing body of research into the function of these organelles. Cilia and flagella (a longer, motile version of cilia) are tiny hair-like structures on cells, that detect and convey important signals to the rest of the cell. Though first discovered over 100 years ago, cells with single cilia like those in the kidney were poorly understood until recently. Today, cilia are believed to play an important role in a number of human genetic disorders, called ciliopathies. PKD is one of the most common ciliopathies. In it, a mutation causes abnormal cyst growth crowding out normal kidney tissue resulting in eventual renal failure. So while incredibly tiny, these seemingly innocuous structures could hold the key to developing gene-based therapies. The more we can study ways to attack this most common genetic kidney disease at its root, the more potential there is for finding treatments. In addition to attracting many of the world s leading researchers like Harvard s David Clapham, M.D., Ph.D., and Jagesh Shah, Ph.D., several PKD Foundation colleagues presented and helped organize this conference. These included Scientific Advisory Committee members Stephan Somlo, M.D., Yale University; vice chair Iain Drummond, Ph.D., Massachusetts General Hospital; and Brad Yoder, Ph.D., University of Alabama Birmingham. The meeting concluded with special recognition from co-organizer and respected scientist Maureen Barr, Ph.D., Rutgers University, who noted that her early research career got a major boost through a PKD Foundation grant. It was inspiring to see the collaborations among the more than 150 attendees from disciplines and institutions across the globe, all with a passion for basic research into cilia-based diseases. More important, it affirms that the Foundation s strategy to underwrite scientific conferences like FASEB is a wise use of our funding dollars. By supporting targeted research into these critically important organelles, there s hope that we will discover better ways to treat people living with PKD. David Baron, Ph.D. Chief Scientific Officer 9

10 DEVELOPMENT Foundation supporters ensure their legacy through estate planning Naming the PKD Foundation as a beneficiary is a way to benefit future generations PKD is a disease that affects entire families and spans generations. One way Foundation supporters can have an impact on future generations is through estate planning. People who include the Foundation in their will ensure their legacy lives on by helping change the course of PKD for their children and grandchildren. Alisha and Colin Graham, Rosalie and Andrew Corbett, and Kathleen Cashman have different stories, but all want to help end PKD for their families by supporting the Foundation through estate planning. All adults, no matter what their age, should have an estate plan. Life is uncertain, even for people without PKD. Estate planning clarifies what s important to you, and for me, I want to end PKD. Grahams find peace of mind with estate plan in place When Colin and Alisha Graham got married, they promised to be there for each other in sickness and in health. That vow held special meaning for the young couple, who learned of Alisha s PKD when she turned 20. As she became increasingly involved in Chapter activities in Denver, Alisha and Colin determined that it was essential to include a contribution to the PKD Foundation in their estate plans. Over the years I began to realize how important a role the Foundation plays in the world of PKD, says Alisha. I transitioned from an annual donor to a monthly contributor, and further cemented that commitment by including the PKD Foundation in our very first will shortly after we married. When they began their estate planning, the Grahams realized that the funds would benefit not only their family, but the greater PKD community. The Graham family (from left) Alisha, Kenna, Colin and Tyler Photo Courtesy of Roka Studios 10

11 Our lawyer helped us structure our estate plan in a way that addressed the specific needs of our young family and also honored how important it is to our family to end PKD, says Colin. I feel at peace knowing I have addressed both of these dimensions for my family. Alisha s campaign to end PKD covers the spectrum. She s participated in the Denver Walk for PKD where they live, and attended the PKD National Convention in Last March she advocated along with other PKD and kidney disease patients in Washington, D.C. Eager to advance the science into treatments and cures, she s participated in two different PKD research studies. Alisha s mother, who has PKD, recently celebrated six years post-kidney transplant. Alisha never got to meet her grandmother, who died from a PKD-related aneurysm at the age of 44. My estate contribution is in memory of my grandmother and to honor the gift of life from my mother s donor. I often think of him and the unknown family that made such a beautiful, but wrenching decision. Their gift of life makes my gift pale in comparison. Estate planning is a way to take care of their family. By including the PKD Foundation in my will, I am taking care of my children and future grandchildren, says Alisha. It s a crucial component in so many areas that go into the battle against PKD. The Foundation is providing hope and support to so many people in all different points in their PKD journey. The Graham s advice to others? All adults, no matter what their age, should have an estate plan, they recommend. Acting sooner not later is important. Life is uncertain, even for people without PKD. Estate planning clarifies what s important to you, and for me, I want to end PKD, says Alisha. Corbetts hope to impact their daughter s future There are a myriad of reasons why including the PKD Foundation in your estate plan makes sense, but for Andrew and Rosalie Corbett, it comes down to one: their daughter Andrea s future. Rosalie has PKD and received a new kidney in 2015 after hers began to fail in her 50s. Andrea, 31, is already struggling with symptoms of the disease. Rosalie is a former Chapter Coordinator in Delaware and stays active in Walk events, as well as advocacy and awareness efforts. Andrew spreads the word by volunteering at the Walk for PKD, discussing it with people, and sharing on Facebook. Rosalie lost her mother to PKD, and I ve watched her face her situation with a lot of bravery, says Andrew. She s been a tireless fighter for the cause and a great role model for our daughter. Making a financial investment is their way of changing the legacy for Andrea and future generations. We wanted to do something that made a material difference in Andrea s future, and by extension others with PKD, says Andrew. I don t see any other organization invested in PKD the way the Foundation is. We re at a point where things are looking very promising in terms of research. Andrew, Andrea and Rosalie Corbett at the Delaware Walk for PKD. They deserve the resources they need to be successful in finding treatments and a cure. I want to make sure that tomorrow gets here. Rosalie believes getting involved is the best form of empowerment. You can sit back and do nothing, or you can become part of the solution, she says. Andrew and I give of our time and our money, and the PKD Foundation is part of our estate planning so that others with this disease can have a chance at a brighter future. (Continued on next page) We wanted to do something that made a material difference in Andrea s future, and by extension others with PKD. 11

12 DEVELOPMENT Kathleen Cashman (Continued from previous page) Kathleen Cashman wants to spare others At 76, Kathleen Cashman feels like she dodged a bullet. Though she doesn t have PKD, she s witnessed first-hand the toll it takes on family members. She nursed her mother through daily home dialysis for three years before she died. She saw her late aunt s painful PKD belly as it grew and bulged out of her side. She s watched other relatives go through transplantation, some with better results than others. It s gone down the line impacting my family, says Kathleen. She decided that including the PKD Foundation in her estate plans was one way to take action by supporting research and education. They re coming along with research, but there s so much more to be done, she says. More research will lead to finding treatments and a cure more quickly. For now she has peace of mind knowing that her financial commitment to the PKD Foundation represents hope for the future. I want something done about this. It s essential, not just to my family, but to the whole PKD community. They re coming along with research, but there s so much more to be done. More research will lead to finding treatments and a cure more quickly. Estate PLANNING GUIDE Estate Planning Guide Life is full of decisions some simple, some complex. But sometimes the biggest and most important decisions we need to make about our lives are delayed until the last minute because we d rather not think about them. Planning your estate is often one of those last-minute decisions. Frequently the source of people s hesitation is that they aren t fully aware of the options available to them so they don t know where to begin. The PKD Foundation s Estate Planning Guide is a resource to answer your questions. If you haven t started planning your estate, this guide will serve as a brief introduction to the options available to you. If your estate plan is in place, this guide can help you review your final instructions to make sure they reflect both your current wishes and the recent developments in tax laws. To receive your free guide, Jackie Hancock, jackieh@pkdcure.org. 12

13 Third annual Gratitude: A Celebratory Benefit April 9, 2016 Chicago The third annual Gratitude: A Celebratory Benefit for the PKD Foundation honors philanthropists, fundraisers, volunteers, leaders, researchers, and physicians who have made a significant impact on the work of the PKD Foundation and local Chapters. Their support has helped the Foundation make strides toward ending PKD. In addition to rasing vital funds to support the work of the Foundation, this special event brings together PKD supporters from the Midwest, and across the country to build stronger relationships and raise funds to fight PKD. Join Honorary Chair Wendy Brown, M.D., and Benefit Chairs Amy and Mike Manelli in congratulating our 2016 Gratitude honorees. Philanthropist and Fundraiser: Scott Goodman Volunteers: Tracee Hicks Dave Kappas Fundraisers: Jen and Pete St. Aubin Physicians and Researchers: Arlene Chapman, M.D. Vicente Torres, M.D., Ph.D. To learn more about the event and honorees visit pkdcure.org/gratitude. For sponsorship opportunities, contact Janea Roberts, Regional Development Director, at PKD.CURE ext Sponsors who sign up by Jan. 11, 2016, will be listed on the invitation. Ticket sales begin Feb. 1, People creating new products for better health worldwide The name Otsuka translates to major milestone. And indeed, for over 85 years, Otsuka s people have achieved major milestones in their quest to create new products for better health. Otsuka is hard at work investigating potential new treatments, with numerous compounds in various stages of development to treat disorders of the cardiovascular, gastrointestinal, respiratory, renal, and central nervous systems, and to treat cancer and ophthalmic disorders. We ve funded new research, supported new clinical trials, and pursued the development of new medications an unfaltering commitment of energy and resources with one goal in mind to create new products for better health worldwide Otsuka America Pharmaceutical, Inc. Otsuka Pharmaceutical Development & Commercialization, Inc. Otsuka Maryland Medicinal Laboratories, Inc Otsuka America Pharmaceutical, Inc. Board of Trustees ( term) Benjamin D. Cowley Jr., M.D. (Chair) Frank Condella Jr. (Immediate Past Chair, Vice Chair) Fran Towey (Treasurer) Anne Ryan (Secretary) Paul T. Conway Lisa Guay-Woodford, M.D. Michele Karl Klee Kleber Andrea Kringstein Brock Nelson Dwight Odland Craig Ramseyer Julia Roberts Jeffrey Rona Terry Watnick, M.D. Jerry Waxman Scientific Advisory Committee Terry Watnick, M.D. (Chair) Benjamin D. Cowley Jr., M.D. (Past Chair) Stefan Somlo, M.D. (Vice Chair) John Bissler, M.D. Alessandra Boletta, Ph.D. Iain Drummond, Ph.D. Michal Mrug, M.D. York Pei, M.D. Ronald Perrone, M.D. Richard Sandford, Ph.D., FRCP Darren Wallace, Ph.D. Angela Wandinger-Ness, Ph.D. Leadership Team Jackie D. Hancock Jr., Chief Executive Officer David Baron, Ph.D., Chief Scientific Officer Angela Connelly, MBA, Chief Marketing and Communications Officer Michelle Davis-Wingate, Chief Development Officer Ray Smith, CPA, MBA, Chief Operating Officer and Chief Financial Officer pkdcure.org pkdcure@pkdcure.org PKD.CURE 13

14 AWARENESS Celebrities put PKD in the spotlight Read their full stories at pkdcure.org/pkd-progress Celebrity chef competes on national TV and wins $16,000 Michael Psilakis, chef and owner of seven New York restaurants, competes in the kitchen for the PKD Foundation. He recently won $16,000 when he competed on Food Network s Guy s Grocery Games (thanks Michael!). He also competed on Chopped All-Stars, coming in at a close second. Michael fights for PKD in honor of his father who passed as a result of PKD related complications. New Orleans Saintsations cheerleader rallies squad to name PKD Foundation charity of choice Kriste Lewis made headlines last year as a 40-year -old cheerleader for the New Orleans Saintsations, and she s back again this year. Kriste has PKD and because of her, the Saintsations named the PKD Foundation as one of their charities of choice. Kriste also stars in the Do You Know PKD? video that has been viewed more than 60,000 times. Jeff Lorber, noted jazz musician, hits the airwaves about CD recorded for PKD Jeff recorded a special CD with fellow musicians, called BOP, to raise funds and awareness for the PKD Foundation. His work has been recognized in the jazz world, resulting in numerous media interviews. BOP even topped the charts in Japan! Jeff received a kidney transplant from his wife. Insert Photo Of You You, too, can put PKD in the spotlight: everyone is a celebrity to someone. You re famous to friends, family members, even co-workers and neighbors. Do THEY know about PKD? The best way to spread the word is for one person to tell another person and on and on. Be that ONE person who gets the chain going about PKD in your circle. There are many ways to get the word out, including sharing Kriste s Do You Know PKD? video and wearing PKD gear. Learn more at pkdcure.org/aware Education programs Webinar Wednesdays PKD Foundation Chief Scientific Officer David Baron Ph.D. speaks about the science of PKD and takes questions the third Wednesday of each month (Noon Central Time). Next webinar: Nov. 18 Genetics of PKD Learn about upcoming topics and register at pkdcure.org/webinar-wednesdays Dr. Baron will also lead a special webinar: The Sequence of Drug Development Dec. 2, 7 p.m. Central Time Register and view past webinars at pkdcure.org/webinars. For more information and to view past webinars, visit pkdcure.org/webinars. 14

15 ORLANDO JUNE 24 TO 26, 2016 Registration opens in January. Learn more at pkdcure.org/convention Chance to rub elbows with top PKD experts is reason Jean Sommer plans to go again Jean Sommer has Orlando in her sights. She s already made travel plans to attend the PKD Foundation s PKD National Convention, June 24-26, I wouldn t miss it! says the St. Louis, Mo., Chapter and Walk Coordinator. She attended the Convention in Kansas City in 2014 and the knowledge she gained and friendships she formed there were invaluable. I enjoyed all aspects, especially meeting the researchers and hearing more about their work. My husband and I attended different seminars so we could learn as much as possible. Jean first learned about her PKD and polycystic liver disease (PLD) in She made a conscious decision to have a positive outlook and enjoy life. I am facing my fears and the unknowns by learning as much as I can so I am prepared for the day when my kidneys fail. Attending the Convention is one way to do that. I highly recommend attending the PKD national convention, Jean said. All the top dedicated experts, researchers, physicians and PKD Foundation staff come together to provide PKD patients and their families the opportunity to learn more about their disease while providing the latest information on PKD research. PKD Teen program at Convention helped Brittnee Boyd find her voice Initially, Brittnee Boyd had reservations about attending the PKD National Convention She wasn t sure the Convention s PKD Teen program was her cup of tea. But any doubts Brittnee had quickly faded as she connected with teenagers she shared a unique bond with living with PKD. It was cool to hang out with people my age in the same boat, said Brittnee. We learned so much about the latest research, and understanding your numbers and taking responsibility for your own health. And it was fun. We hung out and went bowling and out for ice cream. It s a great place to make new friends. Brittnee is the first known family member with PKD, which makes her situation especially isolating. Asymptomatic until she was 14, she s spent the last several years in and out of ERs and hospital rooms coping with kidney stones, infections and pain. Her mother is Amy Boyd, the Chapter Coordinator in Las Vegas. Amy was happy to see Brittnee flourish among other teens and young adults. Brittnee s always been quiet about her PKD, but the Convention gave her a safe place to talk about it among peers who truly understand what it s like to be a young person living with PKD. She was able to find her voice, and now she shares her experience with others to raise awareness and be an advocate for people living with the disease. Amy recommends attending the Convention as a parent. The PKD Parent seminars were informative, and the chance to talk with other parents and share experiences and ideas was invaluable. They re excited about the Orlando Convention. I would definitely tell other teenagers to go in 2016, said Brittnee. There s so much to gain. Jean Sommer Brittnee Boyd (right) made new friends like Lena Keys at the PKD National Convention

16 Are you or a loved one affected by PKD? Join us at the PKD National Convention 2016! Learn more at pkdcure.org/convention