Progress PKD. new PKD research grants awarded. Page 4. Spring READ MORE ONLINE! pkdcure.org/pkd-progress

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1 Progress PKD A magazine to inform and educate PKD patients and families, Foundation supporters, health professionals and researchers. Spring new PKD research grants awarded Learn what four of the researchers are focused on and why they ve dedicated their careers to PKD. Page 4 Grant awardee Dr. Stephen Parnell is dedicated to ending PKD for his family. Page 7 READ MORE ONLINE! pkdcure.org/pkd-progress

2 LEADERSHIP As CEO of the PKD Foundation, I ve had the unique privilege of meeting and working with incredible scientists who are doggedly pursuing an end to PKD. I m consistently impressed with how talented they are, and dedicated to PKD. The Foundation is the common thread that binds them. Promising projects that could lead to therapies can begin and continue because of Foundation funding. David Baron, Ph.D, PKD Foundation Chief Scientific Officer (CSO), talks about how this is a time of breakthroughs in PKD science (see page 10). I m proud to be a part of the group making much of that work possible. There are now more than 30 PKD Foundation-funded projects underway. Without the Foundation s financial support, this work might not be possible. In this issue you ll read about research grants recently awarded, and hear from four of the scientists leading the projects. They all share the same sentiment that funding from the Foundation is critical in moving PKD science forward. This round of grants includes work focused on autosomal recessive PKD (ARPKD) and polycystic liver disease (PLD), and explores the use of the latest technology. READ MORE Learn about the ASN Fellowship program at pkdcure.org/news/jared-grantham-fellowship. Follow Jackie on Leadership Letter In the last issue of Progress we talked about five amazing new fellows we are funding (see pkdcure.org/ researchfellowships). We also recently partnered with the American Society of Nephrology (ASN) Foundation for Kidney Research to fund a fellowship in perpetuity. Our $500,000 commitment was matched by $1.5 million from the ASN, tripling our investment and potential in attracting new talent to PKD research through the Jared J. Grantham Research Fellowship. Funding of grants and fellowships supports the momentum of PKD science by providing resources and support to scientists when they need it most. I hope you re as excited as I am in celebrating such a vibrant, committed group of researchers focused on finding treatments for PKD. Jackie Hancock Jr., CFRE PKD Foundation CEO 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, M.D., Ph.D, FRCP Darren Wallace, Ph.D. Angela Wandinger-Ness, Ph.D. Leadership Team Jackie D. Hancock, Jr., CFRE, Chief Executive Officer David Baron, Ph.D., Chief Scientific Officer Angela Connelly, MBA, Chief Marketing and Communications Officer Ray Smith, CPA, MBA, Chief Operating Officer and Chief Financial Officer 2

3 PKD Foundation funding keeps momentum going on promising research consistent theme heard from A PKD researchers is that there is no single source of funding for PKD reseach. Often, early support from the PKD Foundation provides seed money for new projects to start, or enables projects initially funded by the Foundation to gain funding by the National Institutes of Health (NIH). Thanks to a grant from the PKD Foundation in 2014, Vishal Patel has been able to sustain his research initially started with funding by the NIH. Dr. Patel is one of the recipients of 15 PKD Foundation research grants awarded in He received a twoyear, $160,000 award to pursue his project on the Role of mir-17~92 in the Pathogenesis of PKD. The timing was crucial for Dr. Patel, whose initial NIH award funding was in its last year. He d recently published a seminal paper in the scientific journal, United States, Proceedings of the National Academy of Sciences of the United States demonstrating his lab s success in inactivating microrna to inhibit cyst growth in mouse models and identifying it as a drug research target for autosomal dominant polycystic kidney disease (ADPKD) in humans. The PKD Foundation grant helped to sustain momentum on research used as preliminary data in applications to NIH to further explore his findings. This was an exciting but very early idea one that the NIH likely would not have funded, says Dr. Patel. I m grateful the PKD Foundation found the idea interesting enough to fund. That support could not have come at a better time. Thanks in part to the PKD Foundation grant, Dr. Patel received renewed NIH funding. He continues to publish important findings in major scientific journals, and he s established a collaborative partnership with a biotech company to develop microrna-based drugs for PKD. I m drawn to PKD research because there is a sense of optimism that a therapeutic breakthrough is near. Fast-paced discoveries and access to inspiring mentors are what initially attracted Dr. Patel to PKD research. One of those mentors is Peter Igarashi, M.D., recipient of the Lillian Jean Kaplan International Prize for Advancement in the Understanding of PKD. The award was established by the PKD Foundation and the International Society of Nephrology through the generosity of Thomas S. Kaplan and Daphne Recanati Kaplan of New York, in memory of Tom s mother, Lillian Jean Kaplan, who had PKD and died in Dr. Patel joined Dr. Igarashi s lab at the University of Texas Southwestern Medical Center in Dallas in In 2010 he established his own lab at UT Southwestern, where he mentors up-and-coming PKD physician-scientists, and is active in efforts to launch a comprehensive PKD clinic at the medical center - a potential site for future clinical trials. n Dr. Patel (middle) and his daughter Aarna, with North Texas Chapter Coordinators Sally Wilson (left) and Carolyn Morris (right). EDUCATION AND CAREER HIGHLIGHTS P.S. Medical College, India (medical school) Northwestern University, Evanston Hospital, (Internal Medicine Residency) University of Texas Southwestern Medical Center (Nephrology fellowship) University of Texas Southwestern Medical Center (Basic research fellowship) PKD Foundation Research Grant Recipient Rising star in Nephrology - State of Texas, Super Doctors National Institute of Diabetes and Digestive and Kidney Diseases - Career Development Award RESEARCH I m grateful the PKD Foundation found the idea interesting enough to fund. That support could not have come at a better time. University of Texas Southwestern O Brien Center - Pilot and Feasibility Grant Chief Fellow, University of Texas Southwestern Medical Center 3

4 RESEARCH New PKD Research Grants Awarded 1 in Canada Awarded 15 research grants $80,000 per grant per year 2related to PLD & 3Related to ARPKD 1 in Europe 13 in the U.S. READ THE FULL SUMMARIES of all grants awarded at pkdcure.org/grants Grant awardees Stephen Parnell, Ph.D., and Xia Zhou, Ph.D., at work in the laboratory at the University of Kansas Medical Center. The PKD Foundation recently awarded grants to 15 outstanding PKD researchers. The Foundation s Scientific Advisory Committee (SAC) reviewed 51 research grant applications to determine which ones would merit funding; a total of $2.4 million over the next two years. This investment will propel critical research to broaden our understanding of the genetic and pathological processes involved in PKD and to accelerate the development of potential therapies for PKD patients. David Baron, Ph.D., PKD Foundation Chief Scientific Officer, said I am particularly excited about the quality and scientific diversity of grants this year. Grant topics include basic mechanisms of ADPKD and ARPKD, that provides the foundation for advancements, and new clinical approaches. Three specifically focus on ARPKD, and two focus on polycystic liver disease (PLD). We interviewed four awardees about what they hope to accomplish, and what this funding means to their work and PKD science. While these researchers come from different backgrounds, and study different aspects of the disease, they re all focused on one thing -- ending PKD. n Meet the researchers at the PKD National Convention. Grant awardees Dr. Dell, Dr. Parnell and Dr. Freedman will be there to answer questions. Register at pkdcure.org/convention 4

5 Dr. Parnell, Dr. Zhou and research assistant Brittany Crain look at cystic kidney samples. Grant Awardees 2016 PKD Research Grants Grant Title Dr. Vincent H. Gattone, Research Award for the Top-rated Grant Proposal Katherine M. Dell, M.D. Case Western Reserve University Cleveland Magnetic Resonance Fingerprinting (MRF) to Assess ARPKD Kidney and Liver Disease Progression David Beier, M.D., Ph.D. Seattle Children s Research Institute Seattle Sorin Fedeles, M.S., Ph.D. Yale School of Medicine New Haven, CT Brunella Franco, M.D. Telethon Institute of Genetics and Medicine Naples, Italy Benjamin Freedman, Ph.D. University of Washington Seattle Edward Inscho, Ph.D. University of Alabama at Birmingham Birmingham, AL Timothy Kline, Ph.D. Mayo Clinic Rochester, MN Tetyana Masyuk, Ph.D. Mayo Clinic Rochester, MN Stephen Parnell, Ph.D. University of Kansas Medical Center Kansas City, KS York Pei, M.D. Toronto General Hospital Toronto, Canada Feng Qian, Ph.D. University of Maryland School of Medicine Baltimore Edward Skolnik, M.D. New York University Medical Center New York Zhaoxia Sun, Ph.D. Yale School of Medicine New Haven, CT Bradley Yoder, Ph.D. University of Alabama at Birmingham Birmingham, AL Xia Zhou, M.D., Ph.D. University of Kansas Medical Center Kansas City, KS Screening for Modifiers of PKD Severity Using ENU Mutagenesis (The Dan Larson Research Grant) Examining the Role of XBP1 in the Pathogenesis of Protein Folding-associated Polycystic Kidney Disorders The Role of Autophagy in Renal Cystogenesis Modeling Human PKD Cystogenesis with Pluripotent Stem Cells Renal Vascular Function in ARPKD Development of a Computer-aided Decision Support System for PKD Deregulated Cholangiocyte Autophagy: A New Target for Polycystic Liver Disease (The Eileen Creamer O Neill Award) Polycystin-1 Mediated Cyst Regression Genetic Modifiers of Severe Polycystic Liver Disease (Grant co-funded by the PKD Foundation of Canada) Role of Fibrocystin/Polyductin in Health and ARPKD A Novel Screen to Identify Kinasis that Are Activated in PKD Kidneys Vascular Integrity in Zebrafish PKD Models Injury Response Mediated Pathogenesis in Ciliopathies Functional Roles of DNMT1 in Autosomal Dominant Polycystic Kidney Disease 5

6 RESEARCH Katherine M. Dell, M.D. Case Western Reserve University Vincent H. Gattone Research Award for the Top-rated grant proposal ; Magnetic Resonance Fingerprinting (MRF) to Assess ARPKD Kidney and Liver Disease Progression INTERESTING FACTS Three grants awarded will focus on autosomal recessive polycystic kidney disease (ARPKD), a rare form of PKD that affects approximately 1 in 20,000 infants. Dr. Dell s research will focus on ARPKD and liver fibrosis, a significant related aspect of ARPKD. Dr. Dell was a member of the Foundation s Scientific Advisory Committee (SAC) from 2002 to 2006 Several new therapies have shown promise in ARPKD animal models, however, they haven t been studied in patients because there are no clinically-available, reliable, non-invasive ways to measure the effects of treatment. Magnetic resonance imaging (MRI) may provide this key missing piece, but MRI studies are long and require patients to sit still, which is not possible for young children. A normal MRI can take an hour and a half and, when performed in children, often requires sedation, introducing additional risks. Dr. Dell s research aims to establish a new method, called magnetic resonance fingerprinting (MRF), as a non-invasive and faster way to get the information needed for clinical studies. With MRF, the scanner can take multiple images simultaneously rather than sequentially, which makes the procedure a lot faster. Ultimately, Dr. Dell hopes that the safe and non-invasive nature of MRF will lead to more clinical trials with ARPKD patients of all ages. Dr. Dell has dedicated her career of over 20 years to ARPKD because of the connection to her patients. What first made me interested in the disease was intellectual curiosity, but what has continued to inform my decisions about what I want to research are the patients. Parents are looking for a treatment. Having people pay attention, and knowing they are looking for therapies and trying to help matters a lot. In addition to being a practitioner, Dr. Dell is also a clinical researcher, so she knows what is needed for clinical trials. The questions in the lab have come out of the questions in the clinic (working with the children and their parents). That s really what has driven me. With her perspective as a scientist and previous Scientific Advisory Council (SAC) member, Dr. Dell knows it is incredibly important that the PKD Foundation continues to fund research grants. Disease specific foundations play an important role because they recognize the importance of these projects and may be more willing to take risks for novel ideas that may not work but would be amazing if they did. She became interested in genetic and renal disease, including ARPKD, during her pediatric clinical rotation when she was a fellow at Children s Hospital of Philadelphia. Her Division chief and medical director was Bernard Kaplan, M.D., who conducted early research in ARPKD, and is one of the pioneers of PKD research. We re making progress. I m very excited about this grant because I feel recognition of some of the work we ve done already and, importantly, that what we re doing matters. 6

7 Stephen Parnell, Ph.D. University of Kansas Medical Center Polycystin-1 Mediated Cyst Regression INTERESTING FACTS Dr. Parnell has multiple family members who have PKD, including his father. When Dr. Parnell was a young boy, his father was sent to KU Medical Center to visit the world s preeminent PKD scholar and co-founder of the PKD Foundation, Dr. Jared Grantham. Years later, Darrell Parnell received his kidney transplant at KU Medical Center while Dr. Parnell was there doing his Ph.D. research on PKD. Dr. Parnell s grandfather (left), uncles, and father (right) When Dr. Parnell goes to a family event, they ask him how work is with a different intensity. Dr. Parnell s grandfather died from PKD before dialysis was widely available, and he passed it to all three of his sons Dr. Parnell s father and uncles. One uncle lived for many years after a transplant, but the other died at 48 due to PKD-related complications. His father, who also received a transplant, is the only member of his family with PKD to live beyond 80. Dr. Parnell is researching to find treatments for his own family s disease. By studying a protein called polycystin-1, Dr. Parnell hopes to unlock the secrets of what it does and why renal cysts form when you lose it. To do this, he engineered a mouse that develops PKD due to deficient polycystin-1 that can be turned back on (called re-expression). He will study what happens when it s re-expressed, to determine if a cyst has the ability to get smaller or even disappear. Most current studies are aimed at slowing cyst growth, but this project suggests we might be able to actually reverse it. Serendipity brought Dr. Parnell into the PKD field and to the Kidney Institute at the University of Kansas Medical Center (KUMC). He wanted to pursue a Ph.D. in biochemistry, and having grown up in Topeka, he called the KUMC. He ended up on the phone with his future graduate student mentor, James Calvet M.D., who would later become a recipient of the Lillian Jean Kaplan International Prize for Advancement in the Understanding of PKD. When Dr. Calvet commented that he worked on...a disease that you ve probably never heard of, PKD... Dr. Parnell immediately knew that s where he wanted to study. Dr. Parnell s postdoctoral studies took him to other areas of scientific interest, but a fellowship through the Foundation allowed him to get back into PKD research. I would not have been able to get back into the PKD field without that fellowship from the Foundation. Without it, I might have been forced into a different field. Funding is essential for projects like Dr. Parnell s to continue. We are not working on an easy problem, and the work is expensive. It really helps to have these funds to make the work go faster, get the right materials, and have another set of hands to help. It s absolutely vital, I can t state that enough. It s really quite amazing the pace of progress in PKD compared to other diseases. I think that is because there are very dedicated people who are supportive of the PKD Foundation, and equally dedicated people who have invested their lives in solving the problem of PKD. 7

8 RESEARCH I m excited to see if we can learn what components are most essential to cyst growth, then translate that into potential therapies. INTERESTING FACTS Dr. Freedman spoke about new gene-editing technology called CRISPR and how it could transform the future of PKD care. Hear the recording at pkdcure.org/ newgeneediting. Dr. Freedman wrote a PKD Connection blog entry about minikidney organoids. Read it at pkdconnection. org/research/the-pkdresearch-experience. Benjamin Freedman, Ph.D. University of Washington Modeling Human PKD Cystogenesis With Pluripotent Stem Cells As Dr. Freedman graduated with his Ph.D. from the University of California Berkeley, he became more aware of people with medical problems and wanted to use his knowledge to help them. He asked his mother what he should focus on, and she reminded him they had friends with PKD. That s how his path as a PKD researcher started. Dr. Freedman is approaching PKD from a different angle. He s focused on generating new kidney replacement tissue from a patient s own cells that can be studied for root causes of the disease and used to test drug candidates for future clinical trials. Ultimately, he hopes to produce an entirely new kidney. His project will take advantage of stem cell technology that has only been invented in the past 10 years. In 2007, scientists discovered they could take skin cells from adult patients and essentially turn back the clock to a very early stage, turning the adult cells into stem cells that can develop into any type of tissue. Dr. Freedman made such stem cells from PKD patients with the idea of studying them and using them for regeneration. Dr. Freedman s first major breakthrough was when he and several other researchers first turned stem cells into actual kidney cells, something that had never been done before. People had made heart, liver, and other types of cells out of stem cells but nobody had ever actually made kidney tissue. When we figured out how to do this, the tissue that formed was organized into structured mini-kidney units or organoids that had pieces of kidney tubule in a specific order. It looked like what you would see in an actual kidney. That was a thrilling discovery. Soon after this discovery, Dr. Freedman did the same thing with stem cells carrying mutations that cause PKD. Remarkably, in this case, the mini-pkd organoids formed cysts, which looked like large balloons in the cultures. These cysts were only observed in the mini-pkd organoids, but not in organoids that lacked PKD mutations. That was a very exciting series of discoveries. We re at the tip of the iceberg in terms of what we can do with these cells. Dr. Freedman is grateful for the PKD Foundation grant. The grant will give us the opportunity to turn this into something that can be used to find new therapies. He also appreciates the role of the Foundation beyond funding. The PKD Foundation is a source of connection to the PKD world, to the patients and other PKD researchers. As a young person relatively early in my career, I m very thankful for the support and the opportunity to connect to the Foundation at a deeper level than I had before. YOU CAN HELP FUND RESEARCH Over the next two years, the PKD Foundation will spend $2.4 million to fund critical research that will help find treatments for PKD. To learn how you can fully fund a research grant, which can be named after you or a loved one, Jackie Hancock, CEO, jackieh@pkdcure.org. 8

9 York Pei, M.D. Toronto General Hospital Genetic Modifiers of Severe Polycystic Liver Disease (spld) Most people affected by PKD understand how kidneys can grow so big they may affect an individual s ability to move, eat, and sometimes even breathe. However, many may not know that some people with PKD have a rare complication called severe polycystic liver disease (spld), which may cause their liver to grow four to eight times larger than the size of a healthy kidney. People with spld experience symptoms described as a mass effect, which may include feeling full after a small meal, shortness of breath, and abdominal pain and swelling. The treatment for spld is very limited, and many patients require a liver transplant because the mass effect impact on their health and quality of life. Dr. Pei has seen many patients with PKD over the past 20 years while running kidney disease programs, and he knew there was little to be done for those with spld. He saw an opportunity to use his genetics training with new technology in studying the gene. Dr. Pei s project is focused on identifying the genetic factors behind spld, with hopes it will lead to studies in animal models and new targets for therapies. Funding from the PKD Foundation will allow Dr. Pei to use new technology called Next Generation Sequencing, and study a large enough group of people to substantiate findings. We have experience with this technology but it s expensive. Most of the grant will fund the cost of doing this kind of sequencing in a large enough number of patients from multiple international centers to find the variance that may be underpinning spld. Dr. Pei has worked with PKD patients as a physician and researcher for most of his career. It s becoming a life pursuit to think about PKD and how to treat PKD because of my close association both as a physician and a researcher. INTERESTING FACTS Dr. Pei co-edited the new PKD Patient Handbook. To get your copy visit pkdcure.org/handbook. Two of the grant projects will focus on polycystic liver disease (PLD). Dr. Pei s work will focus on understanding the genetic factors that lead to severe PLD, a rare complication of ADPKD. Dr. Pei is a member of the PKD Foundation s Scientific Advisory Committee (SAC). (He was not involved in the grant review process). Fifty percent of Dr. Pei s research will be funded by the PKD Foundation of Canada (PKDFOC). The PKDFOC has committed $40,000 per year for the next two years. Dr. Pei observes that The PKDFOC has significantly grown in helping advocate for patients, raise money and support research. It s a natural collaboration. As physicians we are somewhat limited and for a long time we had nothing to offer. We are entering a different era in which much of the research done by others in the PKD field is coming to fruition in the way of tools and therapies. 9

10 AWARENESS 10 David Baron, Ph.D. Chief Scientific Officer (CSO) Collaboration with the Kidney Health Initiative (KHI) When KHI was launched in 2012 to foster therapy development for kidney diseases, it was only natural for the PKD Foundation to participate. KHI, led by the American Society of Nephrology (ASN) and the Food and Drug Administration (FDA), brings together key kidney health stakeholders. These include health professionals, patient groups, pharmaceutical companies, device manufacturers, academic research, and governmental and non-profit organizations. The common goal is to achieve breakthroughs in kidney disease treatment. According to former FDA Commissioner, Margaret Hamburg, M.D., the statistics of kidney disease are catastrophic. One in nine Americans has chronic kidney disease (CKD) and about 600,000 are in endstage renal disease (ESRD). Kidney disease kills more people than other diseases such as breast and prostate cancer, which get far more attention. The cost of treating CKD and ESRD is enormous. ADPKD and ARPKD represent eight to 10 percent of people with ESRD, which requires a transplant or dialysis. In the long run, dialysis is more costly than a transplant for patients and Medicare. Better treatments and more living donors could save Medicare a billion dollars a year or more. KHI aims to tackle this gap in treatments of CKD and ESRD. Last May I presented at the KHI Third Annual Stakeholders Meeting, on Achieving Consensus on Clinical Trial Biomarkers and Endpoints to Facilitate the Development of Treatments of ADPKD Early in the Course of the Disease. This is because treating PKD early, when kidney function is still relatively normal, is more likely to be effective at preserving normal kidney tissue by slowing or stopping cyst growth. Treatment in later stages, when cysts are already very large, may pose a greater challenge to preserving the small amount of normal kidney tissue remaining. Through this partnership, and largely the efforts of Ronald Perrone, M.D., at Tufts University, who serves on the Scientific Advisory Committee of the PKD Foundation, Total Kidney Volume (TKV) was established LISTEN TO DR. BARON talk about Total Kidney Volume and PKD through the recorded webinar pkdcure.org/webinarwednesdays as a prognastic biomarker by the FDA. This allows clinical researchers to study patients more likely to respond to treatments. This is critical because PKD kidneys get much larger than normal, a characteristic unique to PKD in contrast to other renal diseases. Our continuing work with KHI is vital to expanding use of the TKV biomarker earlier in the course of PKD that will serve as an indicator of the success of early therapeutic interventions. The time is ripe for breakthrough therapies. We hope the results of this collaboration will lead to understanding of the unique needs of PKD patients, and encourage pharmaceutical companies to develop new therapies. n 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.

11 PKD National Convention JUNE 24 TO 26 DISNEY S CORONADO SPRINGS RESORT AWARENESS This summer, the PKD community will gather for three days of learning, building connections and sharing progress in PKD research. PKD patients and caregivers will hear from PKD experts on topics ranging from how to manage the disease, emotional health, dialysis, transplantation and more. Specific sessions have been developed: Teens and young adults will meet and connect with others their age who live with PKD. Experts will present information in age-appropriate conversations, while providing support and guidance on how teens and young adults can take an active role in their health care. Parents of children who have ARPKD and ADPKD will find an intimate setting to learn, connect and support one another. Pediatric PKD experts will discuss topics specific to children and teens who have PKD. Other highlights: Celebration dinner keynote speaker Shuvo Roy, M.D., will talk about his work on developing an artificial implantable kidney for end-stage renal disease patients. Saintsations cheerleader and Foundation supporter Kriste Lewis will be the emcee for the Convention. Grant recipients Dr. Dell, Dr. Parnell and Dr. Freedman (featured on pages 4-9) will be there to answer your questions. Be sure to meet them! See the full agenda and register at pkdcure.org/convention Tax law change opens new IRA donation options Shannon Clark, a retired Mississippi trial judge, and the PKD Foundation are both benefiting from a tax law that President Obama recently reinstated, allowing donors to make tax-free gifts from their IRAs. You may now move up to $100,000 from your IRA directly to a qualified charity like the PKD Foundation without having to pay income taxes on it. This applies for all of 2015 and remains in effect for 2016 and beyond. Shannon took advantage of this by making a $10,000 IRA rollover donation to the PKD Foundation ($5,000 in 2015 and $5,000 for the 2016 tax year). His late wife, Kathryn, had a transplant and was on dialysis for many years due to PKD, and one of their three sons has PKD. Shannon believes his support will make an impact. Our family knows the problems with PKD first-hand. In my mind, one of the surest methods to find a cure or slow the progression of PKD is by financially supporting the research programs funded by the Foundation. If you have already made a qualifying gift in 2015, contact your advisor to make sure your gift is properly reported on your 2015 income tax returns. To learn more, Jackie Hancock, Jr.,CEO, jackieh@pkdcure.org. n In essence, the law states: If you are 70½ or older, you can give up to $100,000 directly from your IRA to charities such as the PKD Foundation. The transfer generates neither taxable income nor a tax deduction, so you still benefit even if you do not itemize your tax deductions. If you have not taken your required minimum distribution for the year, your IRA charitable rollover gift can satisfy all or part of that requirement. The transfer may be made in addition to any other charitable giving you have planned. 11

12 ORLANDO JUNE 24 TO 26, 2016 DISNEY S CORONADO SPRINGS Resort, Lake Buena Vista, Florida Why attend the PKD National Convention: n Learn how to manage PKD through all stages of the disease from diagnosis to life after a transplant. n Meet, connect and share experiences with others in the PKD community. n Hear the latest in research from PKD experts. n Choose from more than 30 sessions, including sessions for teens and young adults who have PKD, parents of children with ARPKD and ADPKD and caregivers. n Have a blast at the Walt Disney World Theme Parks. Discounted tickets are available with registration. SEE THE FULL AGENDA AND REGISTER AT pkdcure.org/convention 8330 Ward Parkway, Suite 510 Kansas City, Missouri pkdcure.org pkdcure@pkdcure.org NONPROFIT ORG U S POSTAGE PAID KANSAS CITY, MO PERMIT NO Please send your contact information updates or notify us of duplicate mailings of this publication by contacting donate@pkdcure.org or PKD.CURE ( ), ext The mission of the PKD Foundation is to promote programs of research, advocacy, education, support and awareness in order to discover treatments and a cure for polycystic kidney disease and improve the lives of all it affects. A cure is our finish line Every year thousands of people walk to end PKD in more than 50 cities nationwide. Be one of the 10,000 walkers to help fund research toward therapies and a cure, and provide education and support to the hundreds of thousands of families affected by PKD. REGISTRATION OPENS MAY 3 Visit walkforpkd.org to register, start a team, and learn how you can be the difference.