2 INTRODUCING THE PKD FOUNDATION’S NEW CHIEF SCIENTIFIC OFFICER

4 MAYO CLINIC’S ADPKD MUTATION DATABASE PROVIDES INVALUABLE INFORMATION ABOUT PKD

8 JINARC™ (TOLVAPTAN) APPROVED IN CANADA AND RECOMMENDED IN EUROPE

9 ST. AUBINS REMEMBER THEIR DAUGHTER THROUGH HELPING OTHERS

Photo Credit: By permission of Mayo Foundation for Medical Education and Research. All rights reserved.

ProgressPKD

A magazine to inform and educate PKD patients and families, Foundation supporters, health professionals and researchers.

Spring 2015

David Baron, Ph.D., PKD Foundation’s Chief Scientific Officer: Coming to the PKD Foundation is the apex of my career

Coming to lead the research work for the PKD Foundation is more than a job for me - it is a calling. Since I was a child, I have had a passion for science. Having the opportunity to use my skills and expertise to change the course of PKD for all affected families is without doubt the apex of my career.

I know about the impact of PKD firsthand. I received a kidney transplant as a result of PKD in 2009, and several people in my extended family also have the disease. So, I understand the urgency to find treatments, and eventually a cure, to ease the pain of those who are affected by this devastating disease.

I chose to come to the PKD Foundation out of retirement from Takeda Global Research and Development (Deerfield, Ill.) in 2013. I was with Takeda since 2001, where I started as their first U.S. Director of Toxicology and most recently was Vice President, Nonclinical Safety and Risk Evaluation for the U.S. and European Union.

Before that, I was the Senior Science Fellow for Global Toxicology with Pharmacia (Skokie, Ill.), and held increasing positions of responsibility with Searle/Monsanto (Skokie), becoming a Monsanto Science Fellow in Metabolism and Safety Evaluation. I also represented Nonclinical Safety Assessment on drug development teams. During my career in the pharmaceutical industry, I participated in numerous face-to-face discussions and negotiations with the U.S. Food and Drug Administration and European regulatory agencies. I learned what it takes to bring a drug from research through development and onto the market.

Earlier in my career, I was on the faculty of the Medical University of South Carolina (MUSC) in Charleston, S.C. While there, I gained experience working with the National Institutes of Health (NIH). With an NIH grant, I founded the Core Structure-Function Laboratory in the Department of Pharmacology at MUSC.

Throughout my career I have maintained a key interest in the structural correlates of movement of salt and water through and between cells. This movement is one of the main mechanisms by which the kidney goes about its business. The disruption of these mechanisms plays an important role in PKD.

I earned a bachelor’s degree in biology and Ph.D. in anatomy from The University of Chicago. I did my postdoctoral fellowship in pathology and pharmacology at MUSC, where I was also a Drug Science Foundation Fellow. I have been a grant reviewer for the National Cancer Institute, given numerous invited seminars and served on several national scientific boards. I have also been an expert witness in the areas of toxicology and pharmacology.

My wife and I have two grown sons, one in Brooklyn, the other in Chicago. We are avid worldwide travelers and lovers of classical music and the arts. Our travels have helped to improve my photography, and I can still manage a few tricks with a Frisbee.

I believe all of these personal and career experiences have led me to the PKD Foundation to benefit the PKD community. I am dedicated to using the skills I have cultivated to help the Foundation keep making progress toward ending PKD, and changing the narrative for future generations. For me, this means taking end-stage renal disease out of the equation for those with PKD and laying the groundwork for a targeted cure. I look forward to the work ahead!

Sincerely,

David Baron, Ph.D.

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Richard Nelson, a PKD Foundation Board of Trustees member, was diagnosed 28 years ago with PKD at age 38. PKD has had a significant impact on his family, including Richard and his three siblings. All four have gone into end-stage renal failure and have had transplants. His surgeon father died of PKD at the age of 43.

Unfortunately, his sister has been on dialysis for 17 years. She is a hero to Richard, as she strives to find positive ways to always find the silver lining for all those around her and especially herself.

In 1991, Richard received a transplant from a 17-year-old girl who died of a brain aneurysm.

“When I woke up from the transplant, I felt different,” he said. “I was profoundly grateful to the donor family and wanted to do something to honor them. I was given the gift of life and energy, and it changed my life’s priorities to be a part of the solution to finding treatments and cure for PKD.”

Nelson Family does their part to help PKD community

PKD Foundation board member Richard Nelson shares his 20-year journey with the Mayo Clinic.

This decision began a 20-year journey with the Mayo Clinic studying the large Nelson family. Richard met Vicente Torres, M.D., Ph.D., and Peter Harris, Ph.D., with the Mayo Clinic, at the PKD National Convention in Scottsdale, Ariz.

“The Mayo Clinic is beyond cutting edge in the work they do,” he said. “My oldest brother Steve, the family patriarch, had challenged us to be a part of the solution. I spoke with Dr. Torres about my family’s experience. Our experience is not typical of PKD, as we have a mutant gene finally diagnosed last year as the MUC1 gene -- a different form of PKD, medullary cystic kidney disease (MCKD). From there, my family has actively worked with the Mayo Clinic to help move PKD research forward.” (Read more about the Mayo Clinic on pages 4-6.)

Through providing DNA information to the Mayo Clinic and work with collaborators most recently at the Broad Institute, the form of MCKD has been identified. Providing this information also helps the Mayo Clinic and the PKD community better understand this rare form of cystic kidney disease. Dr. Peter Harris was the key for the Nelson family to become engaged and willing to be studied and eventually discover their mutant gene via the Broad Institute.

The next generation of the Nelson family is now participating in a study with the Mayo Clinic and the Broad Institute to better diagnose MCKD. Richard has five children, and they have 16 cousins ranging in age from 18-45.

“It is an ugly disease with a dramatic impact on my family,” he said. “I have

two grandchildren in Boston, and I feel strongly about improving things for the next generation. Participating in this study with Mayo and Broad researchers, along with many others, allows us to be part of the solution.

“With my successful transplant, I have literally been given the gift of life! I’m confident there is an answer to finding treatments and eventual cure for PKD. With the sense of urgency we feel, it’s surprising what a small group or even one person can do to make a difference.” n

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Mayo Clinic’s ADPKD Mutation Database records and provides invaluable information about PKD

Through the PKD Foundation’s Core Research Grants Program, funding of research facilities, databases and services is leveraged to benefit the entire PKD research community, helping to advance PKD research.

Director Peter Harris, Ph.D., analyzes trends in PKD and the common effects caused by specfic mutations.

About the ADPKD Mutation Database The PKD Foundation is the primary funder of the ADPKD Mutation Database (PKDB) at the Mayo Clinic. The PKDB was established by Director Peter Harris, Ph.D., to characterize variants (versions that differ from the standard) in the PKD1 and PKD2 genes, the two genes that cause autosomal dominant polycystic kidney disease (ADPKD).

While ADPKD is one of the most common, genetic diseases, the severity of the disease among people with PKD varies greatly. The database has been set up to collect variants in these two genes. The type of data stored in the

PKDB includes the mutation (change in DNA sequence) type, where the mutation is found in the individual’s genetic code and the frequency with which the mutation occurs. An affected subject can have one of several mutation types to the PKD1 and PKD2 genes. Some of these mutation types are associated with a more severe form of the disease, as measured by the probability of dialysis, transplantation or death at specific ages.

The gene mutation information in the PKDB is collected and organized from scientific publications involving ADPKD patients, as well as from clinical mutation

Photo credit: By permission of Mayo Foundation for Medical Education and Research. All rights reserved.

Mutations in PKD1 and PKD2 genes cause PKD

data. The database is the most comprehensive listing of mutations and other variants to the PKD1 and PKD2 genes. To date, the PKDB includes 2,322 variants for PKD1 and 278 variants for PKD2. Of these, 1,272 PKD1 variants found in 1,874 families are pathogenic (disease-causing) mutations. Corresponding figures for PKD2 are 202 mutations found in 438 families.

How does the information in the PKDB make a difference?Having a central location for ADPKD genetic information allows researchers and clinicians to examine the disease on a population level and over a long period of time. Mutations detected over the past approximately 20 years and from all over the globe are currently contained in the PKDB, rather than only on an individual level. Access to this amount of information allows analyses of trends in the disease, as well as the common effects caused by specific mutations.

“The large dataset has allowed for the first-time analysis of the mutational pattern of both ADPKD genes,” Dr. Harris said. “This type of information is extremely helpful for researchers and clinicians. There is currently about a 50 percent chance a mutation found in a new screening has ever been described, so the more we collect information about mutations, the more we can learn about the disease.”

The database contains information on pathogenic mutations and silent changes. With thousands of individuals (de-identified to protect anonymity) in the database, it is easy to sort the information to analyze which mutations are more common, how harmful they are and where they are found in the

The kidney, like the rest of the body, is made up of millions of cells. Each cell has a nucleus which houses the genetic material, called DNA. Most of the time DNA is wound tightly into structures called chromosomes. There are 23 pairs of chromosomes in each cell nucleus, with half inherited from the mother and half from the father. ADPKD is caused by mutations on either chromosome 4 or chromosome 16, as pictured on the right.

A gene is a length of DNA which codes for a single cell product or function. A mutation occurs when the wrong nucleotides occur in a gene. About 80 percent of people affected with ADPKD have a mutation in the PKD1 gene (chromosome 16). The rest of the ADPKD population has a mutation in the PKD2 gene (chromosome 4).

The PKD1 gene encodes the protein polycystin-1 while PKD2 encodes polycystin-2. When these proteins function normally, they are thought to play a role in kidney development and in the maintenance of normal renal structure. The severity of the kidney disease caused by the PKD1 and PKD2 genes is slightly different. With the PKD1 gene, cyst development, the onset of high blood pressure, and loss of kidney function appear earlier as compared to the PKD2 gene, although, there is some overlap, including some milder PKD1 mutations.

Gene

DNA

Chromosomes (number 4)

Cell

Nucleus

Nucleotides

Product or function of cell

Defective product or function of cell

The kidney, like the rest of the body, is made up of millions of cells. Each cell has a nucleus which houses the genetic material, called DNA. Most of the time DNA in wound tightly into structures called chromosomes. There are 23 pairs of chromosomes in each cell nucleus with half inherited from the mother and half from the father. One pair, X and Y, are referred to as the sex chromosomes becausethey contain genetic information speci�cally for “maleness” or “femaleness.”

DNA is described as a “double helix,” since its structure is like a ladder twistedlength-wise over and over. This incredibly long and thin molecule contains the genetic code for what makes you, “you.” The “rungs” of the “ladder” are made from one of four nucleotides, and the sequence of these nucleotides create the “recipe”for a cell product or function, much like how Morse code transmits language.

A gene is a length of DNA which codes for a single cell product orfunction. For instance, the kidney produced protein Renin, which helps regulate blood pressure, is made from the recipe contained in its gene, comprised of 20,000 pairs of nucleotides.

A mutation occurs when the wrong nucleotidesoccur in a gene. This changes the recipe for the product of that gene, leading to cell malfunction. About 80-85% of people with ADPKD have a mutation in the ADPKD gene located on chromosome 16, called ADPKD1. The remaining ADPKD population have a mutation in the ADPKD gene located on chromosome 4, called ADPKD2 (See above).

1 2 3 4 5 6

7 8 9 10 11 12

13 14 15 16

19 20 21 22 X Y

17 18ADPKD1

ADPKD2

Note: The color of thechromosomes as shownis false, and is due to the staining process usedto make the chromosomesmore visible.

Version 3.0 of the database launched in March 2014. The new version increased the number of variants, including from several large studies. It increased the number of newly described mutations by approximately 20 percent and the total number of records by approximately 50 percent.

5

Continued on page 6

66

genetic code. With this information, Dr. Harris can examine the extent a variant is harmful or neutral.

“The value of the PKDB information for clinicians is seen especially when the same mutation comes up in another patient, and they can see if it is likely pathogenic,” Dr. Harris said. “For those conducting clinical testing, they can use it to evaluate the significance of the variant. It is also helpful for those conducting clinical trials to help select a population for the study. Additionally, helping researchers study the disease could potentially lead to projects that target PKD on a genetic level, which can inform treatment options.”

PKDB and the PKD CommunityThe PKDB is available by visiting pkdb.mayo.edu and it is open to the public. The PKDB can be used to see if the mutation a PKD patient has is described and the likelihood of it being pathogenic. However, patients are advised to get professional advice regarding variants found in their sequence.

Brock Nelson, a PKD Foundation Board of Trustees member, provided his information for the PKDB through a blood draw and a questionnaire after being asked to participate by the Mayo Clinic. Brock has a spontaneous mutation, meaning he has no family history of PKD.

Through the process with the Mayo Clinic, he learned his PKD was caused by the PKD1 gene. It is significant for a patient to know if they have PKD1 or PKD2, as PKD2 can be milder with later end-stage renal disease. Recently researchers have discovered that approximately

10 percent of PKD1 mutations are also associated with milder disease outcomes.

“Providing my genetic information provides invaluable information for PKD researchers,” Nelson said. “I can provide information that could lead to predicting outcomes and potentially developing a treatment that could help others, including my children.”

To further his research, Dr. Harris is interested in hearing from PKD patients with unusual presentations of PKD, such as early onset PKD, late onset PKD or severe extra-renal complications. For more information, please contact Dr. Harris at harris.peter@mayo.edu. The profile on page 3 of Richard Nelson provides more information about participating in a study with the Mayo Clinic.

Next Steps Dr. Harris hopes to merge the PKDB with the PKD Outcomes Consortium Database (PKDOC). PKDOC is a significant collaboration between the PKD Foundation, Critical Path Institute, representatives of the pharmaceutical industry, PKD clinicians and the U.S. Food and Drug Administration (FDA). PKDOC was created to facilitate clinical trial development for PKD therapies by establishing a clear regulatory pathway for the pharmaceutical industry to evaluate the effectiveness of potential treatments.

Merging the two databases would tie together clinical and genetic information, providing better tools to predict the significance of mutations. n

representing 34 countries and

40 states.

“The information in the PKDB could eventually help clinicians target the timing and type of interventions if they know the type of mutation their ADPKD patients have,” Dr. Harris said. “This could lead to physicians potentially tailoring treatments to individuals based on their specific type of mutation; they may be able to know at what age to start treatment, what type of medications will work best, and how quickly or slowly the disease will progress. Access to large amounts of data, like that contained in the PKDB, is the best way to make these types of discoveries.”

Thanks to the PKD Foundation’s support, information is available to researchers and clinicians around the world

at no cost. In 2014, the PKDB had:

6,457 visits

from 1,919 unique visitors

Continued from page 5

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The Lillian Jean Kaplan International Prize for the Advancement in the Understanding of Polycystic Kidney Disease (PKD), created in 2002, recognizes a medical professional or researcher exhibiting excellence and leadership in PKD clinical or basic research. The long-term goal of the prize is to motivate members of the scientific and medical community throughout the world to increase, or to begin, research that will lead to a treatment and cure for people who suffer from PKD. It also aims to generate momentum in the field as well as public awareness about the disease.

The award was established by the PKD Foundation and the International Society of Nephrology (ISN) through the generosity of Thomas S. Kaplan and Daphne Recanati Kaplan of New York, in memory of Tom’s mother, Lillian Jean Kaplan, who was a PKD sufferer and died in 2002. The award is funded by the Recanati-Kaplan Foundation.

The 2015 recipients are Peter Igarashi, M.D., and Gregory J. Pazour, Ph.D. The doctors received their prize in person at the World Congress of Nephrology in Cape Town.

2015 Lillian Jean Kaplan International Prize for Advancement in the Understanding of PKD awarded

Peter Igarashi, M.D.Dr. Igarashi is the Nesbitt Chair and Head of the Department of Medicine at the University of Minnesota Medical School. In his current position, Dr. Igarashi oversees the largest department in the medical school with clinical, teaching and research programs.

Dr. Igarashi has 30 years of research experience in kidney development, stem cells and PKD. His laboratory has identified new proteins that control genes and characterized their roles in cystic kidney disease. In addition, Dr. Igarashi has studied the role of the primary cilium in the progression of PKD. His group was the first to prospectively demonstrate that the loss of the primary cilium was sufficient to produce kidney cysts. Recent studies from his laboratory have revealed that non-protein coding genes play a role in PKD and represent potential therapeutic targets.

He received his bachelor’s degree from the University of California, Riverside, and his M.D. degree from the University of California, Los Angeles School of Medicine. He completed residency training in internal medicine at the University of California, Davis Medical Center and a nephrology fellowship at the Yale University School of Medicine. Dr. Igarashi served on the faculty at Yale for 12 years before moving to the University of Texas Southwestern Medical Center, where he also directed the University of Texas Southwestern O’Brien Kidney Research Core Center.

Gregory J. Pazour, Ph.D.Dr. Pazour is a Professor of Molecular Medicine at the University of Massachusetts Medical School where he is working to understand the function of the primary cilium in mammal development and disease.

Building from studies in green alga, Dr. Pazour

with colleagues Joel Rosenbaum, George Witman and Doug Cole were the first to demonstrate that loss of primary cilia caused PKD. He also demonstrated that polycystin-2, the protein encoded by the human PKD2 gene, is localized to cilia. These studies laid the groundwork for the current appreciation of the role of cilia in PKD and helped establish that the primary cilium is a sensory organelle.

Dr. Pazour earned bachelors’ degrees in chemistry and biology from South Dakota State University and a Ph.D. in biochemistry from the University of Minnesota. He did postdoctoral work at the Worcester Foundation for Biomedical Research. Dr. Pazour has published more than 85 research articles and edited three books on cilia. n

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Two key developments took place in February that show progress in access to tolvaptan as an approved drug for adults living with PKD. In Canada,

JINARC™ (tolvaptan) was approved for patients with availability targeted for summer 2015. In Europe, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended JINARC™ for approval to the European Commission (EC). This is an intermediary step in JINARC’s path to patient access. The CHMP opinion will be sent to the EC for the adoption of a decision on EU-wide marketing authorization.

JINARC™ was discovered in Japan by Otsuka Pharmaceutical and was first approved there as a drug therapy for ADPKD in 2014. The Health Canada approval of JINARC,™ and the EMA recommendation, are based on the results of the pivotal Phase 3 randomized, double-blind and placebo-controlled TEMPO 3:4 Trial, the largest study conducted to date in adults with ADPKD.

In the United States, in late August of 2013, Otsuka received a Complete Response Letter (CRL) from the FDA regarding tolvaptan to treat adult patients with ADPKD. The FDA issues CRLs to convey that their initial review of a new drug application is complete and they cannot approve it in its present form without additional information. In June 2014, Otsuka began patient enrollment for a new Phase 3b study after the company reached an agreement with the FDA on a Special Protocol Assessment (SPA) for the design and planned analysis of this trial. This Phase 3b trial is being conducted to provide the additional information requested by the FDA.

Patient participation in clinical studies is critical in developing treatments. To learn how to get involved in the tolvaptan trial in the United States and other studies, visit pkdcure.org/progress/clinicalstudies.Read more about these developments and sign up to receive future announcements at pkdcure.org/news. n

What is PKD? Video is an easy way to spread the word about PKD

PKD affects thousands in the United States and millions worldwide – and yet, many people have never heard of it. The PKD Foundation raises awareness through marketing and public relations so people know what PKD is, about the Foundation and how they can help.

To make it easier to spread the word about PKD, the Foundation has created a What is PKD? video. At three minutes long, it provides basic facts about what PKD is, how it impacts people and how the PKD Foundation helps.

The video is meant for people unfamiliar with PKD, either newly diagnosed or those who have never heard of it. By sharing this with your family, friends, and even co-workers, they’ll have a better understanding of PKD. Visit pkdcure.org to watch and share the video. The more people who know about PKD, the closer we can get to treatments and a cure. Together, we can raise awareness and fight PKD. n

JINARC™ (tolvaptan) approved in Canada and recommended in Europe

The What is PKD? video is now available at pkdcure.org.

PKD Videos In addition to What is PKD?, there are many other videos about PKD on the Foundation’s YouTube page, including:• Participating in clinical studies• Recent webinars• First person stories about living with PKD

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The St. Aubins remember their daughter through helping others

At a 25-week ultrasound, Pete and Jen received the news. Their baby most likely had autosomal

recessive PKD (ARPKD). When Sophie was born, she fought hard to stay in the world long enough— 12 hours—for her parents to hold her, and tell her how much they loved her. They also promised Sophie that they would do whatever they could to help prevent other babies from suffering from such a horrible disease.

“In the midst of our immense sadness, we began receiving requests from people who wanted to help,” says Jen. “We asked our doctor and he recommended the PKD Foundation.”

The St. Aubins knew a little about the PKD Foundation already from researching ARPKD after Sophie’s initial diagnosis. After Pete conducted further research on the Foundation’s budget and outcomes, they decided to set up a tribute in Sophie’s memory through the PKD Foundation.

Pete and Jen St. Aubin have raised more than $60,000 for the PKD Foundation in tribute to their infant daughter, Sophie Marie.

ARPKD is a relatively rare form of PKD, affecting approximately one in 20,000 children. For ARPKD children who survive the newborn period (about 70 percent), approximately one-third will need dialysis or transplantation by age 10.

“We sent out an email,” Jen explained. “It told Sophie’s story and included a photo of her.”

“It meant a lot to us to include Sophie’s picture. It was comforting for people to see what a pretty baby she was and that she lived a life.”

Pete and Jen had no idea what an effect their email would have and how much money it would raise to help other families like them. Since September of last year, Sophie’s tribute has raised more than $60,000.

“We knew nothing about the disease until our doctor first said it,” Pete says. “We feel strongly that we want to spread awareness of ARPKD and the impact it not only has on the affected child but on the entire family. Sophie was our first child and now we know that any siblings of Sophie would have a 25 percent chance of inheriting the disease.”

Both Pete and Jen express that raising money was therapeutic for them and a helpful part of their grieving process.

“It was so moving to see how many people cared,” Jen says. “So heartwarming to see what an impact Sophie had.”

Pete shared his amazement at the overwhelming generosity of friends, family and co-workers.

“Through our tears at our loss of Sophie, we were also feeling tears of emotion for people giving so generously,” he says.

Pete says they plan to stay involved with the PKD Foundation.

“Overall, we just want to help each other—help other people and families,” says Jen.

You can find out more about making tribute gifts to the PKD Foundation by visiting pkdcure.org/tribute or calling 1.800.PKD.CURE (753.2873), ext. 187. Tribute gifts are a special way to celebrate the life of a loved one or mark a milestone like a transplant anniversary or birthday. n

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Not Your Average Garage Sale“Don’t be afraid to try something—just throw it out there and get creative,” said Christine Massey. She encourages people to go for it when it comes to

Do It Yourself (DIY) for PKD. She has garage sales, bake sales, Super Bowl pools, DVD drives and runs in marathons to raise money for PKD research.

“I started the garage sale four years ago,” she said. “I invite people to donate items and it’s a win-win for everyone.

“They get rid of things they no longer want or need while supporting a great cause. I raised more than $14,000 with all of my fundraising initiatives combined in 2014.”

Christine also has an online garage sale building up to her annual sale in September. She said her online efforts brought in $3,000 before the actual event took place.

“My mother had PKD and my sister and I both have it,” she says. “I’m just one person, but I do what I can do.

“I feel like I’m making a difference for my children’s future.”

Find out how to start your own DIY for PKD fundraiser at pkdcure.org/diyforpkd. n

Walk Day is Family DayLauren Maughan didn’t even know what a nephrologist was when she was diagnosed with PKD during college eight years ago. In 2014, she decided to Walk for PKD and get her family, friends and co-workers involved.

“I decided I was going to commit to it,” she says. “I want to help others and I know that a significant portion of the PKD Foundation’s budget comes from the Walks.”

And commit she did. Lauren’s Walk team, Scoop’s Troops, raised more than $12,000.

“I’m so humbled by everyone’s support,” she continued. “We made it a family day and people traveled to be part of it. We had a BBQ at my house afterward.”

Lauren appreciates the opportunities the Walk for PKD provides to share her story with family and friends and meet other people going through similar experiences.

She’s also grateful for the overwhelming support of her co-workers and her employer that made matching gifts available in support of her Walk team.

Find out how to start your own Walk for PKD team at walkforpkd.org. n

PKD Foundation peer-to-peer programs let you put a personal spin on your fundraising activities

There is no shortage of ways to help end PKD with the PKD Foundation. Take a look at how these three adventurous fundraisers put a personal stamp on their money-raising efforts and get inspired to take on your own project.

Christine Massey is making a difference through DIY for PKD.

Lauren Maughan’s Scoop’s Troops team raised more than $12,000 through the Walk for PKD.

Megan Moses runs in honor of her father, John Burge

Running for Dad“My dad always encouraged me to get into running. After his transplant in 2009, and seeing how he recovered and took charge of his health, I thought ‘There’s no reason I can’t run,’” shared Megan Moses.

She said as soon as she crossed the finish line in her first half marathon she knew she wanted to run a full marathon. Last year, she ran the Bank of America Chicago Marathon in honor of her dad, John Burge, who also happens to be the Chapter Coordinator for the Iowa Chapter.

“I raised more than $1,500 with my run,” said Megan. “It felt amazing.”

Megan also raised money for the PKD Foundation by donating the proceeds from the “dollar dance” at her recent wedding, hosting vendor parties at her house and putting a collection jar out at her job.

“There are so many ways to fundraise,” she explained. “It’s a great way to raise awareness about PKD and share your story.”

Get involved in the PKD Foundation’s Your Local Race Program and be on the lookout for how to participate in your local community’s 4th of July-themed race through Stars and Stripes for PKD. You can learn more at runforpkd.org. n

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Board of Trustees (2014-2015 term)Frank C. Condella, Jr. (Chair)Benjamin D. Cowley, Jr., M.D. (Vice Chair)Blaise Hazelwood (Treasurer)Klee Kleber (Secretary)Paul T. ConwayLisa Guay-Woodford, M.D.Michele KarlBrock NelsonRichard R. NelsonDwight OdlandJulia RobertsJeffrey RonaAnne RyanFran ToweyTerry Watnick, M.D.Hilary Wolfe

Scientific Advisory CommitteeTerry Watnick, M.D. (Chair)Stefan Somlo, M.D.

(Vice Chair)John Bissler, M.D.Alessandra Boletta, Ph.D.Benjamin D. Cowley, Jr., M.D.

Iain Drummond, Ph.D.Michal Mrug, M.D.York Pei, M.D.Ronald Perrone, M.D.Richard Sandford, Ph.D., FRCPDarren Wallace, Ph.D.Angela Wandinger-Ness, Ph.D.

Leadership TeamJackie D. Hancock, Jr., Chief Executive Officer David Baron, Ph.D., Chief Scientific OfficerAngela Connelly, Chief Marketing OfficerMichelle Davis-Wingate, Chief Development OfficerRay Smith, CPA, Chief Financial Officer

pkdcure.orgpkdcure@pkdcure.org 816.931.26001.800.PKD.CUREFAX 816.931.8655

facebook.com/pkdfoundation twitter.com/pkdfoundation

1.800.562.3974 www.otsuka-us.comOtsuka America Pharmaceutical, Inc.Otsuka Pharmaceutical Development & Commercialization, Inc.Otsuka Maryland Medicinal Laboratories, Inc.

© 2015 Otsuka America Pharmaceutical, Inc.

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.

People creating new products for better health worldwide

The name “Otsuka” translates to

“major milestone.”

Information gained from the HALT-PKD trials is critical to understanding PKD.

Results of the HALT-PKD clinical trials were presented at the American Society of Nephrology (ASN) Kidney Week in November 2014. The studies found that

rigorous blood pressure control, early in the disease, could slow cyst growth in ADPKD. In addition, only one type of medication (ACE inhibitors) is necessary to treat hypertension.

The HALT studies are important to the PKD community because the optimal blood pressure target levels, age at which to begin controlling blood pressure or the best medications for control of blood pressure in ADPKD were not known when the study was started in 2006 (the study was completed in June 2014). Currently, the complications of hypertension, including stroke and heart attack, affect many more people with ADPKD than ADPKD-specific complications such as liver cysts or brain aneurysms.

The information gained from the HALT-PKD trials is invaluable in understanding PKD and identifying potential ways to slow its progression. The PKD Foundation is proud that we were one of the funders of the HALT studies, along with the NIH-National Institute of Diabetes & Kidney Diseases (NIDDK).

To learn more about the HALT studies, including abstracts and articles from the New England Journal of Medicine, please visit pkdcure.org/halt. n

HALT-PKD Studies find rigorous blood-pressure control could slow cyst growth in ADPKD

Please send your contact information updates or notify us of duplicate mailings of this publication by contacting donate@pkdcure.org or 1.800.PKD.CURE (753.2873), ext. 187.

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 forpolycystic kidney disease and improve the lives of all it affects.

8330 Ward Parkway, Suite 510Kansas City, Missouri 64114

PKD Patient Handbook coming soonThe PKD Patient Handbook is an education resource for PKD patients and anyone impacted by PKD. It will soon be available to download for free at pkdcure.org. Or, call 1.800.PKD.CURE (1.800.753.2873) to be sent an electronic or print copy.

Produced by the PKD Foundation, and reviewed by PKD physicians and patients, the handbook has current information and illustrations about signs and symptoms of PKD, common tests, genetics and the science of PKD. The focus is on ADPKD, but a handbook on ARPKD is to come.

The PKD Patient Handbook was made possible by an education grant from Otsuka America Pharmaceutical, Inc.

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PKD NATIONAL CONVENTION SUMMER 2016 | ORLANDO

PKD PATIENTHANDBOOKUnderstanding and Living with Autosomal Dominant Polycystic Kidney Disease

SAVE THE DATE

See the promo video and recap of the 2014 Convention at pkdcure.org/convention.