Spring/Summer 2013 Vol. 28, Issue 4
Progress PKD
A magazine for PKD patients, Foundation supporters, health professionals and researchers.
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Message from the CEO: tolvaptan and Drug Repurposing Momentum
Report: 4 Research Core Labs Helping ARPKD 8 Families a Champion: 10 BeUnited on the Hill Unite to Fight 12 and Walk for PKD
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M e s s age
Unite to Fight and Rewrite the Story of PKD for Future Generations
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have been thinking a lot about the power of telling the PKD story. What has struck me the most is how people affected by PKD are connected to the Foundation, and how they have prevailed through challenging circumstances. Their stories have moved me in a profound way, and you’ll read some of them in this issue of Progress. You will notice we’ve redesigned Progress to tell the story better, through first person accounts, and describing the results of work we’re doing. In this issue we focus on the Core Research Grants Program. Read more about telling the PKD story and renewing your Progress subscription on page 15. The PKD Foundation has its own story since being founded in 1982. It has taken 30 years of hard work, research and many dollars from supporters like you to get to today’s point of rapid acceleration toward finding treatments. I’m particularly excited about recent positive outcomes as a result of the Foundation’s work.
We must continue this momentum and bring more treatments to the forefront. I hope you saw the announcement by Otsuka Pharmaceutical Co. Ltd. that the U.S. Food and Drug Administration (FDA) accepted for priority review the company’s new drug application for the potential use of tolvaptan to treat ADPKD. Because of this priority status, tolvaptan should be reviewed by Sept. 1. If approved, this will become the first pharmaceutical therapy for patients with ADPKD. The Foundation is proud to have funded early preclinical animal testing of tolvaptan, the precursor to the human clinical trials that showed positive results in slowing the progression of PKD.
MSDC-0160, a prototype mTOT Modulator™ developed by Metabolic Solutions Development Company (MSDC), has been studied in non-PKD related human clinical trials (one involving patients with type 2 diabetes, and one with patients with dementia due to Alzheimer’s disease). It was found to be safe and well-tolerated. Next steps for MSDC-0160 to treat PKD are being evaluated by MSDC. Learn more about MSDC-0160 by visiting msdrx.com. We must continue this momentum and bring more treatments to the forefront. Because individuals will respond differently to treatments, it will require numerous therapies to address the current and future PKD patient population. We wouldn’t be where we are today without generous supporters like you. At the same time, we won’t be where we need to be tomorrow without your continued help. I hope you are moved enough by our story, and encouraged by our progress, to help us make a difference for generations to come. We are at a clear intersection, with what we know about the disease and a relentless pursuit to discover treatments. This mission requires financial resources. We cannot do this work without people coming together, uniting to fight PKD. This is why several generous donors have offered to match donations made between May 1 and June 30 (up to $75,000). Visit pkdcure.org/doublemygift to make your contribution go even further. Help us write the next chapter of the Foundation’s story, and that of future generations, by donating what is personally significant to you. Please join me in keeping the momentum going…toward ending PKD.
Warm regards, We’ve also done important work in the Drug Repurposing Program. In 2010 when we launched our Accelerating Treatments to Patients (ATP) Program, we set a goal to test 25 to 30 drug candidates over five years, with hopes of identifying one with the possibility of progressing to clinical study in humans. In three years we have tested five candidates, and found one with potential to move to clinical trials. This candidate, called MSDC-0160, showed the potential to reduce cyst size and volume.
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Gary G. Godsey President and CEO
Lumps and Bumps: One Family’s Remarkable Transplant Journey By Stephanie Bost, Guest Writer
My family’s transplant journey began 15 years ago with my grandmother Jean, who was just days away from beginning dialysis to combat the devastating renal failure caused by PKD. In 1997, she received a phone call that would change her life. Transplant doctors discovered that a perfectly matched kidney had been found and Jean received her gift of life that same day. For our family, which includes her husband, Harry, three children, two daughters-inlaw and four grandchildren, that day brought a huge sigh of relief, but our family’s transplant journey was just beginning. In 2010, Jean’s oldest son, Jeff, began to experience symptoms of renal failure and started the long process of getting on the kidney transplant list himself. Jeff and his siblings had known for many years that all three of them had the same disease as their mother. Jeff’s wife, Kathy, was tested to be a living donor for Jeff and amazingly was a close match. The transplant took place in 2011. However, recovery did not bring as big a sigh of relief this time. As Jeff and Kathy were in the hospital, Jeff’s younger brother, Bob, was also going through his testing to be put on the kidney transplant list. Just nine months after Jeff’s transplant, Bob received a kidney from his wife, Courtney, in 2012. The same surgeons performed the transplant on both husband and wife pairs. Jean, Jeff and Bob continue to have
regular blood tests to check that their new kidneys are functioning properly and they are all on extensive anti-rejection medication regimens. Our two living donors, Kathy and Courtney, are both doing very well post-operatively and we continue to be grateful for them, as well as the donor who gave this gift of life to Jean. Our family is truly blessed to have had three successful kidney transplants, but as we look towards the future, there are more hurdles ahead. Jean’s youngest daughter, Cheryl, also has PKD and is currently experiencing the symptoms of kidney failure. She has completed the last of her testing and was recently put on the transplant list. Unlike Jeff and Bob, Cheryl does not have a living donor at this point. She has a hard to match blood type, O-negative, which has prevented many of her close friends and willing donors from meeting matching requirements. As we continue to hope and pray for the health of this generation, the next generation is trying to be proactive about treatment options. Three of Jean’s four grandchildren have been diagnosed with the disease. Getting in on the early clinical trials of potential
The Bost Family wearing “Recycle Yourself ” organ donation sweaters as they celebrate Harry’s 80th birthday!
medications and seeing doctors early on about general health habits will hopefully give the next generation a better outlook. We have learned so much from each of our experiences dealing with this disease, but probably the most resonant is the need of a loving and caring support system. We have benefited from each other’s experience, either as a donor, a recipient or a child of parents with kidney failure. To learn more about kidney transplantation, please visit pkdcure.org/kidneylink. n
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R e se a rc h
research
Since 1982, the PKD Foundation has invested more than $31 million to fund more than 550 research, clinical and scientific grants, as well as fellowships and scientific meetings. These investments position the Foundation as the second largest funder of PKD research after the National Institutes of Health (NIH).
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Core Research Grants Program Benefits PKD Scientists and Promotes PKD Research by Supporting Core Labs
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he Accelerating Treatment to Patients (ATP) initiative is a comprehensive, integrated research and development program that represents the core of our work at the PKD Foundation. Clearly focused on the patient, ATP was launched in 2010 to speed up development of treatments which could slow or stop progression of PKD. One component of ATP, the Core Research Grants Program, is focused on providing grants to research facilities that support or provide services to PKD scientists at no cost (or greatly discounted) to facilitate research. This allows Foundation dollars to be leveraged across the PKD community and benefits the scientific community as a whole. The Foundation is the primary funder of each of these three labs, and $160,000 has been distributed in the past year among: • PKD Electron Microscopy Core for PKD Research led by Dr. Vincent Gattone, Indiana School of Medicine, Electron Microscopy Center • ADPKD Mutation Database (PKDB) led by Dr. Peter Harris, Mayo Clinic • PKD Research Biomaterials and Cellular Models Core led by Dr. Darren Wallace, University of Kansas Medical Center
PKD Electron Microscopy Core
Led by Dr. Vincent Gattone, Indiana School of Medicine, Electron Microscopy Center
Under the direction of Dr. Gattone, an experienced electron microscopist and PKD researcher, this lab performs electron microscopy (EM)* evaluations for researchers. Most hospitals have an electron microscopy lab, but patients are given priority, so it can be challenging for researchers to have timely access to the lab. Through the PKD Electron Microscopy Core Lab, researchers are given priority, and due to the support of the PKD Foundation, the evaluations are available at a 90 percent discount. The discounted rate allows more members of the PKD research community with access to a research EM facility to advance their PKD research program. In addition, Dr. Gattone provides advice and interprets findings. The PKD Foundation has provided funding to this lab since 2006, and funded $60,000 in 2012. The lab is dedicated to facilitating research on PKD (and other hepatorenal fibrocycstic diseases), and provides a broad range of services including transmission and scanning EM. EM is important in evaluating cell biological processes and can help scientists understand how PKD progresses. *An electron microscope (EM) uses an electron beam to illuminate a specimen and produce a magnified image. It has greater power than a light microscope and can reveal the structure of smaller objects. (Wikipedia)
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R e se a rc h “Our goal for the PKD Electron Microscopy Research Core is to help as many researchers as we can to advance their studies at a minimum cost,” said Dr. Gattone. “We hope that our services will help many in the PKD research community, especially in these times of tight research funding.” In 2012, the lab handled 16 projects from 13 researchers around the world. Five publications referenced the Electron Microscopy Core Lab in 2012, and additional manuscripts have been submitted for publication. In addition, poster presentations have been exhibited at the American Society of Nephrology (ASN) and the Federation of American Societies for Experimental Biology (FASEB) conferences.
ADPKD Mutation Database (PKDB)
Led by Dr. Peter Harris, Mayo Clinic
This Core Lab was established to characterize the ADPKD gene variants in PKD1 and PKD2, the two genes known to have mutations that cause PKD. The ADPKD Mutation Database (PKDB) displays the most comprehensive listing of all published PKD1 and PKD2 mutations. This information is available to PKD researchers around the world at no cost through support of the PKD Foundation. Although designed for research rather than clinical use, a doctor can see if a mutation (or other variant) identified by clinical mutation testing of PKD1 and PKD2 is described in the database to help them understand its significance. “The database is increasing awareness that not all mutations have an equal effect,” said Dr. Peter Harris. “There are instances where a PKD1 mutation may be less severe, and more similar to PKD2, which is information that we are starting to reflect in the database.” Because this is the sole database for ADPKD, there is a lot of interest and it is widely used. In 2012, the
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PKDB had 6,260 visits from 1,396 unique visitors representing 62 countries and 40 states. In the last two years, 25 articles were published referencing the PKDB. Version 2.9 of the database was launched in Nov. 2012 and included a complete redesign of the database architecture. The update increased security and stability and decreased webpage load time to only a few seconds, compared to more than a minute before the upgrade. Dr. Harris said they hope to add information on another 1,000 variants to the database in the near future, further increasing the information available. Additionally, the focus is on developing Version 3.0 of the database, which will have a redesigned user interface that will leverage new and existing algorithms and integrate a new code to maximize flexibility. The upgrade will also use smart searches, which will effectively make PKDB similar to a search engine for ADPKD mutations. “The grant is fundamental to keeping our database up to date,” said Dr. Harris. “Without it, we could not update the system or add new information, and we would not be able to maintain this level of accessibility.” The PKD Foundation began funding the PKDB in 2007, and in the past year provided $40,000 in funding.
PKD Research Biomaterials and Cellular Models Core
Led by Dr. Darren Wallace, University of Kansas Medical Center This Core Lab was created to promote PKD research by providing human PKD tissues and high-quality biological materials to investigators. PKD kidneys, retrieved at the time of removal (nephrectomy), are invaluable research specimens. The Core is designed to maximize the use of these rare cystic tissues to meet the research needs of multiple PKD investigators. The Core can provide a variety of biomaterials including fixed and frozen tissues, cyst fluid and primary cells cultured from cysts to investigators in a collaborative arrangement at no cost due to financial support by the Foundation. The funding for this core began in April 2012 through a $60,000 grant from the PKD Foundation. The advantage of studying human PKD kidney tissue and cells derived from the cysts is that the cells harbor the genetic mutations responsible for cyst formation in human disease. The Core also assists in the development
and use of PKD cell models to test specific hypotheses and to evaluate drug effects. In vitro models for PKD have been important for the investigation of key signaling pathways involved in cyst growth and have contributed to significant breakthroughs in PKD research that have advanced our understanding of this disease. “It is gratifying to know that the first therapeutic approaches to be tested in ADPKD patients emerged from studies using cells cultured from cystic kidneys donated by patients,” said Dr. Darren Wallace. The PKD Research Biomaterials and Cellular Models Core is the leading provider of human ADPKD research biomaterials in the U.S. Last year, there were 16 academic research laboratories worldwide that obtained biomaterials from the core, resulting in seven manuscripts submitted for publication. n •
Learn more about this Core Lab at kumc.edu/pkdbiomaterials.
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Learn about donating your polycystic kidney, including questions and answers by Dr. Wallace, at pkdcure.org/research/tissue-donation/faqs, or by calling the Foundation at 800.753.2873.
A Personal Story of Giving Back For work to be conducted at the Core Labs, it is important for researchers to have access to tissue. Because of this, there is a direct connection between the Core Research Grants Program and the Tissue Donation Program, both components of ATP. Researchers rely on people who have donated their polycystic kidneys to one of our participating tissue donation research labs. (Read more about the Tissue Donation Program online, pkdcure.org/research/acceleratingtreatments-to-patients). Frank Condella, incoming PKD Foundation Board Chair, understands how important this is. Frank was diagnosed with PKD in 2002. He is the first in his family to have the disease. In 2011, he received a kidney from a living donor, and donated his native polycystic kidneys to one of the research labs taking part in the Tissue Donation Program. Each donation has the capacity to reach many scientists and is very helpful for researchers to learn how PKD progresses through kidneys. Frank encourages others planning a removal of a polycystic kidney to consider donating it to research. “Tissue can help advance science and is very important to the overall effort to find treatments and a cure.” This is an important way for
people to actively participate and support basic science. He also sees this as an opportunity to educate physicians and surgeons who may not be familiar with the program or need for PKD tissue. For example, according to Frank, “Surgeons may think that the affected kidney needs to be removed intact, however large kidneys often need to be removed in sections and any portion can be of value and utilized by the lab. You need to be an advocate to your surgeon to make sure a removed kidney is directed to a researcher.” Frank has done much more than donate his kidneys–he has also given his time. Being in the pharmaceutical industry, he wanted to understand what was going on in PKD research and potential treatments. He became involved with the PKD Foundation and joined the Board of Trustees in 2009. While he felt there might not be a treatment for him, he believed he could improve hopes of a treatment for his three daughters should it be determined that they inherited the disease. As incoming Board Chair, he believes “there is so much exciting research going on at all different levels, whether at the basic research or clinical level. The Foundation has to be the driving force in finding treatments and a cure for this disease. Whether it’s through direct funding of research or supporting programs like tissue donation and advocacy–all of these efforts go to improving our chances of finding better ways to fight this disease.”
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Educ at ion
Providing Compassion, Connections and Education for Parents of Children with ARPKD
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relatively rare form of PKD called autosomal recessive polycystic kidney disease (ARPKD), affects approximately 1 in 20,000 children, and often causes death in the first month of life. For ARPKD children who survive the newborn period (about 70 percent), approximately one-third will need dialysis or transplantation by age 10. Previously thought to be fatal, the prognosis for children with ARPKD has improved dramatically. Twenty years ago, only half of the children born with the disease survived to their 10th birthday, but now that percentage has increased to 85 percent.
Sage Baldwin, who has ARPKD, cuts the ribbon with her parents at the Kansas City Walk for PKD.
Previously thought to be a fatal condition, the prognosis for children with ARPKD has improved dramatically. To help parents and families impacted by ARPKD*, the PKD Foundation provides resources and supports the virtual ARPKD Chapter. The Foundation has invested nearly $2 million since 2006. Support The virtual ARPKD Chapter, led by volunteers Julia Roberts and Michele Karl, supports families of children with ARPKD, and has served more than 500 families since 2000. • The Chapter provides one-onone support, with volunteers available 24 hours a day, seven days a week. • Families can be connected with Dr. Lisa Guay-Woodford (an ARPKD expert) and other providers, as well as other local families.
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The Chapter participates in the Walk for PKD. Members join the national team (walking on their own), or walk in a local Walk for PKD event to represent the ARPKD Chapter. In 2012 more than 1,000 people walked in these events, raising nearly $300,000. More than $1 million has been raised since 2006. Other local events raise funds for the Foundation to support ARPKD work, such as Bowen’s Hope to Fund a Cure Auction, Ella Grace Race, Buddies of Colton and Emslie’s Fight (Run for PKD team, see Page 9).
Resources • The ARPKD online learning center on the Foundation’s website (pkdcure.org/learn/
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arpkd) features numerous webinars and videos including What Every Family Should Know About ARPKD, Kids and Kidney Disease, and Kidney Transplants in Children. In April 2013, the PKD/CHF and Other Cystic Diseases: From Diagnosis to Daily Living Conference was held in New York City. This conference was for parents and caregivers dealing with ARPKD, advanced complications of ADPKD in young children, other cystic kidney diseases or CHF. *ARPKD-related efforts also benefit children with ADPKD and congenital hepatic fibrosis (CHF), a disease closely associated with ARPKD. n
Visit pkdcure.org to learn about ARPKD and download the ARPKD Report, or email ARPKDChapter@pkdcure.org to learn more.
Run for PKD
Help those affected by PKD, while getting fit
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teve Donboch was not always a runner. In fact, he used to claim he would only run if something was chasing him. Then, something happened in Steve’s life that changed his mind about running: his baby daughter, Emslie. “Emslie means ‘gift from God,’” said Steve. “It was a very fitting name for her.” His baby girl was diagnosed with ARPKD, a form of PKD that affects infants within their first month of life. When Emslie lost her life to the disease, Steve knew he needed to find a way to help other families like his. “Helping others helps me heal,” he said. The selfproclaimed non-runner decided to get involved with the Run for PKD.
their team name: Emslie’s Fight. They held raffles, raised money and educated people about PKD. Their fundraising surpassed their goal and raised more than $7,000 dollars. Then, ten days before the race, Steve’s training took a turn for the worst. His leg gave out during a run. The doctor told him he broke his fibula and couldn’t run the marathon. All Steve could think about was how he promised he would finish the race for Emslie.
Determined, he borrowed a wheelchair for the race, and miraculously finished the entire 26.2 miles by “The best part about Run for PKD is that people pushing himself in the can help others while simultaneously helping wheelchair. His inspiring themselves by becoming healthy,” said Steve. race, added with his teal hair, dyed in honor of the PKD Foundation, gave Steve There are two options for the Run for PKD—a marathon a chance to educate even more people about the disease. series with nationally sponsored PKD Foundation teams, or “Your Local Race,” which allows participants “The best part about Run for PKD is that people can help to fundraise for the PKD Foundation for any race they others while simultaneously helping themselves by choose. Steve chose the “Your Local Race” option, becoming healthy,” said Steve. “It changed my life, and I recruiting seven friends to join him for the 2012 am watching it change other peoples’ lives.” Savannah Rock and Roll Marathon. Then he purchased the book “Running a Marathon for Dummies,” and hit He encourages anyone to join—whether you are an the road. experienced runner who wants to support an important cause, or an inexperienced runner who has friends or Steve’s team found the most success by joining forces on family affected by PKD. Steve signed up for the 2013 their fundraising. Together they formed partnerships Savannah Rock and Roll Marathon again this year. He with local businesses, set up booths at restaurants and is looking forward to running this time, and as always, sold concessions at sporting events and concerts (where doing what he can to end PKD in Emslie’s honor. they were allowed to keep tips and a percentage of To get involved in Run for PKD, visit profits). They had t-shirts, car magnets and bracelets with pkdcure.org/runforpkd. n
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Advo c ac y
Be a PKD Champion and Make a Difference 6th Annual United on the Hill June 17-18
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ou may not feel like a Champion, but that’s exactly who you are. As someone with PKD, or a loved one or parent, you know more about PKD than anyone else. But what can you do to make a difference? Share your story and make your voice heard through United on the Hill or with local legislators year-round. United on the Hill is our annual legislative and public policy conference in Washington, D.C. After participating in training, we spend a day on Capitol Hill meeting with members of Congress, to raise awareness for PKD and discuss our legislative priorities for the year. Anyone affected by PKD is invited – no advocacy experience required. We’ll teach you how to connect with elected officials and become comfortable with meeting members of Congress and their staffs. We will provide a detailed overview of the PKD Foundation’s legislative priorities and a chance to hear from
Washington insiders. In the past, such speakers included: National Institute of Diabetes and Digestive and Kidney Diseases Director Dr. Robert Star; Former Sen. Robert Bennett (R-Utah); and Dr. Sally Satel from the American Enterprise Institute. We are working to bring equally compelling guest speakers to you this year. We encourage you to share your story with some of the nation’s most important leaders. United on the Hill is a critical part of the PKD Foundation’s efforts to secure funding for PKD research, and ultimately, treatments and a cure for PKD. For more information and to register, visit pkdcure.org/ advocate/united-on-the-hill How to Advocate in Your Local Area You don’t have to attend United on the Hill to make your voice heard. You can share your story locally year-round. This grassroots effort can make significant changes
through legislation and policies that improve the quality of life for everyone affected by PKD. We encourage people with PKD and their families to meet with their local congressional representatives because just one personal story and one relationship can make a significant difference. Additionally, members of Congress want and need to hear from you. Former Sen. Robert Bennett (R-Utah), a PKD Foundation Board of Trustees member, knows how important it is to connect with local representatives. “As a senator, I was always happy to hear from people from my home state. I received many requests for consideration, and hearing from people helped me set priorities. They informed me about what mattered to Utah – the state that I represented. It’s critical for people in the PKD community to vocalize why PKD-related legislation is important in their states. The efforts people put into advocacy truly does have an impact.”
Advocacy Toolbox If you are unsure of where to begin, pkdcure.org/advocate features an Advocacy Toolbox, containing valuable and detailed information about how to meet with officials and get your message across. In the Advocacy Toolbox, you can find information regarding the below and more. • Finding Your Elected Officials: Visit house.gov or senate.gov to find the local officials for your zip code • Communicating with Elected Officials: How to schedule a meeting, tips for visiting a congressional office, template thank you letters and talking points • Visiting Your Elected Officials: Introductions, discussion points, telling your story • Meeting Your Elected Officials: Detailed instructions on how to schedule a meeting with a member of Congress • Legislative Glossary: Terminology used in a political setting Visit the Advocacy Toolbox at pkdcure.org/advocate/advocacy-toolbox or email pkdadvocacy@pkdcure.org for more information.
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2013 Legislative Priorities The 2013 legislative priorities were selected to help further programs or legislation benefiting PKD patients and families, as well as others with kidney disease. This legislation we support will: • Improve the quality of life for people who suffer from kidney disease. • Speed up the development of new and better treatments for patients with chronic diseases and disabilities. • Allow a tax credit for the donation of a life-saving organ for transplantation. • Extend the current Medicare Secondary Payer policy for end-stage renal disease (ESRD) patients. • Preserve the charitable tax deduction. • Request PKD research funding from the Department of Defense. Visit pkdcure.org/advocate/legislation for details and timely updates. PKD Alliance The PKD Foundation Washington Alliance Forum was launched earlier this year to bring together partners with a goal of finding treatments and cures for chronic illnesses. Alliances are essential when it comes to presenting common messages, pursuing common goals and advocating policy changes, as they provide an increased bandwidth of support and knowledge when approaching decision makers and solving problems. The forum is designed to highlight the work of the Foundation and to provide a venue for engagement with partners, and to ultimately position the Foundation as a thought leader and convening organization in Washington. The forum will meet quarterly as part of ongoing strategic partnership development. As fellow stakeholders, the PKD Alliance members will play a powerful role in improving the lives of patients. The Alliance brings together leaders in the area of kidney disease, medical research, rare diseases, nursing,
bone health, weight reduction and healthy lifestyles and other collaborative activities. The diverse group will work to educate and advocate for polycystic kidney disease and other serious and rare conditions. PKD Alliance Partners • Aspen Health Stewardship Project • HOSA-Future Health Professionals • Healthy Weight Commitment Foundation • Biotechnology Industry Organization • National Bone Health Alliance For more information about our alliance partners, visit pkdcure.org/advocate/pkd-alliance n
“One of the most important ways those interested in putting an end to PKD can move to that goal is to let the local representatives and senators in federal government know their support of the National Institutes of Health (NIH) has brought great promise for the future, and how very important it is to keep the support level for PKD research high within the NIH. PKD research has just enjoyed a banner year. This marks the end of the beginning. Even greater progress is ahead as long as strong research support makes it possible.” - PKD Foundation Co-founder Jared Grantham, MD
The PKD Foundation Washington Alliance Forum was launched earlier this year to bring together partners with a shared goal of finding treatments and cures for chronic illnesses.
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s u p p or t
Boost Fundraising and Reach New Audiences Through A Challenge Grant
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ason and Amy Omenn wanted to go beyond writing a check to the PKD Foundation and find a way to bring in new donors. “We were looking for a way to pay it forward and turn our single donation into numerous donations for the PKD Foundation,” said Jason. “Our goal was to bring new people and new dollars to the cause.” Jason and Amy did just that by creating a challenge grant for the 2012 and 2013 Walks for PKD. Challenge grants aim to stimulate giving by providing matching funds for donations. Amy’s side of the family has PKD, and after she married Jason, she was diagnosed with PKD as well. Passionate advocates for the PKD Foundation, Jason and Amy were among the top fundraisers at the Seattle Walk for PKD for the past several years. Amy’s aunt, Marjorie Blanchard, is also very involved with the PKD Foundation and serves as the Chapter Coordinator for the San Diego Chapter. When Amy’s mom and Marjorie’s sister, Laura Myers, passed away from the disease in 2012, the family decided to establish a challenge grant to honor her life. The $15,000 per year, two-year grant was funded through the Omenn Family Fund, a fund of The Seattle Foundation. The challenge grant applied to both the Seattle and San Diego Walks for PKD. The Omenns’ challenge grant included three ways for participants to have their funds matched: 1. We Want You Back. If you participated in past Walk for PKD events, but didn’t participate in 2011, the grant matched fundraising efforts
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Amy and Jason Omenn created a challenge grant for the 2012 and 2013 Walks for PKD, aiming to stimulate giving by providing matching funds for donations.
if you raised at least $100 in 2012 (i.e. if you raised $500, it became a $1,000 donation). 2. Join Us. If you never participated in the Walk for PKD, but did so in 2012, your fundraising efforts would be matched 100 percent if you raised at least $100. 3. You Double It, We’ll Match it. 2011 Walk for PKD participants who doubled their past fundraising efforts during the 2012 Walk had the total funds raised matched 100 percent (if they raised at least $100).
“What I like about challenge grants is you can get people engaged with fundraising who aren’t on your team,” said Amy. “While it’s great to have competition, in the end we are all fighting for the same thing. So if we raise more money, that to me is a win!” It was a first-time participant of the Walk for PKD in Seattle, Kim Daly, who ended up becoming the top fundraiser in Seattle, making a huge impact when a portion of her team’s fundraising efforts were matched by the challenge grant. Kim has twin four-year old daughters; one of
In 2013, Jason and Amy’s challenge grant will apply once again to the Seattle and San Diego Walks. For them, the challenge grant is one more way they can help make progress toward a future without PKD.
them, Lexi, has PKD. Although Kim didn’t find out about the challenge grant until after most of her fundraising efforts were complete, she was proud of the impact the challenge grant provided to her team. “Imagine how great it will be to tell Lexi, when she is old enough to understand, that we were able to raise more than $12,000. And because of the challenge grant, we were able to give even more in her honor!” Because of passionate fundraisers like Kim, and funds matched by the challenge grant, the Seattle Walk raised $51,338 to support the PKD Foundation, a 58 percent increase over the 2011 Walk. The San Diego Walk raised $49,998, a 40 percent increase over the 2011 Walk. Challenge grants are rewarding and simple to create. You can establish one as an individual, or gather a group of people to pool money to donate to the PKD Foundation. Instead of donating the money directly, a challenge grant matches the fundraising efforts of others, creating an incentive for others to fundraise. As Kim puts it, “There is something so powerful about the multiplier effect of challenge grants.” If you are a Chapter Coordinator or Walk Coordinator with a challenge grant for your Walk, Marjorie’s advice is to communicate to participants early and clearly. “Communicating early helps inspire more participation,” she said. “For example, if your Walk is in October, I recommend starting to raise awareness about challenge grants in June or July. While email may seem like the easiest option, the more phone calls, the better. It gives people an opportunity to ask questions about how they can become engaged.” In 2013, Jason and Amy’s challenge grant will apply once again to the Seattle and San Diego Walks. For them, the challenge grant is one more way they can help make progress toward a future without PKD. “When I think about my commitment to finding a cure,” said Amy, “I think about my children, and making a difference for future generations.” If you have questions about starting a challenge grant for the PKD Foundation in your city, contact Michelle Davis, Chief Development Officer, at 800.753.2873 x 114 or michelled@pkdcure.org. n
Unite to Fight PKD!
You can lead the way to a better tomorrow for those affected by polycystic kidney disease. On May 1, registration opened for the annual Walk for PKD events occurring nationwide in the fall. Walk for PKD is the signature fundraising event for the PKD Foundation, and the largest gathering of PKD patients, family, friends and supporters; nearly 10,000 strong. More than 50 Walks occur every year in cities across the nation. Since PKD affects both children and adults, the Walk is a familyoriented event that honors and supports loved ones, creates a sense of community and provides hope for a better tomorrow. In 2012, the Walk for PKD raised $1.8 million to support the PKD Foundation’s mission. This year, we are closer than ever to getting a treatment into the hands of those who need it most. With your help, we can take our work another step forward. You can support the Walk for PKD by becoming a team captain, signing up as a walker, volunteering, donating or becoming a corporate sponsor. The Walk for PKD signifies a united group, moving forward toward treatments, and ultimately a cure. Honor patients, connect with other supporters and unite to fight PKD through raising awareness and fundraising for the PKD Foundation. As a supporter of the Walk for PKD, you help pave the way to a future without PKD. For more information and to register for the Walk in your area, visit walkforpkd.org. Registration is open.
Personal fundraising pages for the Walk are improved this year! You can access the page through your social media login, giving you access to share messages through social media that link directly to your page. You can also download an app to your smartphone where you can access your page at any time. If you are interested in becoming a corporate sponsor of the Walk for PKD, please contact cindyw@pkdcure.org.
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S u p p or t
Three Women, 25 Years of Loyal Donations
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inston Churchill once said, “We make a living by what we get, but we make a life by what we give.” At the PKD Foundation, we are endlessly grateful for the support of our donors, and their contributions that help us reach our goals. We recently spoke with three donors who have been giving to the Foundation every year for 25 years (since 1988). They shared with us what inspires their loyal support of the PKD Foundation. Judy Ehrlich is both a steadfast donor and volunteer. When she was diagnosed in 1983 with PKD, her nephrologist told her about the PKD Foundation and she quickly got involved. For the better part of the last 20 years, Judy has been a Chapter leader organizing education events for the New England Chapter. “Volunteering is so meaningful to me because I connect with others affected by the disease.” Over the years Judy
has seen a tremendous scientific shift. “When I first got involved they didn’t even know the basic genetics of PKD. Now we have made huge strides towards treatments.” Judy is committed to doing all she can to help end PKD. “Anything I can do, I want to do, because I can. I still have my health and my drive.” Corrine Smith donates to the Foundation in support of her husband. Diagnosed with PKD in 1979, her husband was one of four brothers; three of whom had PKD. He was on dialysis for 25 years before he passed away. Corrine has three daughters who have not tested positive for the disease. The Foundation’s emphasis on research inspires Corrine’s dedicated support. “The Foundation’s biggest strength is the work it does with scientists to discover treatments and a cure,” she said. Long-term donor Marlene Schwartz says the PKD Foundation should “keep up the good work.”
Leadership Conference Brings Chapters Together More than 60 PKD Foundation Chapter and Walk Coordinators from across the country met in February in Kansas City for our annual PKD Leadership Conference. Together they learned the latest updates within the Foundation and gathered new tools and tips for growing and improving their local Chapters. It’s now easier for people to find local Chapters. Visit pkdcure.org/connect/ chapter-locations to see the new Chapter locator. If you are interested in volunteering, contact cindyl@pkdcure.org.
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Over 25 years, Marlene has watched the organization experience great growth, increasing its supporter base and the number of activities that educate and raise awareness about the disease. She believes one of the biggest strengths of the Foundation is its strong management. While Marlene has seen progress over the years, she knows the work doesn’t end until we meet our goal. “We can’t stop working until we find a cure,” she said. The generosity and loyalty of our long-term donors is so meaningful to us, and we are grateful for their support, as well as everyone who makes financial contributions to the PKD Foundation. You are integral to putting treatments within reach and ending PKD for future generations. There are lots of different ways to donate to the Foundation. Learn more by visiting pkdcure.org/ donate/ways-to-donate. Tell your story of why you give at pkdcure. org/connect/share-your-story. n
Awa r e n e s s
New Progress: Update Your Subscription Today I hope you enjoyed reading our newly redesigned issue of Progress Magazine. This contemporary look reflects how we are moving forward as an organization, focused on the future. As Gary mentioned on page 2, we also want to tell the story better, through personal accounts, and describing the work we’re doing. We are also updating our mailing list, and giving you the opportunity to receive Progress online or in print (or both). You are receiving the magazine as a result of your past financial support of the Foundation. Progress is a resource we want to share with anyone who needs it, regardless of their ability to donate, so it’s now available to anyone who wishes to receive it. We do ask you to help underwrite production costs through a suggested $25 donation, but it is not required. Because of this change in how we will distribute Progress in the future, we are asking you to subscribe (or update your subscription) to continue to receive the magazine. To ensure that you continue to receive Progress, please visit us online at pkdcure.org/pkd-progress, or mail us the enclosed yellow remit envelope. With this issue and redesign, we are launching our focus on telling the PKD story. I have had the privilege to meet many PKD Foundation supporters, and hear their rich experiences. Our goal is to start sharing these stories. We want you to share your story and help connect others. These will be used in a variety of ways including: • In Progress (maybe you’ll be highlighted in a future issue) • On the PKD Foundation website – pkdcure.org • In PKDNews (monthly email newsletter) • Through social media To submit your story, please visit pkdcure.org/connect/share-your-story, or email pkdcure@pkdcure.org. Please keep reading about other exciting marketing updates below. Angela Connelly Chief Marketing Officer
Pkdcure.org Homepage Improvements The pkdcure.org website is a vital resource we provide the PKD community. It contains a variety of PKD-related health information, including the latest in research, education and ways to connect. We’ve made improvements that will make the website easier to navigate and help you get the information you need in an easy, efficient way. Whether you’re someone with PKD, a caregiver or health professional – you’re now just one click away from getting the specific information you need. We will also be launching discussion forums – stay tuned for updates.
Lullabies Album Available with Proceeds Benefiting the PKD Foundation The album Lullabies, by recording artist Sara Mann, former backup singer to Miley Cyrus and Katharine McPhee, is now available on iTunes. Sara lost her infant son to ARPKD in 2011 and recorded this album to comfort parents of ARPKD children, and others living with kidney disease or who have lost a loved one. Lullabies features the song Tears In Heaven, with Sara joined by Billy Ray Cyrus. Proceeds go to the PKD Foundation. To purchase the album, go to iTunes or saramann.net.
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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.
Double Your Contribution Today! The PKD Foundation Will MATCH Your Gift
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or a limited time, the PKD Foundation will double the impact of your gift to fight PKD. As part of a special Matching Gift Campaign, every dollar you contribute (up to $75,000) by June 30, 2013, will be matched by generous friends of the Foundation.
Double your gift and keep up the momentum toward finding treatments today! Mail: PKD Foundation, PO Box 871847, Kansas City, MO 64187-1847 Phone: 800.PKD.CURE (800.753.2873)
When making your gift, please indicate that it is for the Matching Gift Campaign.
Board of Trustees (2012-2013 term) Scott L. Goodman (Chair) Frank Condella (Vice Chair & Treasurer) Sen. Robert Bennett Bill Brazell Benjamin Cowley, Jr., M.D. Dr. Lisa Guay-Woodford Michael A. Haggard, Esq. Blaise Hazelwood Walter A. Hunt, Ph.D. Jerome T. Lienhard, II Richard R. Nelson Julia R. Roberts Anne Ryan Hilary Wolfe
Scientific Advisory Committee Benjamin Cowley, Jr., M.D. (Chair) Terry Watnick, M.D. (Vice Chair) John J. Bissler, M.D. Iain Drummond, Ph.D. Vincent H. Gattone II, Ph.D. Benjamin Margolis, M.D. York Pei, M.D. Ronald D. Perrone, M.D. Richard Sandford, Ph.D., FRCP Stefan Somlo, M.D. Darren Wallace, Ph.D. Angela Wandinger-Ness, Ph.D.
Please don’t wait. Take advantage of this rare opportunity to make your gift go twice as far. Visit pkdcure.org/doublemygift so the PKD Foundation can match your contribution to end PKD. Double your gift today: Online: pkdcure.org/doublemygift
PKD Foundation 8330 Ward Parkway, Suite 510 Kansas City, Missouri 64114 Website: pkdcure.org Email: pkdcure@pkdcure.org 816.931.2600 800.PKD.CURE FAX 816.931.8655 Like us! facebook.com/pkdfoundation Follow us! twitter.com/pkdfoundation
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Leadership Team Gary G. Godsey, President and CEO Angela Connelly, Chief Marketing Officer Michelle Davis, Chief Development Officer Ray Smith, Chief Financial Officer