www.pkdcure.org

SPECIAL EDITION

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PROGRESS

Thanks

REPORT

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I love this time of the year when families come together to share meals and memories, when we all pause to reflect on our past blessings and hopes for the future.

Another reason I like this time of year – my son comes home from college for not one but two holiday breaks!

You see, I never knew if my son would grow up to be a college student. He was diagnosed with ARPKD at birth, and from then on, our future was uncertain. Would his kidneys fail? Would he make it to high school without a transplant? Would he grow into a man with a family of his own?

While many questions still remain, what I knew then – and still know today – is how important, how absolutely essential, it is to fight PKD, fund research and find treatments and a cure.

That’s why we’re using this issue of PKD Progress to celebrate some of the exciting advancements of the past year – and the past 30 years (happy anniversary!) – and also thank our amazing supporters. Our volunteers, fundraisers and donors do so much to help further the fight against PKD.

We couldn’t do it without you!

Please know how grateful we are for your support in 2012 and over the last three decades. You deserve our most sincere thanks. Not just today but every day.

Happy holidays, and thank you once again.

Warm regards,

Scott Goodman Board of Trustees Chairman

As the Chair of the Board of Trustees, it is my great pleasure to welcome you to my favorite issue of PKD Progress magazine … the one where we give thanks.

A MESSAGE FROM OUR BOARD OF TRUSTEES CHAIRMAN

GO GREEN!PKD Progress Now Available Online at

pkdcure.org/progressonline

RESEARCH

CONNECT

ACT

LEARN

6 | 30 Milestones in 30 Years: What a Timeline!

8 | Giving Thanks For Our Many Supporters

12 | A Brand New Day For Marketing

10 | Meet Our New Chapter and Fundraising Teams

11 | Many Ways to Give

2 | A Time for Progress

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.

PKD Progress is our magazine for patients, Foundation supporters,

health professionals and research scientists. Views expressed in

the professional articles are those of the individual contributors and

do not reflect the opinion of the PKD Foundation.

ADVERTISING STATEMENT: The acceptance of advertising in this pub-lication does not constitute or imply endorsement by the PKD Foundation or any of its Chapters of any advertised product or service. People with PKD should consult their physicians before using any medication or therapy. The PKD Foun-dation accepts no responsibility for any claims made in any advertisement in this publication.

PKD PROGRESS VOL. 28 , ISSUE 3 FALL 2012/WINTER 2013

Website: pkdcure.org E-mail: pkdcure@pkdcure.org

(816) 931-2600(800) PKD-CUREFAX (816) 931-8655

Please contact the PKD Foundation for reprint information

PKD Foundation8330 Ward Parkway, Suite 510Kansas City, Missouri 64114

Like us on Facebook, and be a part of the discussion on finding a cure for PKD.

facebook.com/pkdfoundation

LIKE US!Get up to the minute information from

the PKD Foundation on everything from events to important kidney news.

twitter.com/pkdfoundation

FOLLOW US!

3

PKD

PRO

GRE

SS

ww

w.p

kdcu

re.o

rg

I love this time of the year when families come together to share meals and memories, when we all pause to reflect on our past blessings and hopes for the future.

Another reason I like this time of year – my son comes home from college for not one but two holiday breaks!

You see, I never knew if my son would grow up to be a college student. He was diagnosed with ARPKD at birth, and from then on, our future was uncertain. Would his kidneys fail? Would he make it to high school without a transplant? Would he grow into a man with a family of his own?

While many questions still remain, what I knew then – and still know today – is how important, how absolutely essential, it is to fight PKD, fund research and find treatments and a cure.

That’s why we’re using this issue of PKD Progress to celebrate some of the exciting advancements of the past year – and the past 30 years (happy anniversary!) – and also thank our amazing supporters. Our volunteers, fundraisers and donors do so much to help further the fight against PKD.

We couldn’t do it without you!

Please know how grateful we are for your support in 2012 and over the last three decades. You deserve our most sincere thanks. Not just today but every day.

Happy holidays, and thank you once again.

Warm regards,

Scott Goodman Board of Trustees Chairman

As the Chair of the Board of Trustees, it is my great pleasure to welcome you to my favorite issue of PKD Progress magazine … the one where we give thanks.

A MESSAGE FROM OUR BOARD OF TRUSTEES CHAIRMAN

GO GREEN!PKD Progress Now Available Online at

pkdcure.org/progressonline

RESEARCH

CONNECT

ACT

LEARN

6 | 30 Milestones in 30 Years: What a Timeline!

8 | Giving Thanks For Our Many Supporters

12 | A Brand New Day For Marketing

10 | Meet Our New Chapter and Fundraising Teams

11 | Many Ways to Give

2 | A Time for Progress

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.

PKD Progress is our magazine for patients, Foundation supporters,

health professionals and research scientists. Views expressed in

the professional articles are those of the individual contributors and

do not reflect the opinion of the PKD Foundation.

ADVERTISING STATEMENT: The acceptance of advertising in this pub-lication does not constitute or imply endorsement by the PKD Foundation or any of its Chapters of any advertised product or service. People with PKD should consult their physicians before using any medication or therapy. The PKD Foun-dation accepts no responsibility for any claims made in any advertisement in this publication.

PKD PROGRESS VOL. 28 , ISSUE 3 FALL 2012/WINTER 2013

Website: pkdcure.org E-mail: pkdcure@pkdcure.org

(816) 931-2600(800) PKD-CUREFAX (816) 931-8655

Please contact the PKD Foundation for reprint information

PKD Foundation8330 Ward Parkway, Suite 510Kansas City, Missouri 64114

Like us on Facebook, and be a part of the discussion on finding a cure for PKD.

facebook.com/pkdfoundation

LIKE US!Get up to the minute information from

the PKD Foundation on everything from events to important kidney news.

twitter.com/pkdfoundation

FOLLOW US!

The Accelerating Treatments to Patients (ATP) initiative is a comprehensive, inte-grated research and development program that represents the core of our work. ATP was launched in 2010 to speed up develop-ment of treatments which could slow or stop progression of PKD. Each of the programs in ATP is important and interconnected.

Celebrating PROGRESSTISSUE DONATION PROGRAMThe PKD Foundation coordinates donations of dis-carded human PKD kidneys to research labs around the country. These kidneys are living tissue and come from patients who have had their kidneys removed (a procedure called a nephrectomy). This program provides an important way for individuals to actively participate and support the basic science. This tissue is highly valuable for researchers to learn about how PKD progresses through kidneys.

This program has been ongoing since 2003, with approximately $5,000 to $10,000 invested each year (nearly $100,000 total).

Since January 2012, we have contacted research-ers on behalf of patients 17 times for potential kidney donations.

CORE RESEARCH GRANTSIn 2012 the PKD Foundation went from funding two core labs to three. This program is focused on pro-viding grants that support facilities or services to PKD scientists at no cost (or at a greatly discounted cost). This allows Foundation dollars to be leveraged across the PKD research community.

In the past year, $160,000 has been distributed among these core labs. They include:

Electron Microscopy Core for PKD Research led by Dr. Vincent Gattone, Indiana University School of Medicine.

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.

DRUG REPURPOSINGThis program is focused on assisting biotech and pharmaceutical companies or other research organiza-tions in evaluating whether their drug candidates have potential as treatments for PKD. The goal is to foster the discovery and development of new therapies for PKD and to build a robust clinical trial pipeline in the most cost effective, least risky and expedient way. The aver-age time to develop a new drug is 13 years, with an average cost of $750 million. Drug repurposing shaves years and millions off the process.

The strategy is to evaluate at a minimum 25-30 promising, existing drug candidates over the next three to five years.

While time is saved by using a repurposed drug, the studies are still a lengthy and complex process. It costs approximately $600,000 and takes two and a half years to move a drug candidate preclinical evaluation from start to finish. Of that amount, it costs $240,000 annually ($20,000 per month) to care for the models the drugs are tested on.

There are currently five promising drug candi-dates in various stages being evaluated in three animal models with renal cystic disease. There are three additional drug candidates currently being considered for evaluation when we have additional funding ($600,000 per candidate, or $1.8 million total).

In developing the Foundation’s drug repurposing pro-gram, much work went into establishing a solid plat-form to be able to successfully conduct testing. With that groundwork laid, progress has been made in mov-ing forward with evaluating several drug candidates.

Since the beginning of this program we have invested $1.73 million and accomplished the following:

Established a Drug Development Advisory Group with more than 160 years collectively of drug dis-covery experience.

Established a relationship with a Contract Research Laboratory (CRL) to conduct the tests.

Established three animal models with renal cystic disease at the contract research organization. This includes a model that is related to ARPKD.

Through the Drug Advisory group’s network with-in the biotech and pharmaceutical industries we have identified many drug candidates that the group believes are worth pursuing and meet our criteria for selection.

Thirty years ago Dr. Jared J. Grantham and Joseph H. Bruening started the PKD Foundation with a vision to find treatments and a cure for PKD. The genes for PKD hadn't been identified, and there were only a handful of researchers working in PKD science. They knew there was a lot of work ahead, but they were determined.

Today, we are getting closer to finding treatments, and helping improve people’s lives so they do not suffer the full effects of PKD. Since 1982, more than $31 million has been invested in more than 550 research, clinical and scientific grants, fellowships, scientific meetings and education programs, making us the second largest funder of PKD research after the National Institutes of Health (NIH).

Our current work is moving us forward faster than ever. Please continue reading to learn about the progress being made.

The goal is to foster the discovery and development of new therapies for PKD and

to build a robust clinical trial pipeline in the most cost effective, least risky and expedient way."

PROGRAMS PURPOSE

Drug Repurposing

Scientific Meetings

PKD Outcomes Consortium Project (PKDOC)

Clinical Trial Awareness Program

Coordinating donations of discarded PKD kid-neys to research labs, allowing individuals to actively participate and support basic science.

Supporting services and facilities for PKD scientists, for Foundation dollars to be lever-aged across the PKD research community.

Finding potential drug candidates or new uses for existing drugs.

Working with the (Food and Drug Administra-tion) FDA for regulatory approval to use total kidney volume as an outcome measure in PKD drug trials.

Supporting clinical trials by raising awareness through PKD Foundation patients, families and health professionals.

Funding PKD related meetings that bring experts together and provide continuing education for health professionals.

Core Research Grants

Tissue Donation Program

RESEARCH: ACCELERATING TREATMENTS TO PATIENTS

4 5

PKD

PRO

GRE

SS

ww

w.p

kdcu

re.o

rg

The Accelerating Treatments to Patients (ATP) initiative is a comprehensive, inte-grated research and development program that represents the core of our work. ATP was launched in 2010 to speed up develop-ment of treatments which could slow or stop progression of PKD. Each of the programs in ATP is important and interconnected.

Celebrating PROGRESSTISSUE DONATION PROGRAMThe PKD Foundation coordinates donations of dis-carded human PKD kidneys to research labs around the country. These kidneys are living tissue and come from patients who have had their kidneys removed (a procedure called a nephrectomy). This program provides an important way for individuals to actively participate and support the basic science. This tissue is highly valuable for researchers to learn about how PKD progresses through kidneys.

This program has been ongoing since 2003, with approximately $5,000 to $10,000 invested each year (nearly $100,000 total).

Since January 2012, we have contacted research-ers on behalf of patients 17 times for potential kidney donations.

CORE RESEARCH GRANTSIn 2012 the PKD Foundation went from funding two core labs to three. This program is focused on pro-viding grants that support facilities or services to PKD scientists at no cost (or at a greatly discounted cost). This allows Foundation dollars to be leveraged across the PKD research community.

In the past year, $160,000 has been distributed among these core labs. They include:

Electron Microscopy Core for PKD Research led by Dr. Vincent Gattone, Indiana University School of Medicine.

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.

DRUG REPURPOSINGThis program is focused on assisting biotech and pharmaceutical companies or other research organiza-tions in evaluating whether their drug candidates have potential as treatments for PKD. The goal is to foster the discovery and development of new therapies for PKD and to build a robust clinical trial pipeline in the most cost effective, least risky and expedient way. The aver-age time to develop a new drug is 13 years, with an average cost of $750 million. Drug repurposing shaves years and millions off the process.

The strategy is to evaluate at a minimum 25-30 promising, existing drug candidates over the next three to five years.

While time is saved by using a repurposed drug, the studies are still a lengthy and complex process. It costs approximately $600,000 and takes two and a half years to move a drug candidate preclinical evaluation from start to finish. Of that amount, it costs $240,000 annually ($20,000 per month) to care for the models the drugs are tested on.

There are currently five promising drug candi-dates in various stages being evaluated in three animal models with renal cystic disease. There are three additional drug candidates currently being considered for evaluation when we have additional funding ($600,000 per candidate, or $1.8 million total).

In developing the Foundation’s drug repurposing pro-gram, much work went into establishing a solid plat-form to be able to successfully conduct testing. With that groundwork laid, progress has been made in mov-ing forward with evaluating several drug candidates.

Since the beginning of this program we have invested $1.73 million and accomplished the following:

Established a Drug Development Advisory Group with more than 160 years collectively of drug dis-covery experience.

Established a relationship with a Contract Research Laboratory (CRL) to conduct the tests.

Established three animal models with renal cystic disease at the contract research organization. This includes a model that is related to ARPKD.

Through the Drug Advisory group’s network with-in the biotech and pharmaceutical industries we have identified many drug candidates that the group believes are worth pursuing and meet our criteria for selection.

Thirty years ago Dr. Jared J. Grantham and Joseph H. Bruening started the PKD Foundation with a vision to find treatments and a cure for PKD. The genes for PKD hadn't been identified, and there were only a handful of researchers working in PKD science. They knew there was a lot of work ahead, but they were determined.

Today, we are getting closer to finding treatments, and helping improve people’s lives so they do not suffer the full effects of PKD. Since 1982, more than $31 million has been invested in more than 550 research, clinical and scientific grants, fellowships, scientific meetings and education programs, making us the second largest funder of PKD research after the National Institutes of Health (NIH).

Our current work is moving us forward faster than ever. Please continue reading to learn about the progress being made.

The goal is to foster the discovery and development of new therapies for PKD and

to build a robust clinical trial pipeline in the most cost effective, least risky and expedient way."

PROGRAMS PURPOSE

Drug Repurposing

Scientific Meetings

PKD Outcomes Consortium Project (PKDOC)

Clinical Trial Awareness Program

Coordinating donations of discarded PKD kid-neys to research labs, allowing individuals to actively participate and support basic science.

Supporting services and facilities for PKD scientists, for Foundation dollars to be lever-aged across the PKD research community.

Finding potential drug candidates or new uses for existing drugs.

Working with the (Food and Drug Administra-tion) FDA for regulatory approval to use total kidney volume as an outcome measure in PKD drug trials.

Supporting clinical trials by raising awareness through PKD Foundation patients, families and health professionals.

Funding PKD related meetings that bring experts together and provide continuing education for health professionals.

Core Research Grants

Tissue Donation Program

RESEARCH: ACCELERATING TREATMENTS TO PATIENTS

4 5

PKD

PRO

GRE

SS

ww

w.p

kdcu

re.o

rg

We have Material Transfer Agreements in place and have access to five promising drug candi-dates that work on distinctly different therapeutic targets which are believed to be important in de-velopment of the disease.

One of these drug candidates is showing promise in one of the renal cystic models. It has moved to the next model. Each candidate must be tested on each model.

Several drug candidates are just entering testing and we await results.

PKD OUTCOMES CONSORTIUM PROjECT (PKDOC) PKDOC is a significant collaboration between the Foundation, Critical Path Institute, members of the pharmaceutical industry, PKD researchers and clini-cians, and the Food and Drug Administration (FDA). The goal is to facilitate clinical trial development

for PKD therapies by establish-ing regulatory endpoints (or biomarkers) to measure early disease progression. Current traditional endpoints only show changes late in the course of the disease when kidneys are ap-proximately five times normal size and normal tissue has been destroyed by hundreds of cysts and fibrotic tissue. At this stage, it may be too late for treatment.

The PKDOC has identified total kidney volume (TKV) as an imaging biomarker that is most relevant for following the natural course of the disease. (A bio-marker is a characteristic that can be measured reli-ably as an indicator of disease processes.)

This work has been solely funded by the PKD Foundation, an investment of more than $2.3 million.

A new shared database of aggregated patient data was created from three existing research registries. This is the largest database ever created of PKD patients using standardized terminology.

On Nov. 9, an in-person meeting with the FDA took place to review the PKDOC summary and analysis. The steering committee believed it was

a productive meeting and that good progress is being made. The analysis will continue to be re-fined for another submission. A second in-per-son meeting is anticipated for early 2013, which will be followed by the formal submission of the biomarker qualification package.

CLINICAL TRIAL AWARENESS PROGRAM The PKD Foundation be-lieves empowered patients are the key to accelerating the development of a robust clinical trial pipeline. With-out patient participation in medical research, medical breakthroughs cannot hap-pen. This program is focused on educating patients about current studies so they can make educated decisions about participating.

This program was launched in 2010 and we have in-vested approximately $20,000 to $25,000 per year.

We are increasing awareness about trials by pro-viding emails to patients and physicians. These are called ACT (accelerating clinical trial) alerts. There are approximately 30,000 people who receive alerts.

SCIENTIFIC MEETINGSThis program is focused on funding PKD-relevant scientific and clinical meetings that bring together PKD scientists from around the world to discuss current findings and encourage research collabora-tions. The PKD Foundation has also funded medical education programs at national clinical meetings to educate health professionals in the basic science, diagnosis, disease management and potential treat-ments for PKD.

We have provided funding through educational grants to support scientific conferences and meetings that support the Foundation’s mission. The funding has ranged from $5,000 to $50,000 per conference.

Many partnerships are being cultivated to support collaboration with other key organizations, includ-ing the National Kidney Foundation (NKF), and the International Society of Nephrology (ISN).

ADvOCACy The Foundation plays a key role in legislative advocacy on behalf of the PKD community to support PKD related initiatives. For example, we worked to ensure that the Genetic Informa-tion Nondiscrimination Act (GINA) was signed into law so that people with PKD do not en-counter hurdles in obtaining health insurance.

In 2012 we invested $133,500 in this work.

Every year, the Foundation organizes United on the Hill (UOTH), a legislative and policy conference in Washington D.C. Foundation leaders and PKD community participants meet with members of Congress to raise PKD awareness and discuss legislative pri-orities. In June 2012, 41 people including 22 new advocates representing 14 states con-ducted more than 40 meetings on Capitol Hill during this event.

PATIENT EDUCATIONBy providing in-depth educational information about PKD and issues related to health care and lifestyle around the disease, we can empower patients to manage their health. This work in-cludes:

In 2012, five educational webinars were attended by 750 patients, families and health professionals, an investment of more than $4,000. Topics included Autosomal Reces-sive PKD (ARPKD), clinical trials, research updates, living positively with PKD, kidney transplants in children, and the Affordable Care Act.

The Foundation developed the online national education conference “What’s New in PKD” in partnership with the PKD Foundation’s New England Chapter. More than 200 people attended in 2012, and the 2013 event will be March 9 (see page 11).

We support local PKD Chapters through as-sistance with support groups and seminars.

This program

brings together

PKD scientists from

around the world…

to encourage research

collaborations."

TOLvAPTAN UPDATE

Results of the Tolvaptan TEMPO 3/4 trial show that the drug slows the progression of PKD. This was announced by Otsuka Pharmaceutical Co. LTD in November, at the same time results were published online in the New England Jour-nal of Medicine (NWJM.org). Dr. Jared Grantham, co-founder of the PKD Foundation, was co-author of the report.

The NEJM study reports that tolvaptan slows the enlarge-ment of cystic kidneys while also slowing the loss of kidney function. Patients in the trial who took the drug saw their kid-neys grow at about half the rate of the control group over three years. The rate of declining kidney function was also about half, compared to patients taking the placebo.

The Foundation played a key role in developing this treat-ment by funding and supporting research that made this work possible.

“This is an exciting milestone for PKD patients and the Founda-tion,” said Gary Godsey, PKD Foundation president and CEO.

ABOUT THE STUDy

This study was conducted over three years at multiple inter-national sites, with 1,445 participants. Patients with PKD took either a placebo or tolvaptan – a drug that was tested on the basis of decades-long research started by PKD Foundation co-founder Dr. Grantham in the 1970s. The concept of using vasopressin blockade as a way of slowing ADPKD progres-sion was originally developed by Dr. Vincent Gattone, whose research has been in part supported by the PKD Foundation.

It is important to know that though tolvaptan is already ap-proved for treatment of other medical conditions, the doses of tolvaptan used in the TEMPO trial were significantly higher than used in previous studies of other diseases. In addition, ADPKD patients are a unique patient population. Because of this, until the data is reviewed in detail by Otsuka and the FDA, it could be dangerous to take tolvaptan for treatment of PKD.)

The Foundation is proud to have been a

part of this critical work, and our hope

is that this is just the first step toward finding

other treatments that will improve the quality of

people’s lives who suffer from PKD."

This work has

been solely

funded by the PKD

Foundation, an

investment of more

than $2.3 million."

6

PKD

PRO

GRE

SS

FALL

201

2/W

INTE

R 20

13

7

We have Material Transfer Agreements in place and have access to five promising drug candi-dates that work on distinctly different therapeutic targets which are believed to be important in de-velopment of the disease.

One of these drug candidates is showing promise in one of the renal cystic models. It has moved to the next model. Each candidate must be tested on each model.

Several drug candidates are just entering testing and we await results.

PKD OUTCOMES CONSORTIUM PROjECT (PKDOC) PKDOC is a significant collaboration between the Foundation, Critical Path Institute, members of the pharmaceutical industry, PKD researchers and clini-cians, and the Food and Drug Administration (FDA). The goal is to facilitate clinical trial development

for PKD therapies by establish-ing regulatory endpoints (or biomarkers) to measure early disease progression. Current traditional endpoints only show changes late in the course of the disease when kidneys are ap-proximately five times normal size and normal tissue has been destroyed by hundreds of cysts and fibrotic tissue. At this stage, it may be too late for treatment.

The PKDOC has identified total kidney volume (TKV) as an imaging biomarker that is most relevant for following the natural course of the disease. (A bio-marker is a characteristic that can be measured reli-ably as an indicator of disease processes.)

This work has been solely funded by the PKD Foundation, an investment of more than $2.3 million.

A new shared database of aggregated patient data was created from three existing research registries. This is the largest database ever created of PKD patients using standardized terminology.

On Nov. 9, an in-person meeting with the FDA took place to review the PKDOC summary and analysis. The steering committee believed it was

a productive meeting and that good progress is being made. The analysis will continue to be re-fined for another submission. A second in-per-son meeting is anticipated for early 2013, which will be followed by the formal submission of the biomarker qualification package.

CLINICAL TRIAL AWARENESS PROGRAM The PKD Foundation be-lieves empowered patients are the key to accelerating the development of a robust clinical trial pipeline. With-out patient participation in medical research, medical breakthroughs cannot hap-pen. This program is focused on educating patients about current studies so they can make educated decisions about participating.

This program was launched in 2010 and we have in-vested approximately $20,000 to $25,000 per year.

We are increasing awareness about trials by pro-viding emails to patients and physicians. These are called ACT (accelerating clinical trial) alerts. There are approximately 30,000 people who receive alerts.

SCIENTIFIC MEETINGSThis program is focused on funding PKD-relevant scientific and clinical meetings that bring together PKD scientists from around the world to discuss current findings and encourage research collabora-tions. The PKD Foundation has also funded medical education programs at national clinical meetings to educate health professionals in the basic science, diagnosis, disease management and potential treat-ments for PKD.

We have provided funding through educational grants to support scientific conferences and meetings that support the Foundation’s mission. The funding has ranged from $5,000 to $50,000 per conference.

Many partnerships are being cultivated to support collaboration with other key organizations, includ-ing the National Kidney Foundation (NKF), and the International Society of Nephrology (ISN).

ADvOCACy The Foundation plays a key role in legislative advocacy on behalf of the PKD community to support PKD related initiatives. For example, we worked to ensure that the Genetic Informa-tion Nondiscrimination Act (GINA) was signed into law so that people with PKD do not en-counter hurdles in obtaining health insurance.

In 2012 we invested $133,500 in this work.

Every year, the Foundation organizes United on the Hill (UOTH), a legislative and policy conference in Washington D.C. Foundation leaders and PKD community participants meet with members of Congress to raise PKD awareness and discuss legislative pri-orities. In June 2012, 41 people including 22 new advocates representing 14 states con-ducted more than 40 meetings on Capitol Hill during this event.

PATIENT EDUCATIONBy providing in-depth educational information about PKD and issues related to health care and lifestyle around the disease, we can empower patients to manage their health. This work in-cludes:

In 2012, five educational webinars were attended by 750 patients, families and health professionals, an investment of more than $4,000. Topics included Autosomal Reces-sive PKD (ARPKD), clinical trials, research updates, living positively with PKD, kidney transplants in children, and the Affordable Care Act.

The Foundation developed the online national education conference “What’s New in PKD” in partnership with the PKD Foundation’s New England Chapter. More than 200 people attended in 2012, and the 2013 event will be March 9 (see page 11).

We support local PKD Chapters through as-sistance with support groups and seminars.

This program

brings together

PKD scientists from

around the world…

to encourage research

collaborations."

TOLvAPTAN UPDATE

Results of the Tolvaptan TEMPO 3/4 trial show that the drug slows the progression of PKD. This was announced by Otsuka Pharmaceutical Co. LTD in November, at the same time results were published online in the New England Jour-nal of Medicine (NWJM.org). Dr. Jared Grantham, co-founder of the PKD Foundation, was co-author of the report.

The NEJM study reports that tolvaptan slows the enlarge-ment of cystic kidneys while also slowing the loss of kidney function. Patients in the trial who took the drug saw their kid-neys grow at about half the rate of the control group over three years. The rate of declining kidney function was also about half, compared to patients taking the placebo.

The Foundation played a key role in developing this treat-ment by funding and supporting research that made this work possible.

“This is an exciting milestone for PKD patients and the Founda-tion,” said Gary Godsey, PKD Foundation president and CEO.

ABOUT THE STUDy

This study was conducted over three years at multiple inter-national sites, with 1,445 participants. Patients with PKD took either a placebo or tolvaptan – a drug that was tested on the basis of decades-long research started by PKD Foundation co-founder Dr. Grantham in the 1970s. The concept of using vasopressin blockade as a way of slowing ADPKD progres-sion was originally developed by Dr. Vincent Gattone, whose research has been in part supported by the PKD Foundation.

It is important to know that though tolvaptan is already ap-proved for treatment of other medical conditions, the doses of tolvaptan used in the TEMPO trial were significantly higher than used in previous studies of other diseases. In addition, ADPKD patients are a unique patient population. Because of this, until the data is reviewed in detail by Otsuka and the FDA, it could be dangerous to take tolvaptan for treatment of PKD.)

The Foundation is proud to have been a

part of this critical work, and our hope

is that this is just the first step toward finding

other treatments that will improve the quality of

people’s lives who suffer from PKD."

This work has

been solely

funded by the PKD

Foundation, an

investment of more

than $2.3 million."

6

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2008PKD Foundation hosts

its first educational webinar, “Basics of PKD,” presented by

Dr. Ron Perrone.

1990PKD Foundation successfully lobbies Congress to include the first Appropriations Committee report language regarding PKD research at the National Institutes of Health (NIH). The report language encourages NIH to expand its research agenda in the PKD field.

The NIH makes first federal investment in PKD research of $5.73 million.1982

PKD Foundation is established by Dr. Jared Grantham and Joseph Bruening to find a treatment and cure for polycystic kidney disease.

First grant of $25,000 awarded to Dr. William Bennett.

PKD Progress magazine created to educate patients and health professionals about advancements in the field of PKD research, education and more.

First local PKD Chapter “Friends Group” begins in Chicago. Now more than 60 volunteer-led Chapters operate across the country.

First National Convention on PKD for patients and medical professionals occurs in Kansas City.

1984

1993

CRISP (Consortium for Radiologic Imaging Study Program) Study begins.

2000

Discovery of the PKD1 gene, responsible for 85% of ADPKD cases.

1994

1995

1987

1985

1989

2012Tolvaptan Tempo ¾ Clinical Trial is completed. Results are announced that show the drug as being effective in slowing down progression of PKD.

2012PKD Foundation partners with PKD advocate Valen Keefer to launch new blog, PKD Will Not Beat Me.

2011PKD Foundation partners with Renal Dietitian Kelly Welsh for its educational health blog, PKD

Health Notes.

2010PKD Foundation creates Drug Development Advisory Group (DDAG), made up of world renowned specialists in drug discovery.

1998NIH establishes PKD Centers of Excellence at four research institutions to focus on cutting-edge PKD research.

1998PKD Foundation’s website, pkdcure.org, is launched

2007FDA and PKDF workshop takes place regarding finding clinical trial end points in PKD.

2010PKD Outcomes Consortium (PKDOC) formed to support total kidney volume as an outcome measurement in clinical trials.

Discovery of the PKD2 gene, responsible for 15% of ADPKD cases.

2000900 PKD patients, family members and friends raise $214,000 in the first Walk for PKD.

2008After a 13-year fight, GINA is passed by Congress and signed into law by President Bush.

2008PKD Foundation’s first United on the Hill advocacy event is held in Washington, D.C. Advocates from 24 states push for more federal PKD research funding and the passage of GINA*.

*(Genetic InformationNondiscrimination Act)

2006U.S. Senate passes first-ever National PKD Awareness Week Resolution to help promote the PKD Foundation’s Walk for PKD.

2002Vasopressin receptor identified as possible therapeutic target in PKD treatment.

2002

Discovery of ARPKD gene.

2004Tolvaptan, the first drug to potentially treat PKD, enters clinical trials in humans.

2005The HALT PKD Clinical Trials, which studies effects of blood pressure treatments on PKD, begins.

2010PKD Foundation launches its Accelerating Treatments to Patients initiative, designed to speed up the development of treatments for PKD.

2012Tolvaptan is granted Orphan Drug Status by the U.S. Federal Food and Drug Administration (FDA). This status may help speed up the development of potential treatments and may also encourage more pharmaceutical companies to develop drugs to treat ADPKD.

2012PKD Foundation meets with FDA to present PKDOC analysis.

98

2008PKD Foundation hosts

its first educational webinar, “Basics of PKD,” presented by

Dr. Ron Perrone.

1990PKD Foundation successfully lobbies Congress to include the first Appropriations Committee report language regarding PKD research at the National Institutes of Health (NIH). The report language encourages NIH to expand its research agenda in the PKD field.

The NIH makes first federal investment in PKD research of $5.73 million.1982

PKD Foundation is established by Dr. Jared Grantham and Joseph Bruening to find a treatment and cure for polycystic kidney disease.

First grant of $25,000 awarded to Dr. William Bennett.

PKD Progress magazine created to educate patients and health professionals about advancements in the field of PKD research, education and more.

First local PKD Chapter “Friends Group” begins in Chicago. Now more than 60 volunteer-led Chapters operate across the country.

First National Convention on PKD for patients and medical professionals occurs in Kansas City.

1984

1993

CRISP (Consortium for Radiologic Imaging Study Program) Study begins.

2000

Discovery of the PKD1 gene, responsible for 85% of ADPKD cases.

1994

1995

1987

1985

1989

2012Tolvaptan Tempo ¾ Clinical Trial is completed. Results are announced that show the drug as being effective in slowing down progression of PKD.

2012PKD Foundation partners with PKD advocate Valen Keefer to launch new blog, PKD Will Not Beat Me.

2011PKD Foundation partners with Renal Dietitian Kelly Welsh for its educational health blog, PKD

Health Notes.

2010PKD Foundation creates Drug Development Advisory Group (DDAG), made up of world renowned specialists in drug discovery.

1998NIH establishes PKD Centers of Excellence at four research institutions to focus on cutting-edge PKD research.

1998PKD Foundation’s website, pkdcure.org, is launched

2007FDA and PKDF workshop takes place regarding finding clinical trial end points in PKD.

2010PKD Outcomes Consortium (PKDOC) formed to support total kidney volume as an outcome measurement in clinical trials.

Discovery of the PKD2 gene, responsible for 15% of ADPKD cases.

2000900 PKD patients, family members and friends raise $214,000 in the first Walk for PKD.

2008After a 13-year fight, GINA is passed by Congress and signed into law by President Bush.

2008PKD Foundation’s first United on the Hill advocacy event is held in Washington, D.C. Advocates from 24 states push for more federal PKD research funding and the passage of GINA*.

*(Genetic InformationNondiscrimination Act)

2006U.S. Senate passes first-ever National PKD Awareness Week Resolution to help promote the PKD Foundation’s Walk for PKD.

2002Vasopressin receptor identified as possible therapeutic target in PKD treatment.

2002

Discovery of ARPKD gene.

2004Tolvaptan, the first drug to potentially treat PKD, enters clinical trials in humans.

2005The HALT PKD Clinical Trials, which studies effects of blood pressure treatments on PKD, begins.

2010PKD Foundation launches its Accelerating Treatments to Patients initiative, designed to speed up the development of treatments for PKD.

2012Tolvaptan is granted Orphan Drug Status by the U.S. Federal Food and Drug Administration (FDA). This status may help speed up the development of potential treatments and may also encourage more pharmaceutical companies to develop drugs to treat ADPKD.

2012PKD Foundation meets with FDA to present PKDOC analysis.

98

THIS yEAR, THE PKD FOUNDATION HAS MANy REASONS TO GIvE THANKS.

MARIE HAyS We often speak of donors and volunteers who devote much of their time and money to finding a cure for PKD. Marie Hays is an example of one such donor. For the last 22 years, the PKD Foundation has been the fortunate recipient of Marie’s generosity.

Marie began donating to PKD when she was diagnosed with the disease at the age of 40.

Adopted as a baby, Marie set out to discover which side of her family also had PKD. She later learned her father had the disease and had previously received a kidney transplant.

This October, Marie will celebrate five years since her own kidney transplant.

With no plans to curb her giving, Marie says even though a cure might be out of reach for her lifetime, she says she has hopes for the future - among other practical reasons for giving.

“I figure I’d rather give my money to PKD than the government,” Hays jokes.

It’s this casual reference toward her giving that is why Marie remains a cherished member of the PKD family and a reminder that no donation is too small to make an impact on one of the most common, life-threatening diseases in the world.

GABRIELLE COLE Gabrielle Cole stepped up as this year’s San Francisco Walk for PKD Coordinator for six very compelling reasons:

Her mother, three brothers and two nephews.

All have PKD. All are hopeful for treatments and a cure.

“Once my mom was going through her second kidney transplant is when I really realized I needed to learn everything there is to know about this disease,”

GIvING THANKSfor our MANy SUPPORTERS

During this annual season of thanks, we’d like to introduce you to some of our many dedicated volunteers, fundraisers and donors who give their time, treasure and talents.

says Cole. “It has truly affected my entire family.”

When the opportunity for Walk Coordinator became available, Cole knew this was her chance to make a difference.

“I took this challenge because I believe with all my heart a cure is in our reach,” says Cole.

Last February, still new in her role, Cole was preparing for her first PKD Foundation Leadership Conference in Kansas City when she received some startling news.

Her efforts to raise money and awareness for PKD would not only benefit her mother, brothers and nephews; she would be working toward a cure for herself. She, too, had inherited PKD.

With even more passion for the cause, Cole threw herself into plans for her first Walk as coordinator, enhancing the event with a live band, face painters and a children’s choir, which sang a dedication song before the Walk began.

“My goal was to provide a fun-filled day to help PKD patients and supporters create amazing memories and provide encouragement knowing we are taking incredible steps toward finding a cure,” says Cole.

LISA WAxMANHouston’s Walk for PKD has been the benefactor of Lisa Waxman’s unique fundraising efforts for five years now.

After being diagnosed with PKD during a routine CT scan, Waxman immediately signed up for the Walk for PKD and has been walking ever since.

This year, her team, PolyScentastic (meaning many scents), met their goal of $15,000.

But Lisa isn’t sending out your conventional email pleas for donations; she’s gotten more creative over the years.

Lisa has made fundraising a year-round, part-time job by joining and becoming a director with Scentsy Wickless, a company that sells scented, flameless candles and other scented products.

She regularly hosts Scentsy parties or sets up tables at events, such as her local church bazaar or the Chocolate and Wine Festival of Texas. All of her profits, minus overhead expenses, go to her team’s fundraising goal.

“I tell my Scentsy story at every party and event,” says Waxman. “Everyone knows I sell these products because I have PKD, along with members of my family, and want to help make a difference.”

Lisa has also received support from her employer, Albemarle Corporation, which matches all her fundraising dollars. Lisa says the company has been really great to her and another coworker who also has PKD.

Lisa suggests other people who are having difficulty fundraising consider direct sales as well.

“It’s been much easier this way because people know they aren’t just getting a great product, but they are giving to an even greater cause,” says Waxman.

To become a coordinator, contact Cindy LeBlanc, Chapter Relations Manager, at cindyl@pkdcure.org

My goal was to provide a fun-filled day to help PKD

patients and supporters create amazing memories and provide encouragement knowing we are taking incredible steps toward finding a cure.”

Gabrielle ColeSan Francisco Walk for PKD Coordinator

10 11

THIS yEAR, THE PKD FOUNDATION HAS MANy REASONS TO GIvE THANKS.

MARIE HAyS We often speak of donors and volunteers who devote much of their time and money to finding a cure for PKD. Marie Hays is an example of one such donor. For the last 22 years, the PKD Foundation has been the fortunate recipient of Marie’s generosity.

Marie began donating to PKD when she was diagnosed with the disease at the age of 40.

Adopted as a baby, Marie set out to discover which side of her family also had PKD. She later learned her father had the disease and had previously received a kidney transplant.

This October, Marie will celebrate five years since her own kidney transplant.

With no plans to curb her giving, Marie says even though a cure might be out of reach for her lifetime, she says she has hopes for the future - among other practical reasons for giving.

“I figure I’d rather give my money to PKD than the government,” Hays jokes.

It’s this casual reference toward her giving that is why Marie remains a cherished member of the PKD family and a reminder that no donation is too small to make an impact on one of the most common, life-threatening diseases in the world.

GABRIELLE COLE Gabrielle Cole stepped up as this year’s San Francisco Walk for PKD Coordinator for six very compelling reasons:

Her mother, three brothers and two nephews.

All have PKD. All are hopeful for treatments and a cure.

“Once my mom was going through her second kidney transplant is when I really realized I needed to learn everything there is to know about this disease,”

GIvING THANKSfor our MANy SUPPORTERS

During this annual season of thanks, we’d like to introduce you to some of our many dedicated volunteers, fundraisers and donors who give their time, treasure and talents.

says Cole. “It has truly affected my entire family.”

When the opportunity for Walk Coordinator became available, Cole knew this was her chance to make a difference.

“I took this challenge because I believe with all my heart a cure is in our reach,” says Cole.

Last February, still new in her role, Cole was preparing for her first PKD Foundation Leadership Conference in Kansas City when she received some startling news.

Her efforts to raise money and awareness for PKD would not only benefit her mother, brothers and nephews; she would be working toward a cure for herself. She, too, had inherited PKD.

With even more passion for the cause, Cole threw herself into plans for her first Walk as coordinator, enhancing the event with a live band, face painters and a children’s choir, which sang a dedication song before the Walk began.

“My goal was to provide a fun-filled day to help PKD patients and supporters create amazing memories and provide encouragement knowing we are taking incredible steps toward finding a cure,” says Cole.

LISA WAxMANHouston’s Walk for PKD has been the benefactor of Lisa Waxman’s unique fundraising efforts for five years now.

After being diagnosed with PKD during a routine CT scan, Waxman immediately signed up for the Walk for PKD and has been walking ever since.

This year, her team, PolyScentastic (meaning many scents), met their goal of $15,000.

But Lisa isn’t sending out your conventional email pleas for donations; she’s gotten more creative over the years.

Lisa has made fundraising a year-round, part-time job by joining and becoming a director with Scentsy Wickless, a company that sells scented, flameless candles and other scented products.

She regularly hosts Scentsy parties or sets up tables at events, such as her local church bazaar or the Chocolate and Wine Festival of Texas. All of her profits, minus overhead expenses, go to her team’s fundraising goal.

“I tell my Scentsy story at every party and event,” says Waxman. “Everyone knows I sell these products because I have PKD, along with members of my family, and want to help make a difference.”

Lisa has also received support from her employer, Albemarle Corporation, which matches all her fundraising dollars. Lisa says the company has been really great to her and another coworker who also has PKD.

Lisa suggests other people who are having difficulty fundraising consider direct sales as well.

“It’s been much easier this way because people know they aren’t just getting a great product, but they are giving to an even greater cause,” says Waxman.

To become a coordinator, contact Cindy LeBlanc, Chapter Relations Manager, at cindyl@pkdcure.org

My goal was to provide a fun-filled day to help PKD

patients and supporters create amazing memories and provide encouragement knowing we are taking incredible steps toward finding a cure.”

Gabrielle ColeSan Francisco Walk for PKD Coordinator

10 11

Leading the way is Chief Development Officer Michelle Davis, who joined the PKD Foundation in April.

According to Davis, the team has a wealth of experience in the non-profit sector, including some members who have worked in national kidney organizations.

New ideas you may see coming from the team include new giving societies for donors; extensive support for Chapters who are looking for best practices on how to hold educational seminars, support groups and fund-raising events; and a second national fundraising event.

The Resource Development Team is also considering holding a national convention again, or joining forces

During fiscal year 2013 (ending June 30) our volun-teers, National Resource Development Committee, Board of Trustees and the Foundation’s staff are committed to raising $6.8 million for the Foundation.

These dollars come in through:

Annual giving including mail appeals, workplace giving, tribute gifts and car donations

Major gifts from individuals and private foundations

Corporate support

Walk for PKD

Chapter fundraising events

with another kidney organization to host a joint con-ference with a PKD-specific track.

One new event the PKD Foundation is planning for in 2013 is a Children and Cystic Kidney Disease Con-vention. Slated for April 5-7, this conference is open to parents of children with PKD (both ADPKD and ARPKD), as well as those with other types of cystic kidney disease. It will be held at Mt. Sinai Hospital in New York City and feature guest speakers, including Lisa Guay-Woodford, MD, a pediatric nephrologist and longtime advisor to the PKD Foundation.

In the coming year donors will receive better com-munication from the Foundation about the differ-ence their support makes and they will learn about creative ways they can give to the Foundation. With the news that our work and their donations have made a difference with tolvaptan, we hope to grow our base of supporters and increase the amount of each gift in order to do more work in accelerating treatments to patients.

MANy WAyS TO GIvE AND FIGHT PKDEvery gift – large or small – makes a big difference. There are many ways to help further the mission to end PKD. We hope one of these options is a good fit for you.

MARK A MILESTONE, like a transplant anniversary, celebrate a special occasion or honor someone’s memory by making a tribute donation.

PARTICIPATE in your employer’s matching gift program.

INCLuDE the PKD Foundation in your will/estate plan.

DONATE your vehicle and designate the funds to the PKD Foundation. Call V-DAC (Vehicle Donations to Any Charity) at 877-999-8322.

BuY A COPY OF THE BRILLIANT EATS COOKBOOK, by renal dietician Kelly Welsh, R.D., C.D., for $10 and get one free (+ $10 shipping). No limit -- for each book you buy, you get one free, and pay only once for shipping. Buy one for yourself; give one as a gift. Visit www.pkdcure.org/brilliant-eats or call 1-800-PKD-CuRE (1-800-753-2873). Proceeds go to PKD Foundation.

Visit www.pkdcure.org/donate/ways-to-donate to learn more about how you can support the PKD Foundation.

After the announcement about tolvaptan, a friend of the PKD Foundation posted on our Face-book page – ‘It's news like this that helps me know we aren't fighting and fundraising in vain!’

The Foundation is extremely grateful for the support of nearly 30,000 people each year through the Walk, local fundraising events and direct donations to the Foundation." – MICHELLE DAVIS

HELP US MEET OUR GOAL OF $6.8 MILLION

We are proud that:

In fiscal year 2012, 79 cents of every dollar raised was spent on programs of research, advocacy, education, support and awareness.

In fiscal year 2013, we’re spending more money on research, as a percentage of our total budget, than ever before.

This means that gifts you make to the PKD Foun-dation go toward mission-critical work to discover treatments and a cure for PKD. To continue funding this work, we need your help. Please come together today to help us reach $6.8 million, and come closer to fulfilling our mission.

Meet Our New Chapter and Fundraising Teams

March 9 – Virtual Educational Seminar Focusing on what is new in PKD research, this seminar will be presented virtually at Chapter loca-tions (recorded seminar will be available during PKD Awareness Week in June). Topics will include the latest news and updates in research, drug repurposing, clinical trials, and tissue donation.

April 5-7 – Cystic Disease in Children Confer-ence in New York City, NY This 1½ day conference is targeted to families that have children with cystic disease (ARPKD or ADPKD). This is an opportunity to learn from specialists in pediatric care, and meet others with shared experiences.

June 16-22 – PKD Awareness WeekWe’re planning some fun virtual activities to increase national awareness about PKD.

June 17-18 – united on the HillOur annual legislative and public policy conference in Washington, D.C. Open to anyone who wants to help advance the PKD legislative agenda with con-gressional representatives. It’s a full day of training and visits on Capitol Hill.

THE PKD FOUNDATION IS GOING THROUGH AN ExCITING TRANSFORMATION THAT INCLUDES THE CREATION OF A NEw RESOURCE DEVELOPMENT TEAM.

Comprised of Development, Chapter Services and Education staff members, the new Resource

Development Team is working in two key areas. Education is charged with providing patients,

families and health professionals with the latest, most relevant information about managing

the disease and living with PKD. Development and Chapter Services are broadening the PKD

Foundation’s base of support and providing the organization with growing resources.

Save the Date for Education March is National Kidney Month – We’ll recognize this throughout the month.

For more information about any of these events, email nicoleh@pkdcure.org

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Leading the way is Chief Development Officer Michelle Davis, who joined the PKD Foundation in April.

According to Davis, the team has a wealth of experience in the non-profit sector, including some members who have worked in national kidney organizations.

New ideas you may see coming from the team include new giving societies for donors; extensive support for Chapters who are looking for best practices on how to hold educational seminars, support groups and fund-raising events; and a second national fundraising event.

The Resource Development Team is also considering holding a national convention again, or joining forces

During fiscal year 2013 (ending June 30) our volun-teers, National Resource Development Committee, Board of Trustees and the Foundation’s staff are committed to raising $6.8 million for the Foundation.

These dollars come in through:

Annual giving including mail appeals, workplace giving, tribute gifts and car donations

Major gifts from individuals and private foundations

Corporate support

Walk for PKD

Chapter fundraising events

with another kidney organization to host a joint con-ference with a PKD-specific track.

One new event the PKD Foundation is planning for in 2013 is a Children and Cystic Kidney Disease Con-vention. Slated for April 5-7, this conference is open to parents of children with PKD (both ADPKD and ARPKD), as well as those with other types of cystic kidney disease. It will be held at Mt. Sinai Hospital in New York City and feature guest speakers, including Lisa Guay-Woodford, MD, a pediatric nephrologist and longtime advisor to the PKD Foundation.

In the coming year donors will receive better com-munication from the Foundation about the differ-ence their support makes and they will learn about creative ways they can give to the Foundation. With the news that our work and their donations have made a difference with tolvaptan, we hope to grow our base of supporters and increase the amount of each gift in order to do more work in accelerating treatments to patients.

MANy WAyS TO GIvE AND FIGHT PKDEvery gift – large or small – makes a big difference. There are many ways to help further the mission to end PKD. We hope one of these options is a good fit for you.

MARK A MILESTONE, like a transplant anniversary, celebrate a special occasion or honor someone’s memory by making a tribute donation.

PARTICIPATE in your employer’s matching gift program.

INCLuDE the PKD Foundation in your will/estate plan.

DONATE your vehicle and designate the funds to the PKD Foundation. Call V-DAC (Vehicle Donations to Any Charity) at 877-999-8322.

BuY A COPY OF THE BRILLIANT EATS COOKBOOK, by renal dietician Kelly Welsh, R.D., C.D., for $10 and get one free (+ $10 shipping). No limit -- for each book you buy, you get one free, and pay only once for shipping. Buy one for yourself; give one as a gift. Visit www.pkdcure.org/brilliant-eats or call 1-800-PKD-CuRE (1-800-753-2873). Proceeds go to PKD Foundation.

Visit www.pkdcure.org/donate/ways-to-donate to learn more about how you can support the PKD Foundation.

After the announcement about tolvaptan, a friend of the PKD Foundation posted on our Face-book page – ‘It's news like this that helps me know we aren't fighting and fundraising in vain!’

The Foundation is extremely grateful for the support of nearly 30,000 people each year through the Walk, local fundraising events and direct donations to the Foundation." – MICHELLE DAVIS

HELP US MEET OUR GOAL OF $6.8 MILLION

We are proud that:

In fiscal year 2012, 79 cents of every dollar raised was spent on programs of research, advocacy, education, support and awareness.

In fiscal year 2013, we’re spending more money on research, as a percentage of our total budget, than ever before.

This means that gifts you make to the PKD Foun-dation go toward mission-critical work to discover treatments and a cure for PKD. To continue funding this work, we need your help. Please come together today to help us reach $6.8 million, and come closer to fulfilling our mission.

Meet Our New Chapter and Fundraising Teams

March 9 – Virtual Educational Seminar Focusing on what is new in PKD research, this seminar will be presented virtually at Chapter loca-tions (recorded seminar will be available during PKD Awareness Week in June). Topics will include the latest news and updates in research, drug repurposing, clinical trials, and tissue donation.

April 5-7 – Cystic Disease in Children Confer-ence in New York City, NY This 1½ day conference is targeted to families that have children with cystic disease (ARPKD or ADPKD). This is an opportunity to learn from specialists in pediatric care, and meet others with shared experiences.

June 16-22 – PKD Awareness WeekWe’re planning some fun virtual activities to increase national awareness about PKD.

June 17-18 – united on the HillOur annual legislative and public policy conference in Washington, D.C. Open to anyone who wants to help advance the PKD legislative agenda with con-gressional representatives. It’s a full day of training and visits on Capitol Hill.

THE PKD FOUNDATION IS GOING THROUGH AN ExCITING TRANSFORMATION THAT INCLUDES THE CREATION OF A NEw RESOURCE DEVELOPMENT TEAM.

Comprised of Development, Chapter Services and Education staff members, the new Resource

Development Team is working in two key areas. Education is charged with providing patients,

families and health professionals with the latest, most relevant information about managing

the disease and living with PKD. Development and Chapter Services are broadening the PKD

Foundation’s base of support and providing the organization with growing resources.

Save the Date for Education March is National Kidney Month – We’ll recognize this throughout the month.

For more information about any of these events, email nicoleh@pkdcure.org

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I recently joined the PKD Foundation as Chief Marketing Officer. What I am thankful for is the opportunity to serve the Foundation, by putting to full use my skills in marketing and communications, and background in health care and not-for-profit organizations. There is great opportunity to raise awareness about PKD and the work of the Foundation. My focus has been collaborating with leadership and others to create an integrated marketing communication plan that will:

SOME OF THE THINGS WE WILL BE DOING INCLUDE:

Increasing our social media presence through a strategic and interactive approach – we hope to see you on Twitter, Facebook, Pinterest and LinkedIn.

Developing online channels, including forums, and a personalized website for those who choose to register. We are also improving the pkdcure.org

A BRAND NEw DAY

FOR MARKETING

Angela ConnellyChief Marketing Officer

Increase awareness to reach new and existing audiences and engage them in supporting the PKD Foundation, thereby furthering the organization’s mission to find treatments and a cure, so that no one suffers the full effects of PKD.

website to make it easier to navigate, more reader-friendly, and a robust resource.

Creating one core blog that will feature weekly postings on a variety of topics. This will still in-clude the popular PKD Will Not Beat Me by Valen Keefer, and Health Notes by Kelly Welsh. We’ll be adding guest bloggers and new segments includ-ing an Inside the PKD Foundation contributed by our leaders.

Taking advantage of low-cost advertising. We’ve al-ready done this with a print ad (see page 13) that ran in Good Housekeeping, Woman’s Day, and Redbook to 280,000 subscribers in Atlanta, Ga., Kansas City, Mo., Las Vegas, Nev., and Little Rock, Ark. The ad will run again soon in Albany, NY, Chi-cago, Ill., Atlanta, Ga., Madison, Wis., and Peoria, Ill (special thanks to Ad Ink for providing this).

We have also placed 20 billboards in Raleigh, N.C., and Boston, Mass., using art similar to the print ad (special thanks to OOH for Good).

Conducting pro-active media relations and public relations activities to increase positive coverage nationally and locally.

And finally, we will improve our communications to you so you are in touch and connected with what is happening at the Foundation and in the PKD Community.

I hope you enjoyed this special issue that celebrates the Foundation’s anniversary and provides a comprehensive look at our programs and recent progress made. Thank you for your support.

HOPE looks like.

Hope for a brighter future, a fuller life - a treatment for the disease

Polycystic kidney disease is a common, but little known life-threatening, genetic disease currently without a treatment or a cure.

Accelerating Treatments to Patients initiative is turning hope into

something more. Something within reach.

Learn more at

pkdcure.org/hope

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I recently joined the PKD Foundation as Chief Marketing Officer. What I am thankful for is the opportunity to serve the Foundation, by putting to full use my skills in marketing and communications, and background in health care and not-for-profit organizations. There is great opportunity to raise awareness about PKD and the work of the Foundation. My focus has been collaborating with leadership and others to create an integrated marketing communication plan that will:

SOME OF THE THINGS WE WILL BE DOING INCLUDE:

Increasing our social media presence through a strategic and interactive approach – we hope to see you on Twitter, Facebook, Pinterest and LinkedIn.

Developing online channels, including forums, and a personalized website for those who choose to register. We are also improving the pkdcure.org

A BRAND NEw DAY

FOR MARKETING

Angela ConnellyChief Marketing Officer

Increase awareness to reach new and existing audiences and engage them in supporting the PKD Foundation, thereby furthering the organization’s mission to find treatments and a cure, so that no one suffers the full effects of PKD.

website to make it easier to navigate, more reader-friendly, and a robust resource.

Creating one core blog that will feature weekly postings on a variety of topics. This will still in-clude the popular PKD Will Not Beat Me by Valen Keefer, and Health Notes by Kelly Welsh. We’ll be adding guest bloggers and new segments includ-ing an Inside the PKD Foundation contributed by our leaders.

Taking advantage of low-cost advertising. We’ve al-ready done this with a print ad (see page 13) that ran in Good Housekeeping, Woman’s Day, and Redbook to 280,000 subscribers in Atlanta, Ga., Kansas City, Mo., Las Vegas, Nev., and Little Rock, Ark. The ad will run again soon in Albany, NY, Chi-cago, Ill., Atlanta, Ga., Madison, Wis., and Peoria, Ill (special thanks to Ad Ink for providing this).

We have also placed 20 billboards in Raleigh, N.C., and Boston, Mass., using art similar to the print ad (special thanks to OOH for Good).

Conducting pro-active media relations and public relations activities to increase positive coverage nationally and locally.

And finally, we will improve our communications to you so you are in touch and connected with what is happening at the Foundation and in the PKD Community.

I hope you enjoyed this special issue that celebrates the Foundation’s anniversary and provides a comprehensive look at our programs and recent progress made. Thank you for your support.

HOPE looks like.

Hope for a brighter future, a fuller life - a treatment for the disease

Polycystic kidney disease is a common, but little known life-threatening, genetic disease currently without a treatment or a cure.

Accelerating Treatments to Patients initiative is turning hope into

something more. Something within reach.

Learn more at

pkdcure.org/hope

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A special thanks to our Board of Trustees and national committees who help the PKD Foundation is so many ways.

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.

Jim Bristol, Ph.D.

Claire Deselle

Don Frail, Ph.D.

Larry Gelbert, Ph.D.

John M. McCall, Ph.D. (Chair)

Jill A. Panetta, Ph.D.

JoAnn Scatina, Ph.D.

Gary G. Godsey, President and CEO

Angela Connelly, Chief Marketing Officer

Michelle Davis, Chief Development Officer

Jill A. Panetta, Ph.D., Chief Scientific Officer

Ray Smith, Chief Financial Officer

Scientific Advisory Committee

Drug Development Advisory Committee

Leadership Team

Sen. Robert Bennett

Bill T. Brazell

Frank Condella (Vice Chair & Treasurer)

Benjamin Cowley, Jr., M.D.

Scott L. Goodman (Chair)

Dr. Lisa Guay-Woodford

Michael A. Haggard, Esq.

Blaise Hazelwood

Walter A. Hunt, Ph.D.

Jerome T. Lienhard, II

John M. McCall, Ph.D.

Richard R. Nelson

Stephanie A. Parker (Secretary)

Julia R. Roberts

Anne Ryan

Hilary Wolfe

Board of Trustees (2012-2013 term)

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