-
new PKD research grants awarded Learn what four of the
researchers are focused on and why they’ve dedicated their careers
to PKD.
Page 4
READ MORE ONLINE!pkdcure.org/pkd-progress
ProgressPKD
Spring 2016
A magazine to inform and educate PKD patients and families,
Foundation supporters, health professionals and researchers.
Page 7
Grant awardee Dr. Stephen Parnell is dedicated to ending PKD for
his family.
15
-
Leadership Letter
As CEO of the PKD Foundation, I’ve had the unique privilege of
meeting and working with incredible scientists who are doggedly
pursuing an end to PKD. I’m consistently impressed with how
talented they are, and dedicated to PKD.
The Foundation is the common thread that binds them. Promising
projects that could lead to therapies can begin and continue
because of Foundation funding. David Baron, Ph.D, PKD Foundation
Chief Scientifi c Offi cer (CSO), talks about how this is a time of
breakthroughs in PKD science (see page 10). I’m proud to be a part
of the group making much of that work possible.
There are now more than 30 PKD Foundation-funded projects
underway. Without the Foundation’s fi nancial support, this work
might not be possible. In this issue you’ll read about research
grants recently awarded, and hear from four of the scientists
leading the projects. They all share the same sentiment that
funding from the Foundation is critical in moving PKD science
forward.
This round of grants includes work focused on autosomal
recessive PKD (ARPKD) and polycystic liver disease (PLD), and
explores the use of the latest technology.
In the last issue of Progress we talked about fi ve amazing new
fellows we are funding (see pkdcure.org/researchfellowships). We
also recently partnered with the American Society of Nephrology
(ASN) Foundation for Kidney Research to fund a fellowship in
perpetuity. Our $500,000 commitment was matched by $1.5 million
from the ASN, tripling our investment and potential in attracting
new talent to PKD research through the Jared J. Grantham Research
Fellowship.
Funding of grants and fellowships supports the momentum of PKD
science by providing resources and support to scientists when they
need it most.
I hope you’re as excited as I am in celebrating such a vibrant,
committed group of researchers focused on fi nding treatments for
PKD.
2
Jackie Hancock Jr., CFREPKD Foundation CEO
READ MORELearn about the ASN Fellowship program at
pkdcure.org/news/jared-grantham-fellowship.
Follow Jackie on Twitter! @jackiehancock
LEA
DE
RSH
IP
Board of Trustees (2015-2016 term)Benjamin D. Cowley Jr., M.D.
(Chair)Frank Condella Jr.(Immediate Past Chair, Vice Chair)Fran
Towey (Treasurer)Anne Ryan (Secretary)Paul T. ConwayLisa
Guay-Woodford, M.D.Michele KarlKlee KleberAndrea KringsteinBrock
NelsonDwight OdlandCraig RamseyerJulia RobertsJeff rey RonaTerry
Watnick, M.D.Jerry Waxman
Scientifi c Advisory CommitteeTerry Watnick, M.D.
(Chair)Benjamin D. Cowley Jr., M.D.(Past Chair)Stefan Somlo, M.D.
(Vice Chair)John Bissler, M.D.Alessandra Boletta, Ph.D.Iain
Drummond, Ph.D.Michal Mrug, M.D.York Pei, M.D.Ronald Perrone,
M.D.Richard Sandford, M.D., Ph.D, FRCPDarren Wallace, Ph.D.Angela
Wandinger-Ness, Ph.D.
Leadership TeamJackie D. Hancock, Jr., CFRE,Chief Executive Offi
cer David Baron, Ph.D., Chief Scientifi c Offi cerAngela Connelly,
MBA,Chief Marketing and Communications Offi cerRay Smith, CPA,
MBA,Chief Operating Offi cer and Chief Financial Offi cer
-
3
A consistent theme heard from PKD researchers is that there is
no single source of funding for PKD reseach. Often, early support
from the PKD Foundation provides seed money for new projects to
start, or enables projects initially funded by the Foundation to
gain funding by the National Institutes of Health (NIH).
Thanks to a grant from the PKD Foundation in 2014, Vishal Patel
has been able to sustain his research initially started with
funding by the NIH. Dr. Patel is one of the recipients of 15 PKD
Foundation research grants awarded in 2014. He received a two-year,
$160,000 award to pursue his project on the “Role of miR-17~92 in
the Pathogenesis of PKD.”
The timing was crucial for Dr. Patel, whose initial NIH award
funding was in its last year. He’d recently published a seminal
paper in the scientific journal, United States, Proceedings of the
National Academy of Sciences of the United States demonstrating his
lab’s success in inactivating microRNA to inhibit cyst growth in
mouse models and identifying it as a drug research target for
autosomal dominant polycystic kidney disease (ADPKD) in humans.
The PKD Foundation grant helped to sustain momentum on research
used as preliminary data in applications to NIH to further explore
his findings.
“This was an exciting but very early idea – one that the NIH
likely would not have funded,” says Dr. Patel. “I’m grateful the
PKD Foundation
found the idea interesting enough to fund. That support could
not have come at a better time.”
Thanks in part to the PKD Foundation grant, Dr. Patel received
renewed NIH funding. He continues to publish important findings in
major scientific journals, and he’s established a collaborative
partnership with a biotech company to develop microRNA-based drugs
for PKD.
“I’m drawn to PKD research because there is a sense of optimism
that a therapeutic breakthrough is near.”
Fast-paced discoveries and access to inspiring mentors are what
initially attracted Dr. Patel to PKD research. One of those mentors
is Peter Igarashi, M.D., recipient of the Lillian Jean Kaplan
International Prize for Advancement in the Understanding of PKD.
The award was established by the PKD Foundation and the
International Society of Nephrology through the generosity of
Thomas S. Kaplan and Daphne Recanati Kaplan of New York, in memory
of Tom’s mother, Lillian Jean Kaplan, who had PKD and died in
2002.
Dr. Patel joined Dr. Igarashi’s lab at the University of Texas
Southwestern Medical Center in Dallas in 2005. In 2010 he
established his own lab at UT Southwestern, where he mentors
up-and-coming PKD physician-scientists, and is active in efforts to
launch a comprehensive PKD clinic at the medical center - a
potential site for future clinical trials. n
PKD Foundation funding keeps momentum going on promising
research
Dr. Patel (middle) and his daughter Aarna, with North Texas
Chapter Coordinators Sally Wilson (left) and Carolyn Morris
(right).
EDUCATION AND CAREER HIGHLIGHTS• P.S. Medical College, India
(medical school)
• Northwestern University, Evanston Hospital, (Internal Medicine
Residency)
• University of Texas Southwestern Medical Center (Nephrology
fellowship)
• University of Texas Southwestern Medical Center (Basic
research fellowship)
• PKD Foundation Research Grant Recipient
• Rising star in Nephrology - State of Texas, Super Doctors
• National Institute of Diabetes and Digestive and Kidney
Diseases - Career Development Award
• University of Texas Southwestern O’Brien Center - Pilot and
Feasibility Grant
• Chief Fellow, University of Texas Southwestern Medical
Center
RE
SEA
RC
H
“I’m grateful the PKD Foundation found the idea interesting
enough to fund. That support could not have come at a better
time.”
-
44
New PKD Research Grants AwardedR
ESE
AR
CH
The PKD Foundation recently awarded grants to 15 outstanding PKD
researchers. The Foundation’s Scientifi c Advisory Committee (SAC)
reviewed 51 research grant applications to determine which ones
would merit funding; a total of $2.4 million over the next two
years. This investment will propel critical research to broaden our
understanding of the genetic and pathological processes involved in
PKD and to accelerate the development of potential therapies for
PKD patients.
David Baron, Ph.D., PKD Foundation Chief Scientifi c Offi cer,
said “I am particularly excited about the quality and scientifi c
diversity of grants this year.” Grant topics include basic
mechanisms of ADPKD and ARPKD, that provides the foundation for
advancements, and new
clinical approaches. Three specifi cally focus on ARPKD, and two
focus on polycystic liver disease (PLD).
We interviewed four awardees about what they hope to accomplish,
and what this funding means to their work and PKD science. While
these researchers come from diff erent backgrounds, and study diff
erent aspects of the disease, they’re all focused on one thing --
ending PKD. n
Grant awardees Stephen Parnell, Ph.D., and Xia Zhou, Ph.D., at
work in the laboratory at the University of Kansas Medical
Center.
READ THE FULL SUMMARIES of all grants awarded at
pkdcure.org/grants
Meet the researchers at the PKD National Convention. Grant
awardees Dr. Dell, Dr. Parnell and Dr. Freedman will be there to
answer questions. Register at pkdcure.org/convention
research grants
Awarded
2 3$80,000 per grant per year
related to PLD & Related to ARPKD
151 in Canada
13 in
th
e U.
S.
1 in Europe
-
New PKD Research Grants Awarded
David Beier, M.D., Ph.D.Seattle Children’s Research Institute –
Seattle
Screening for Modifi ers of PKD Severity Using ENU Mutagenesis
(The Dan Larson Research Grant)
Sorin Fedeles, M.S., Ph.D.Yale School of Medicine – New Haven,
CT
Examining the Role of XBP1 in the Pathogenesis of Protein
Folding-associated Polycystic Kidney Disorders
Brunella Franco, M.D.Telethon Institute of Genetics and Medicine
– Naples, Italy
The Role of Autophagy in Renal Cystogenesis
Benjamin Freedman, Ph.D.University of Washington – Seattle
Modeling Human PKD Cystogenesis with Pluripotent Stem Cells
Edward Inscho, Ph.D.University of Alabama at Birmingham
–Birmingham, AL
Renal Vascular Function in ARPKD
Timothy Kline, Ph.D.Mayo Clinic – Rochester, MN
Development of a Computer-aided Decision Support System for
PKD
Tetyana Masyuk, Ph.D.Mayo Clinic – Rochester, MN
Deregulated Cholangiocyte Autophagy: A New Target for Polycystic
Liver Disease (The Eileen Creamer O’Neill Award)
Stephen Parnell, Ph.D.University of Kansas Medical Center –
Kansas City, KS
Polycystin-1 Mediated Cyst Regression
York Pei, M.D.Toronto General Hospital – Toronto, Canada
Genetic Modifi ers of Severe Polycystic Liver Disease (Grant
co-funded by the PKD Foundation of Canada)
Feng Qian, Ph.D.University of Maryland School of Medicine –
Baltimore
Role of Fibrocystin/Polyductin in Health and ARPKD
Edward Skolnik, M.D.New York University Medical Center – New
York
A Novel Screen to Identify Kinasis that Are Activated in PKD
Kidneys
Zhaoxia Sun, Ph.D.Yale School of Medicine – New Haven, CT
Vascular Integrity in Zebrafi sh PKD Models
Bradley Yoder, Ph.D.University of Alabama at Birmingham –
Birmingham, AL
Injury Response Mediated Pathogenesis in Ciliopathies
Xia Zhou, M.D., Ph.D.University of Kansas Medical Center –
Kansas City, KS
Functional Roles of DNMT1 in Autosomal Dominant Polycystic
Kidney Disease
5
Dr. Parnell, Dr. Zhou and research assistant Brittany Crain look
at cystic kidney samples.
2016 PKD Research Grants
Dr. Vincent H. Gattone, Research Award for the Top-rated Grant
ProposalKatherine M. Dell, M.D.Case Western Reserve University –
Cleveland
Magnetic Resonance Fingerprinting (MRF) to Assess ARPKD Kidney
and Liver Disease Progression
Grant Awardees Grant Title
-
6
RE
SEA
RC
H
Katherine M. Dell, M.D.Case Western Reserve UniversityVincent H.
Gattone Research Award for the Top-rated grant proposal ; Magnetic
Resonance Fingerprinting (MRF) to Assess ARPKD Kidney and Liver
Disease Progression
Several new therapies have shown promise in ARPKD animal models,
however, they haven’t been studied in patients because there are no
clinically-available, reliable, non-invasive ways to measure the
effects of treatment. Magnetic resonance imaging (MRI) may provide
this key missing piece, but MRI studies are long and require
patients to sit still, which is not possible for young children. A
normal MRI can take an hour and a half and, when performed in
children, often requires sedation, introducing additional
risks.
Dr. Dell’s research aims to establish a new method, called
magnetic resonance fingerprinting (MRF), as a non-invasive and
faster way to get the information needed for clinical studies. With
MRF, the scanner can take multiple images simultaneously rather
than sequentially, which makes the procedure a lot faster.
Ultimately, Dr. Dell hopes that the safe and non-invasive nature of
MRF will lead to more clinical trials with ARPKD patients of all
ages.
Dr. Dell has dedicated her career of over 20 years to ARPKD
because of the connection to her patients. “What first made me
interested in the disease was intellectual curiosity, but what has
continued to inform my decisions about what I want to research are
the patients.
Parents are looking for a treatment. Having people pay
attention, and knowing they are looking for therapies and trying to
help matters a lot.”
In addition to being a practitioner, Dr. Dell is also a clinical
researcher, so she knows what is needed for clinical trials. “The
questions in the lab have come out of the questions in the clinic
(working with the children and their parents). That’s really what
has driven me.”
With her perspective as a scientist and previous Scientific
Advisory Council (SAC) member, Dr. Dell knows it is incredibly
important that the PKD Foundation continues to fund research
grants. “Disease specific foundations play an important role
because … they recognize the importance of these projects and may
be more willing to take risks for novel ideas that may not work but
would be amazing if they did.”
She became interested in genetic and renal disease, including
ARPKD, during her pediatric clinical rotation when she was a fellow
at Children’s Hospital of Philadelphia. Her Division chief and
medical director was Bernard Kaplan, M.D., who conducted early
research in ARPKD, and is one of the pioneers of PKD research.
“We’re making progress. I’m very excited about this grant
because I feel recognition of some of the work we’ve done already
and, importantly, that what we’re doing matters.”
INTERESTING FACTS
• Three grants awarded will focus on autosomal recessive
polycystic kidney disease (ARPKD), a rare form of PKD that affects
approximately 1 in 20,000 infants.
• Dr. Dell’s research will focus on ARPKD and liver fibrosis, a
significant related aspect of ARPKD.
• Dr. Dell was a member of the Foundation’s Scientific Advisory
Committee (SAC) from 2002 to 2006
-
7
Stephen Parnell, Ph.D.University of Kansas Medical Center
Polycystin-1 Mediated Cyst Regression
When Dr. Parnell goes to a family event, they ask him how work
is with a different intensity. Dr. Parnell’s grandfather died from
PKD before dialysis was widely available, and he passed it to all
three of his sons – Dr. Parnell’s father and uncles. One uncle
lived for many years after a transplant, but the other died at 48
due to PKD-related complications. His father, who also received a
transplant, is the only member of his family with PKD to live
beyond 80. Dr. Parnell is researching to find treatments for his
own family’s disease.
By studying a protein called polycystin-1, Dr. Parnell hopes to
unlock the secrets of what it does and why renal cysts form when
you lose it. To do this, he engineered a mouse that develops PKD
due to deficient polycystin-1 that can be turned back on (called
re-expression). He will study what happens when it’s re-expressed,
to determine if a cyst has the ability to get smaller or even
disappear. Most current studies are aimed at slowing cyst growth,
but this project suggests we might be able to actually reverse
it.
Serendipity brought Dr. Parnell into the PKD field and to the
Kidney Institute at the University of Kansas
Medical Center (KUMC). He wanted to pursue a Ph.D. in
biochemistry, and having grown up in Topeka, he called the KUMC. He
ended up on the phone with his future graduate student mentor,
James Calvet M.D., who would later become a recipient of the
Lillian Jean Kaplan International Prize for Advancement in the
Understanding of PKD. When Dr. Calvet commented that he worked on
“...a disease that you’ve probably never heard of, PKD...” Dr.
Parnell immediately knew that’s where he wanted to study.
Dr. Parnell’s postdoctoral studies took him to other areas of
scientific interest, but a fellowship through the Foundation
allowed him to get back into PKD research. “I would not have been
able to get back into the PKD field without that fellowship from
the Foundation. Without it, I might have been forced into a
different field.”
Funding is essential for projects like Dr. Parnell’s to
continue. “We are not working on an easy problem, and the work is
expensive. It really helps to have these funds to make the work go
faster, get the right materials, and have another set of hands to
help. It’s absolutely vital, I can’t state that enough.”
“It’s really quite amazing the pace of progress in PKD compared
to other diseases. I think that is because there are very dedicated
people who are supportive of the PKD Foundation, and equally
dedicated people who have invested their lives in solving the
problem of PKD.”
INTERESTING FACTS
• Dr. Parnell has multiple family members who have PKD,
including his father.
• When Dr. Parnell was a young boy, his father was sent to KU
Medical Center to visit the world’s preeminent PKD scholar and
co-founder of the PKD Foundation, Dr. Jared Grantham.
• Years later, Darrell Parnell received his kidney transplant at
KU Medical Center while Dr. Parnell was there doing his Ph.D.
research on PKD.
Dr. Parnell’s grandfather (left), uncles, and father (right)
Katherine M. Dell, M.D.Case Western Reserve UniversityVincent H.
Gattone Research Award for the Top-rated grant proposal ; Magnetic
Resonance Fingerprinting (MRF) to Assess ARPKD Kidney and Liver
Disease Progression
-
8
RE
SEA
RC
H
Benjamin Freedman, Ph.D.University of WashingtonModeling Human
PKD Cystogenesis With Pluripotent Stem CellsAs Dr. Freedman
graduated with his Ph.D. from the University of California
Berkeley, he became more aware of people with medical problems and
wanted to use his knowledge to help them. He asked his mother what
he should focus on, and she reminded him they had friends with PKD.
That’s how his path as a PKD researcher started.
Dr. Freedman is approaching PKD from a different angle. He’s
focused on generating new kidney replacement tissue from a
patient’s own cells that can be studied for root causes of the
disease and used to test drug candidates for future clinical
trials. Ultimately, he hopes to produce an entirely new kidney.
His project will take advantage of stem cell technology that has
only been invented in the past 10 years. In 2007, scientists
discovered they could take skin cells from adult patients and
essentially turn back the clock to a very early stage, turning the
adult cells into stem cells that can develop into any type of
tissue. Dr. Freedman made such stem cells from PKD patients with
the idea of studying them and using them for regeneration.
Dr. Freedman’s first major breakthrough was when he and several
other researchers first turned stem cells into actual kidney cells,
something that had never been done before. “People had made heart,
liver, and other types of cells out of stem cells but nobody had
ever
actually made kidney tissue. When we figured out how to do this,
the tissue that formed was organized into structured mini-kidney
units – or organoids – that had pieces of kidney tubule in a
specific order. It looked like what you would see in an actual
kidney. That was a thrilling discovery.”
Soon after this discovery, Dr. Freedman did the same thing with
stem cells carrying mutations that cause PKD. Remarkably, in this
case, the ‘mini-PKD’ organoids formed cysts, which looked like
large balloons in the cultures. These cysts were only observed in
the mini-PKD organoids, but not in organoids that lacked PKD
mutations. “That was a very exciting series of discoveries. We’re
at the tip of the iceberg in terms of what we can do with these
cells.”
Dr. Freedman is grateful for the PKD Foundation grant. “The
grant will give us the opportunity to turn this into something that
can be used to find new therapies. He also appreciates the role of
the Foundation beyond funding. “The PKD Foundation is a source of
connection to the PKD world, to the patients and other PKD
researchers. As a young person relatively early in my career, I’m
very thankful for the support and the opportunity to connect to the
Foundation at a deeper level than I had before.”
“I’m excited to see if we can learn what components are most
essential to cyst growth, then translate that into potential
therapies.”
INTERESTING FACTS
• Dr. Freedman spoke about new gene-editing technology called
CRISPR and how it could transform the future of PKD care. Hear the
recording at pkdcure.org/newgeneediting.
• Dr. Freedman wrote a PKD Connection blog entry about
mini-kidney organoids. Read it at
pkdconnection.org/research/the-pkd-research-experience.
YOU CAN HELP FUND RESEARCHOver the next two years, the PKD
Foundation will spend $2.4 million to fund critical research that
will help find treatments for PKD. To learn how you can fully fund
a research grant, which can be named after you or a loved one,
email Jackie Hancock, CEO, [email protected].
-
9
York Pei, M.D.Toronto General Hospital Genetic Modifiers of
Severe Polycystic Liver Disease (sPLD)
Most people affected by PKD understand how kidneys can grow so
big they may affect an individual’s ability to move, eat, and
sometimes even breathe. However, many may not know that some people
with PKD have a rare complication called severe polycystic liver
disease (sPLD), which may cause their liver to grow four to eight
times larger than the size of a healthy kidney. People with sPLD
experience symptoms described as a “mass effect,” which may include
feeling full after a small meal, shortness of breath, and abdominal
pain and swelling. The treatment for sPLD is very limited, and many
patients require a liver transplant because the mass effect impact
on their health and quality of life.
Dr. Pei has seen many patients with PKD over the past 20 years
while running kidney disease programs, and he knew there was little
to be done for those with sPLD. He saw an opportunity to use his
genetics training with new technology in
studying the gene. Dr. Pei’s project is focused on identifying
the genetic factors behind sPLD, with hopes it will lead to studies
in animal models and new targets for therapies.
Funding from the PKD Foundation will allow Dr. Pei to use new
technology called Next Generation Sequencing, and study a large
enough group of people to substantiate findings. “We have
experience with this technology but it’s expensive. Most of the
grant will fund the cost of doing this kind of sequencing in a
large enough number of patients from multiple international centers
to find the variance that may be underpinning sPLD.”
Dr. Pei has worked with PKD patients as a physician and
researcher for most of his career. “It’s becoming a life pursuit to
think about PKD and how to treat PKD because of my close
association both as a physician and a researcher.”
“As physicians we are somewhat limited and for a long time we
had nothing to offer. We are entering a different era in which much
of the research done by others in the PKD field is coming to
fruition in the way of tools and therapies.”
INTERESTING FACTS
• Dr. Pei co-edited the new PKD Patient Handbook. To get your
copy visit pkdcure.org/handbook.
• Two of the grant projects will focus on polycystic liver
disease (PLD). Dr. Pei’s work will focus on understanding the
genetic factors that lead to severe PLD, a rare complication of
ADPKD.
• Dr. Pei is a member of the PKD Foundation’s Scientific
Advisory Committee (SAC). (He was not involved in the grant review
process).
Fifty percent of Dr. Pei’s research will be funded by the PKD
Foundation of Canada (PKDFOC). The PKDFOC has committed $40,000 per
year for the next two years. Dr. Pei observes that “The PKDFOC has
significantly grown in helping advocate for patients, raise money
and support research. It’s a natural collaboration.”
-
1010
Collaboration with the Kidney Health Initiative (KHI)
When KHI was launched in 2012 to foster therapy development for
kidney diseases, it was only natural for the PKD Foundation to
participate.
KHI, led by the American Society of Nephrology (ASN) and the
Food and Drug Administration (FDA), brings together key kidney
health stakeholders. These include health professionals, patient
groups, pharmaceutical companies, device manufacturers, academic
research, and governmental and non-profi t organizations. The
common goal is to achieve breakthroughs in kidney disease
treatment.
According to former FDA Commissioner, Margaret Hamburg, M.D.,
the statistics of kidney disease are “catastrophic.” One in nine
Americans has chronic kidney disease (CKD) and about 600,000 are in
end- stage renal disease (ESRD). Kidney disease kills more people
than other diseases such as breast and prostate cancer, which get
far more attention. The cost of treating CKD and ESRD is
enormous.
ADPKD and ARPKD represent eight to 10 percent of people with
ESRD, which requires a transplant or dialysis. In the long run,
dialysis is more costly than a transplant for patients and
Medicare. Better treatments and more living donors could save
Medicare a billion dollars a year or more. KHI aims to tackle this
gap in treatments of CKD and ESRD.
Last May I presented at the KHI Third Annual Stakeholders
Meeting, on “Achieving Consensus on Clinical Trial Biomarkers and
Endpoints to Facilitate the Development of Treatments of ADPKD
Early in the Course of the Disease.” This is because treating PKD
early, when kidney function is still relatively normal, is more
likely to be eff ective at preserving normal kidney tissue by
slowing or stopping cyst growth. Treatment in later stages, when
cysts are already very large, may pose a greater challenge to
preserving the small amount of normal kidney tissue remaining.
Through this partnership, and largely the eff orts of Ronald
Perrone, M.D., at Tufts University, who serves on the Scientifi c
Advisory Committee of the PKD Foundation, Total Kidney Volume (TKV)
was established
as a prognastic biomarker by the FDA. This allows clinical
researchers to study patients more likely to respond to treatments.
This is critical because PKD kidneys get much larger than normal, a
characteristic unique to PKD in contrast to other renal diseases.
Our continuing work with KHI is vital to expanding use of the TKV
biomarker earlier in the course of PKD that will serve as an
indicator of the success of early therapeutic interventions.
The time is ripe for breakthrough therapies. We hope the results
of this collaboration will lead to understanding of the unique
needs of PKD patients, and encourage pharmaceutical companies to
develop new therapies. n
1.800.562.3974 www.otsuka-us.comOtsuka America Pharmaceutical,
Inc.Otsuka Pharmaceutical Development & Commercialization,
Inc.Otsuka Maryland Medicinal Laboratories, Inc.
© 2015 Otsuka America Pharmaceutical, Inc.
And indeed, for over 85 years, Otsuka’s people have achieved
major milestones in their quest to create new products for better
health. Otsuka is hard at work investigating potential new
treatments, with numerous compounds in various stages of
development to treat disorders of the cardiovascular,
gastrointestinal, respiratory, renal, and central nervous systems,
and to treat cancer and ophthalmic disorders. We’ve funded new
research, supported new clinical trials, and pursued the
development of new medications – an unfaltering commitment of
energy and resources with one goal in mind – to create new products
for better health worldwide.
People creating new products for better health worldwide
The name “Otsuka” translates to
“major milestone.”
AW
AR
EN
ESS David Baron, Ph.D.
Chief Scientifi c Offi cer (CSO)
LISTEN TO DR. BARON talk about Total Kidney Volume and PKD
through the recorded webinarpkdcure.org/webinarwednesdays
-
11
JUNE 24 TO 26DISNEY’S CORONADO SPRINGS RESORT Collaboration with
the
Kidney Health Initiative (KHI)
1.800.562.3974 www.otsuka-us.comOtsuka America Pharmaceutical,
Inc.Otsuka Pharmaceutical Development & Commercialization,
Inc.Otsuka Maryland Medicinal Laboratories, Inc.
© 2015 Otsuka America Pharmaceutical, Inc.
And indeed, for over 85 years, Otsuka’s people have achieved
major milestones in their quest to create new products for better
health. Otsuka is hard at work investigating potential new
treatments, with numerous compounds in various stages of
development to treat disorders of the cardiovascular,
gastrointestinal, respiratory, renal, and central nervous systems,
and to treat cancer and ophthalmic disorders. We’ve funded new
research, supported new clinical trials, and pursued the
development of new medications – an unfaltering commitment of
energy and resources with one goal in mind – to create new products
for better health worldwide.
People creating new products for better health worldwide
The name “Otsuka” translates to
“major milestone.”
AW
AR
EN
ESS
Shannon Clark, a retired Mississippi trial judge, and the PKD
Foundation are both benefi ting from a tax law that President Obama
recently reinstated, allowing donors to make tax-free gifts from
their IRAs. You may now move up to $100,000 from your IRA directly
to a qualifi ed charity like the PKD Foundation without having to
pay income taxes on it. This applies for all of 2015 and remains in
eff ect for 2016 and beyond.
Shannon took advantage of this by making a $10,000 IRA rollover
donation to the PKD Foundation ($5,000 in 2015 and $5,000 for the
2016 tax year). His late wife, Kathryn, had a transplant and was on
dialysis for many years due to PKD, and one of their three sons has
PKD. Shannon believes his support will make an impact. “Our family
knows the problems with PKD fi rst-hand. In my mind, one of the
surest
methods to fi nd a cure or slow the progression of PKD is by fi
nancially supporting the research programs funded by the
Foundation.”
If you have already made a qualifying gift in 2015, contact your
advisor to make sure your gift is properly reported on your 2015
income tax returns. To learn more, email Jackie Hancock, Jr.,CEO,
[email protected]. n
In essence, the law states: If you are 70½ or older, you can
give up to $100,000 directly from your IRA to charities such as the
PKD Foundation.
The transfer generates neither taxable income nor a tax
deduction, so you still benefi t even if you do not itemize your
tax deductions.
If you have not taken your required minimum distribution for the
year, your IRA charitable rollover gift can satisfy all or part of
that requirement.
The transfer may be made in addition to any other charitable
giving you have planned.
Tax law change opens new IRA donation options
This summer, the PKD community will gather for three days of
learning, building connections and sharing progress in PKD
research. PKD patients and caregivers will hear from PKD experts on
topics ranging from how to manage the disease, emotional health,
dialysis, transplantation and more.
Specifi c sessions have been developed:Teens and young adults
will meet and connect with others their age who live with PKD.
Experts will present information in age-appropriate conversations,
while providing support and guidance on how teens and young adults
can take an active role in their health care.
Parents of children who have ARPKD and ADPKD will fi nd an
intimate setting to learn, connect and support one another.
Pediatric PKD experts will discuss topics specifi c to children and
teens who have PKD.
Other highlights:• Celebration dinner keynote speaker Shuvo Roy,
M.D., will talk about his work on developing an artifi cial
implantable kidney for end-stage renal disease patients.
• Saintsations cheerleader and Foundation supporter Kriste Lewis
will be the emcee for the Convention.
• Grant recipients Dr. Dell, Dr. Parnell and Dr. Freedman
(featured on pages 4-9) will be there to answeryour questions. Be
sure to meet them!
See the full agenda and register at pkdcure.org/convention
PKD National Convention
-
Please send your contact information updates or notify us of
duplicate mailings of this publication by contacting
[email protected] or 1.800.PKD.CURE (753.2873), ext. 187.
The mission of the PKD Foundation is to promote programs of
research, advocacy, education, support and awareness in order to
discover treatments and a cure for polycystic kidney disease and
improve the lives of all it affects.
8330 Ward Parkway, Suite 510Kansas City, Missouri 64114
Every year thousands of people walk to end PKD in more than 50
cities nationwide. Be one of the 10,000 walkers to help fund
research toward therapies and a cure, and provide education and
support to the hundreds of thousands of families affected by
PKD.
REGISTRATION OPENS MAY 3Visit walkforpkd.org to register, start
a team, and learn how you can be the difference.
NONPROFIT ORGU S POSTAGE
PAIDKANSAS CITY, MOPERMIT NO. 2344
A cure is our finish line
facebook.com/pkdfoundation @pkdfoundation
pkdcure.org | [email protected] | 1.800.753.2873DISNEY’S
CORONADO SPRINGS Resort, Lake Buena Vista, Florida Why attend the
PKD National Convention:n Learn how to manage PKD through all
stages of the
disease—from diagnosis to life after a transplant.
n Meet, connect and share experiences with others in the PKD
community.
n Hear the latest in research from PKD experts.
n Choose from more than 30 sessions, including sessions for
teens and young adults who have PKD, parents of children with ARPKD
and ADPKD and caregivers.
n Have a blast at the Walt Disney World® Theme Parks. Discounted
tickets are available with registration.
ORLANDOJUNE 24 TO 26, 2016
SEE THE FULL AGENDA AND REGISTER AT pkdcure.org/convention