PKD Life - Winter 2020 - 16

POWERING
A CURE
The new ADPKD Patient Registry has myriad
benefits for both researchers and patients.

By Denise Todaro

PKD LIFE * WINTER 2020

diagnosis, family history, medications, symptoms, and the impact PKD
has on their quality of life, as well as
geographic and demographic information. This allows for a more direct
and focused approach to getting the
right clinical trial participants on
board by making it more likely that
they may qualify when the Foundation shares new studies.

PATIENT-CENTERED
APPROACH

The benefits for researchers are
clear, but the Registry is also good
for patients, says Elise Hoover,
the PKD Foundation's director of
research, because it takes the element of chance out of hearing about
relevant trials and puts the control
into patients' hands.
"We know that there's an expertise
that has been missed in research, and
that comes from the patient voice,"
Hoover says. "We want to hear from
patients, because they are the experts
in their own disease."

VANESSA BRANCHI

n September 4, PKD Awareinvestigators' work. It's also a valuness Day, the PKD commuable means of assessing "the burden
nity across the country was
of PKD symptoms in the population,"
out in full force as usual-
he adds. In essence, the Registry
wearing teal, sharing #endPKD posts
opens up a much-needed channel
on social media, and signing up for
of communication and exchange
Walks throughout the US.
between patients and researchers.
But last year's PKD Awareness Day
The ability of investigators, both
had a special significance. It marked
at academic institutions and pharthe launch of the ADPKD Registry,
maceutical companies, to identify
the first patient-centered nationwilling and appropriate study particiwide registry dedicated to and selfpants through the Registry is a major
reported by people with autosomal
change from how they've been idendominant polycystic kidney disease.
tified in the past, says David Baron,
Developed with input
Ph.D., scientific advisor
As of December, at the PKD Foundation.
from clinicians, patients,
and their advocates, the
"What we used to do
nearly 1,300
Registry opens the door
was send out something
people with
for greater strides in
called an ACT, or AccelADPKD had
research into treatments
erating Clinical Trials,
signed up for
for ADPKD.
alert by email," he says.
the Registry
Ronald D. Perrone,
But patients didn't know
M.D., scientific direcfor sure if their current
tor of the Clinical and Translational
health status made them eligible or
Research Center at Tufts Medical
ineligible for a particular trial. "Some
Center in Boston, says one of the
studies want patients who essentially
main advantages of the Registry for
still have normal kidney function,
researchers is that "it allows us to
while others might want people who
identify patients who are willing to be are further along in the course of the
contacted and who might be eligible
disease," Dr. Baron says.
for clinical research trials of new
Then there's the matter of geograpotential treatments for PKD."
phy: As an example, patients in CaliforDr. Perrone's main research focus
nia would get excited about a trial and
is polycystic kidney disease, so he's in then find out it's taking place in Texas.
a unique position to see the Registry's
In the new Registry, patients selfpotential to advance his and other
report basic information, such as their

PKD Life - Winter 2020

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