PKD Life - Fall 2022 - 23

reabsorbing glucose and
releasing it into the bloodstream.
Instead, the excess
sugar leaves the body via
urine. They've also been
found to be effective at
reducing the risk of hospitalization
and cardiovascular
events in those with heart
failure and at improving
chronic kidney disease even
in those without diabetes.
They have not, however,
been evaluated in people
with ADPKD.
New diabetes
were encouraging, " we didn't
have enough people to draw
a conclusion about efficacy, "
Dr. Watnick says. For that,
they would need hundreds
of participants in a multiyear
study, which is costly. Since
metformin is now a generic
drug, there is no incentive for
a pharmaceutical company
to fund such a trial. However,
an Italian study is comparing
the effects of the PKD drug
tolvaptan with metformin in
people with ADPKD. A larger
study looking at metformin
versus placebo in people with
ADPKD will soon launch
in Australia.
" We're grateful to all of the
participants who took part in
our study, " Dr. Watnick says.
" We rely on the support of the
medications can
improve chronic
kidney disease
even in people
who don't have
diabetes.
PKD community to find new
treatments for our patients. "
INVESTIGATING A
SECOND MEDICATION
The potential for type 2 diabetes
drugs to benefit people
with ADPKD continues with
a soon-to-be-launched study
of empagliflozin (Jardiance),
which is part of a newer
class of diabetes medications
called sodium-glucose
co-transporter 2 (SGLT2)
inhibitors.
These drugs work by preventing
the kidneys from
Some studies in animal
models of ADPKD find significant
benefits with these
medications, including
reduced cysts and improved
markers of kidney disease.
To see if they have the
same effects in humans,
Stephen Seliger, M.D., who
directs the Baltimore PKD
Center Clinical and Translational
Core at the University
of Maryland, and Michel
Chonchol, M.D., who directs
the Polycystic Kidney Disease
Research Program at the University
of Colorado School
of Medicine in Aurora, are
launching a phase 2 clinical
trial this fall, funded by the
National Institutes of Health
and the PKD Foundation. " It's
an early-stage study to look
at the tolerability and safety
of this class of drugs, " Dr.
Seliger says.
" There's no question that
SGLT2 inhibitors have substantial
benefits in terms of
reducing the risk of worsening
kidney function and endstage
kidney disease needing
dialysis or transplantation, "
he says, but he stressed that
those studies were done in
people with other forms of
kidney disease, not ADPKD.
Fifty participants will be
randomly assigned to either
empagliflozin or a placebo
and followed for a year with
frequent on-site and remote
visits as well as MRI scans of
the kidneys. The investigators
will also assess how the
drug affects blood flow in the
arteries and gather information
on the effects of the
drug on patients' quality of
life. " We want to make sure
the drug doesn't make anything
worse for participants, "
Dr. Seliger says.
The study will last for three
years, after which, he says,
" we hope it can be used to
support the design of larger,
longer-term treatment trials
to really demonstrate the efficacy
of this class of drugs in
preventing progression. "
Although both drugs are
on the market for other conditions,
Dr. Watnick stresses
that patients should not ask
their doctors to prescribe
them " off label. "
" We don't know if it works.
It's that simple. You need to
have human trials to really
look at this, " she says. Plus,
every drug has side effects.
In the metformin trial, for
instance, about half of the
participants (including some
in the placebo group) experienced
nausea and other gastrointestinal
symptoms.
To get updates on when
this clinical trial starts
recruiting, sign up for the
PKD Foundation's ACT
Alert program.
*
TO LEARN MORE ABOUT PKD RESEARCH AND SIGN UP FOR ACT ALERTS, VISIT PKDCURE.ORG/RESEARCH
23
http://www.PKDCURE.ORG/RESEARCH
PKD Life - Fall 2022

Table of Contents for the Digital Edition of PKD Life - Fall 2022
