PKD Life - Summer 2022 - 23

Which brings up the quesdidn't
measure kidney function
as they shrank. " We'd
like to know whether kidney
function actually improves;
whether the filtration rate
can also increase as the kidneys
get smaller, " he says.
Dr. Somlo also wants to
The findings
highlight the
extraordinary
discovery that
the kidney
can adapt
and change
to differing
environments.
says. That's because it's only
recently that researchers had
simple, affordable tools they
could use to create genetic
diseases like ADPKD in mammalian
animal models like
mice. Another surprising
thing, Dr. Somlo says, is that
a single protein can drive the
significant remodeling they
saw in the mouse kidney.
His lab is now exploring
those and other questions.
For instance, while the MRIs
on the mice showed the kidneys
got smaller, the team
know what happens when
the gene is knocked out in a
more random fashion, which
is how it happens in humans,
rather than completely
turned off as in this experiment.
" Can you knock out a
cell here, a cell there, and still
get the same results? "
Another big question is
what immediate molecular
changes occur in kidney cells
when they once again start
receiving polycystin. Understanding
those mechanisms,
he says, is key to identifying
targets for drug therapy.
tion everyone wants to know:
When can we expect to see
clinical applications of this
very basic research? Not for
a long time, Dr. Somlo says.
However, notes PKD Foundation
Chief Research Officer
Chris Rusconi, Ph.D., " overall,
findings provide hope that
restoring polycystin function,
or 'reparative remodeling,'
is a viable approach to treating
ADPKD. "
In addition, the research
seems to have galvanized
the nephrology community,
with much interest from the
pharmaceutical industry
as well as other academic
centers, which could speed
the research.
" An unexpected finding
like this tells us we have a lot
more work to do, " Dr. Somlo
says. But it also " tells us that
the therapy is very possible
and we should look harder. "
The PKD Foundation will
continue funding ongoing
research supporting this and
other discoveries.
*
TO LEARN MORE ABOUT PKD RESEARCH, VISIT PKDCURE.ORG/RESEARCH
23
ILLUSTRATION BY MI-VIRI/GETTY IMAGES. RIGHT: LUIS ALVAREZ/GETTY IMAGES.
http://www.PKDCURE.ORG/RESEARCH
PKD Life - Summer 2022

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