Page 12 - CF Roundtable - Winter/Spring 2026

PEARLS OF WISDOM By: Xan Nowakowski, Ph.D., M.P.H.
TRIALS AND TRIBULATIONS:
BARRIERS TO CLINICAL
RESEARCH PARTICIPATION
Even though I teach research methods
at a medical school, I’ve never
participated in a clinical trial myself—
not for CF-specific drugs or for any other
kind of innovative therapy that might
help with some of my symptoms. Why?
As I often tell my students, participating
in medical research requires more than
just interest. And whether one feels
interested in the first place can depend
on a lot of factors before even getting
into the many external barriers that can
prevent people from getting involved
with trials.
Working with different CF organizations
over the years has shown me the
diversity of perspective in our adult
community surrounding the idea of a
cure. Personally, I don’t focus my own
health care activities around the idea
of one day not having CF anymore.
Although I understand that desire, I
don’t share it. My overarching goal has
always been to live as well as I can
with my CF while still being a person
who has this disease and is shaped
by it. I don’t want to know who I would
be without CF. Moreover, I don’t want
to live in a world “free” of people with
genetic conditions—because that
doesn’t seem very free at all to me.
Family history—going back to how I
joined the world in the first place as
well as the legacies of genocide and
ethnic cleansing in multiple branches
of my ancestry—makes me innately
suspicious of efforts to rid the world
of a given genetic disease. I don’t
look at health conditions that people
live with from birth the same way I
look at infectious diseases like polio
or malaria. Lifelong chronic disease
shapes who we are and who we
become. Whether or not that influence
always makes people better than we
might otherwise have been doesn’t
matter in my view. What does matter
is the message to the world at large
that people with CF and other genetic
diseases are not problems to be
solved. When people suggest that I
should chase a cure for a condition
that is inextricably intertwined with so
much of who I am and what I give back
to the world, I question the motivations
behind that thinking.
That said, my interest in living as well as
I can with my CF certainly invites plenty
of interest in participating in research
when suitable opportunities arise. I say
“suitable” because thus far I’ve only
contributed data to studies that don’t
involve an interventional component.
We often use that term in the clinical
research world to mean “doing
something to address an underlying
issue” like giving someone a drug or
providing them with counseling.
Over the years I’ve contributed
samples of different bodily fluids and
secretions, plus the occasional tissue
biopsy. I’ve also benefited immensely
from certain drugs that my providers
have used off-label as part of my
home care regimen. That term refers
to taking an already-approved drug
for a purpose outside the one for
which it generally gets prescribed.
For example, inhaled corticosteroids
give me a lot of help with controlling
pleuritic inflammation—swelling in the
muscle walls of the lungs—that can
make it much more difficult and painful
to breathe well even when my airways
are fairly clear. Some people with CF
deal with pleurisy more than others.
Off-label use of beclomethasone has
made a massive difference in my daily
quality of life and made it easier to stay
physically active, which in turn helps
to keep my airways clear and free of
infection.
I know quite a few other folks in the
10 percent of adult CF community
members not currently eligible for
CFTR protein modulator drugs who
have likewise benefited strongly from
off-label use of medications initially
developed for other conditions. I also
know several fellow “10 percenters”
who have participated in trials of the
triple combination modulator Trikafta
for people with very rare mutations not
initially included in the eligibility criteria,
12 CF Roundtable Winter/Spring 2026