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Robyn J. Barst -- Juggling Roles
of Patient Advocate and Director of Leading Research Center,
Robyn Barst Meets the Challenge
 Great
clinicians often distinguish themselves by fulfilling
several demanding roles at the same time, by not only
establishing their reputation as preeminent caregivers
but also leading their colleagues toward excellence in
other ways—for instance, in patient advocacy and
establishment of referral centers. A case in point is
Robyn J. Barst, MD.
There is her role as a pioneer in the early studies
of epoprostenol in pulmonary hypertension, a time when
she battled for a patient’s right to receive the
best available treatment, persuading the FDA to allow
investigators to use epoprostenol. Then there is the role
Dr Barst played—and continues to enlarge upon—as
Director of the Pulmonary Hypertension Center at New York
Presbyterian Hospital, a center currently serving at least
800 patients of all ages. Regardless of which role occupies
her at the moment—and they frequently overlap—she
has been in the forefront of virtually every significant
advance in the treatment of pulmonary hypertension and
will oversee future research developments as Chair of
the Scientific Leadership Council of the Pulmonary Hypertension
Association.
A pediatric cardiologist by training, Dr Barst has been
Director of the New York Presbyterian Pulmonary Hypertension
Center since 1987. The center’s prodigious growth
over the last two decades has largely been because of
her organizational efforts, her commitment to building
a firstrate facility, and her fund-raising skills in obtaining
the requisite support for its programs. Yet all of it
began on a personal level with Dr Barst’s relationship
with each patient, including the first patient she saw
with primary pulmonary hypertension, a 16-year-old girl
who died on the eve of receiving a lung transplant. “I
was devastated the next day and started reading about
the condition, realizing that there was no treatment for
it. I went into pediatrics because I’ve always believed
that if you make a difference with children you are making
a very long-term difference. They told me at that time
that I would never see another case of primary pulmonary
hypertension because it was so rare.”
Pioneer in the Epoprostenol
Experience
During several years of basic research on the pulmonary
circulation, including two fellowships, one in pulmonary
medicine and one in cardiology, at Columbia University’s
College of Physicians and Surgeons, Dr Barst returned
to her interest in pulmonary hypertension and learned
of the investigative work being done with epoprostenol
for acute vasodilator testing in pulmonary hypertension.
At that time it was not being allowed for use in children;
however, Dr Barst obtained permission
from the FDA to begin using it in children, initially
only at the dosages used in adult patients, 2 to 12 ng/kg/min.
She subsequently persuaded the FDA to allow higher dosages
in children and in 1986 she published the first paper
on its use in a pediatric setting, a report involving
only 9 patients.
Through the support of Burroughs Wellcome, research
began with the long-term use of epoprostenol, enrolling
25 patients in the first long-term intravenous study.
“Prior to using chronic intravenous epoprostenol,
at least 40% of our patients were dying while awaiting
transplantation. We did our first study with epoprostenol
over 8 weeks and at the end of that time we were convinced
that the drug was lifesaving.” The FDA remained
skeptical, however, and wanted more evidence of the drug’s
safety and efficacy in a large, randomized trial.
Enrolling 81 patients, Dr Barst and her colleagues reported
that 8 of 40 patients receiving conventional therapy died
while none of the 41 patients receiving epoprostenol died.“
We had 4 patients at our center who were not randomized
to
epoprostenol and I had to watch those 4 patients die because
they were not on active drug. It was one of the worst
things I had to deal with as a physician— wanting
to give the best clinical care I could yet also wanting
to be aggressive about clinical drug development. I’m
convinced that when we went to the FDA the drug would
not have been approved if it had not been for the survival
benefit.”
From epoprostenol, Dr Barst has moved on to more recent
trials evaluating the use of treprostinil, bosentan, sitaxsentan,
ambrisentan, and iloprost. “One thing we’ve
tried to do at our center, and what makes our center somewhat
different, is that we treat patients of all ages from
newborns to patients in their 80s, thereby giving us the
opportunity to explore the disease process in patients
of all ages.
“We also are involved in as many clinical development
programs as is feasible, thereby giving us firsthand experience
with how patients respond to one drug versus another.”
As she looks back on the clinical trial experience, Dr
Barst has seen significant progress made since the time
when clinicians routinely told patients with a diagnosis
of pulmonary hypertension to “put your affairs in
order.” Today, she says, “we can guarantee
that we can make patients feel better and live longer.”
With a new generation of studies on the horizon, Dr Barst,
as Chair of the Pulmonary Hypertension Association’s
Scientific Leadership Council, looks forward to more advances
in treatment as she and her colleagues evaluate new strategies
involving a more complex and broader spectrum of therapy.
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