A dozen or so students
with cystic fibrosis have arrived at Stanford during
the past 20 years. Some of them, like Leslie Hotson,
died before they could graduate. All of them endured
physical and emotional ordeals during college.
Their struggles often involve the most basic human
activities—eating and breathing. CF produces secretions
that plug the lungs, inhibit digestion and make patients
vulnerable to a host of infections. And the disease
carries with it social indignities that can lead to
embarrassment, loss of confidence, even shame.
Richard Moss, professor of pediatrics and director
of the Cystic Fibrosis Center at Lucile Packard Children’s
Hospital, says college-age CF patients take a barrage
of medications and spend one to two hours per day undergoing
respiratory treatments, often using pneumatic vests
that vibrate the chest to loosen mucus.
They are dogged by a heavy, phlegmatic cough that’s
particularly troubling for students living among their
peers. “They’re noisy, they’re bringing
up stuff—it can be very embarrassing for them,”
says Moss. “Some students I’ve known have
requested single rooms because they were worried about
imposing on other people.”
Intestinal dysfunction is the other major problem.
CF secretions prevent digestive enzymes in the pancreas
from reaching the intestines, leading to malnourishment,
chronic bowel difficulty and stunted physical development.
Students with CF can be as much as two years behind
their peers in their sexual maturation, says Moss.
Put together, he says, “That’s a pretty
heavy stew of problems for a kid in college. It’s
hard enough being a Stanford student without having
to deal with all of that.”
Treatment advances in recent years have extended and
improved the lives of CF patients, primarily through
inhaled medicines that thin mucus and keep infections
at bay.
Researchers are attacking the source of the disease
as well as its symptoms. Stanford is conducting experiments
using genes manufactured from recombinant DNA to replace
the malfunctioning gene that leads to CF. These genes,
copies of those that keep a healthy person from developing
CF, are placed inside a harmless viral particle and
administered to the patient via an aerosol cloud. After
inhalation, the viral particles enter cells lining the
airways and spread the genetic payload that counters
the development of CF symptoms.
Moss, whose team of physicians usually plays the role
of primary care provider for students with CF, says
it’s rare nowadays for the illness to prove fatal
for a college-age patient. “Most of the students
we see are reasonably healthy,” he says. “Leslie
[Hotson] was unfortunate to have had very serious lung
disease quite young.”
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