Robert C. Stern, M.D., Rainbow Babies and Children's Hospital, Cleveland, Ohio
Fall 1997
If there was one significant message that permeated Dr. Stern's talk, it
was that we should all question assumptions when reading CF clinical study results,
medical reports, medical discussions on-line or even when given verbal advice (either lay
or medical) about cystic fibrosis treatment.
Research into cystic fibrosis, whether basic or clinical, must follow strict design and
analysis procedures. Even still, Dr. Stern believes that clinical research has additional
pitfalls and problems beyond those of basic research that must be faced. This includes the
physical risk to human subjects, the ability of humans (whether physicians or subjects) to
undermine a study's design, or the career and/or legal risks associated with unexpected
catastrophic results. Additionally, many other medical issues facing people with CF need
more attention by clinical researchers. Many of these issues indirectly affect life
expectancy and/or quality of life, such as the substantial disagreement among physicians
and CF centers concerning certain aspects of treatment. For these reasons, he urges us to
examine research carefully, with a critical eye on how it was set up and any anticipated
outcomes.
Dr. Stern also pointed out that recent clinical research in CF, especially as presented to
the public, has focused on extraordinarily high goals: "cure," or at the very
least a complete medical control of the disease. He reminds us there is at least a
reasonable possibility that these goals will not be met soon. In the meantime, we all have
to continue to manage with and improve upon the day-to-day treatment of CF and the
obstacles that patients and their families face. Many important treatment recommendations
appear unquestionable at certain CF centers, (e.g., advice about how to maintain caloric
intake), yet in reality, the value of many of the everyday recommendations of CF
physicians are still unproven. These issues deserve attention. The current focus on a
cure, while important, may be making some other types of clinical research less
attractive. Probability of receiving funds affects research direction.
In this same vein, Dr. Stern encouraged the audience to question what he calls the
"raspiness" on the Internet, the stuff that comes through discussion groups
about scientific reports, research findings, and the latest "treatments"
available for those with cystic fibrosis.