Clinical CF Research: Trials and Tribulations

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.

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