As scientists, we are trained to describe our work with a factual clarity that may seem dry and opaque. However, in the summaries and abstracts, there is often a convention to use hopeful, forward looking statements to indicate to why we believe our work is relevant, noteworthy and important. This optimism is uncharacteristic in the context of the rest of scientific text, and can easily be misinterpreted by patients and their families.
This was brought home to me at a Friedreich’s Ataxia (FA) scientific meeting. The parents of a young girl with FA asked me to explain a poster describing an early stage small molecule therapy research effort that had concluded that it could be a safe effective therapy for FA. These parents had latched on to this forward looking statement and wanted to know when it would be available to their wheelchair bound daughter.
How do we, as scientists, project the potential for a therapeutic breakthrough at the earliest stages of drug development, while also communicating the immense amount of work that still needs to be done and the time that it would require?
While it is next to impossible to describe scientific findings in a manner that would satisfy all audiences, perhaps we could begin by adding a statement for projects that get presented at scientific meetings that patients and their families attend. This statement might include some of the caveats that we as drug developers appreciate based on the stage of development, that may go unrecognized by the lay population, including high rates of failure in preclinical and clinical safety studies, regulatory hurdles, and challenges defining and achieving clinical evidence of efficacy. Perhaps this can be simplified by estimating of how far from patient clinical trials the therapy might be. We might also be mindful about differentiating approaches that might lead to potential therapies from those theoretical cures. I offer these suggestions thinking that the work we do is not in a vacuum but rather part of a larger deeply invested community to whom we have a responsibility.