The Goal:PHAROS is the acronym for the Prospective Huntington's at Risk Observational Study being conducted by the Huntington Study Group (HSG). This paper describes the participants in the PHAROS study all of whom are at risk for Huntington's but are not aware of gene status.
The Method:PHAROS is an observational trial in which participants are tested but not treated. Even symptoms that are eminently treatable, such as depression, are apparently observed but not treated. Participants are tested at periodic intervals (approximately every 9 months) using the standard Unified Huntington's Disease Rating Scale (UHDRS). Doctors look for changes in UHDRS scores to see how early they can diagnose HD using this test. In effect, worsening of disease is measured over time.
The Result:One thousand people of various descriptions from 43 research centers are described. There are no study results.
Why is it Important?PHAROS is important because of the Huntington's people who are participating. One thousand at risk individuals have signed up, trusting and hoping that the knowledge obtained through this trial will hasten the search for effective treatments.
Comment:PHAROS is an old study started in 1999. It may have made sense back then, but it is past prime now. The UHDRS test is obsolete, and the data collected using this test will have little value in future HD drug trials. The fact that doctors watch people getting sick but don't treat them is distasteful at best, and strikes me as downright wrong. It's unclear from the paper whether the study is continuing, but the HSG website lists it under Clinical Trials in Progress. In my opinion, this study should be stopped at once and participants given full information on available treatments.
Why would doctors watch people getting sick and do nothing? Because they wear two hats. First hat: When working on a clinical trial, doctors want people to wait for the trial to end, no matter how long it takes, because that's the accepted way the medical community decides whether a treatment works. Hat two: Doctors whose primary job is caring for people are supposed to give patients the best possible care and information so patients can make the best informed choice. These roles can be contradictory.
The solution is to give trial participants full and honest information about the goals of the trial, the likelihood of success, and other treatment options. This is what we do in HDDW. We expect no less from others.