When designing clinical trials for huntington's disease, first ask the experts

Progress in understanding the genetic mutation responsible for Huntington's disease (HD) and at least some molecular underpinnings of the disease has resulted in a new era of clinical testing of potential treatments. How best to design clinical trials in which HD patients are willing to participate and comply is a question faced by researchers. For that reason, investigators in the Perelman School of Medicine at the University of Pennsylvania (Penn) surveyed HD patients at different stages of the disease about their attitudes and treatment goals. The results, published in the Journal of Huntington's Disease, should be useful for designing future clinical trials of gene therapies for HD and other genetic disorders. "Clinical trials in HD are creating a sense of optimism and hope within the HD community, with the majority of respondents [to our survey] reporting willingness to participate in trials. As new trials are developed and implemented, it is important to take the opinions and attitudes of the patient population into account," explained Tanya M. Bardakjian, MS, CGC, senior genetic counselor in the department of Neurology at Penn.