Stanford University researchers study concludes that clinical trials can offer patients with Huntington’s disease (HD) hope when there are no treatments available despite unknowns whether the therapy will work or is safe. The study results show that although the HD community appears highly optimistic about HD research, patients are at risk for therapeutic misconception. To allay any patients’ misgivings, investigators recommend improvements to patient-doctor communication to better convey clinical trial goals, risks and benefits.
Huntington Disease (HD)
HD is a fatal genetic neurodegenerative disease characterized by atrophy of certain regions of the brain. It causes the progressive breakdown of nerve cells in the brain. HD patients experience behavioral changes and uncontrolled movements. Symptoms include personality changes, mood swings and depression, forgetfulness and impaired judgment, and unsteady gait and involuntary movements (chorea). Every child of an HD parent has a 50% chance of inheriting the gene. Patients usually survive 10-20 years after diagnosis.
Published in the Journal of Huntington’s Disease, the study found that patients and families affected by HD believe that individuals with HD should participate in research. They viewed clinical trials positively and generally safe. They were able to appraise risks and benefits of research and were usually optimistic a new therapy would work. Individuals with prior HD-related research experience were less likely to have negative expectations about trials than those without research experience. Respondents also wanted as much information about a study as possible, with women exhibiting higher information needs than men. Investigators also learned that willingness to participate was highest when the route of administration was minimally invasive and that invasive therapies (such as those injected into the spinal column, brain, or eye) might decrease the likelihood that an individual would participate in research. Interestingly, investigators found that patients with HD were less likely to recognize the difference between clinical trials and the typical care received in an HD clinic. Finally, they found that patients with HD are more likely to believe that research participation is for personal benefit and that care they receive in a clinical trial is similar to that received in an HD clinic.
Lead investigator Kristina Cotter, PhD, CGC, Department of Genetics, Stanford University, School of Medicine, reports that “Currently, there are no therapies that slow down the progression of HD; existing treatments only help manage some of the disease symptoms. There are several therapies being studied in trials that could potentially slow down the disease. However, there is a risk inherent in clinical trial participation because the trial is studying whether or not these new therapies are safe. Potential participants should be able to discuss their hopes and fears associated with the clinical trial.”
It is possible that this belief is linked to the cognitive decline that occurs in HD,” noted Cotter. “This tells us that as we recruit for clinical trials, we should take extra care to share detailed information about trial goals, risks and benefits, how a new therapy is administered, and how the trial is different from clinical care.”
Suggestions for Patient Physician
The study authors acknowledge that participating in a clinical trial is a big deal and that decisions for patients is no trivial matter: physicians need to prepare their patients appropriately. The authors of this study offer clinicians some information that hopefully helps them discuss research with HD community members:
- Emphasize the distinction between research and clinical standard of care and consider a patient’s past experiences with clinical research when discussing trial opportunities
- Evaluate patients and caregivers for misunderstandings around the clinical research process
- Anticipate questions and concerns surrounding more invasive routes of administration
- Provide patients and caregivers with both written and verbal information about the trial, bearing in mind that women may require more details than men
The investigators hope that the findings from this study will help guide conversations among clinicians, researchers, and patients to help patients make the best decision for themselves and their families. “The better clinicians and researchers understand HD patients’ and families’ beliefs and values, the better equipped we will be to help them decide whether participation in clinical trials is appropriate for them,” added co-investigator Andrea Hanson-Kahn, MS, LCGC, Department of Genetics, Stanford University School of Medicine and Department of Pediatrics, and Division of Medical Genetics, Stanford University Medical Center, Stanford, CA.
Kristina Cotter, PhD, CGC, Department of Genetics, Stanford University, School of Medicine
Andrea Hanson-Kahn, MS, LCGC, Department of Genetics, Stanford University School of Medicine and Department of Pediatrics, and Division of Medical Genetics, Stanford University Medical Center, Stanford, CA