Recently, my mother-in-law invited her daughter, Jill, to join a Zoom call with a Huntington’s disease (HD) support group she belongs to. One of the guest speakers was Patty Conlon, clinical research coordinator in the Department of Neurology at the University of Pittsburgh Medical Center (UPMC).
Conlon shared with the group updates on recent research and a revamped observational study website called Enroll HD, which has been actively recruiting study participants since 2013.
Jill, who is genetically positive for HD, found Conlon wise and insightful. In this column, I wanted to be both inspiring and informative, so I reached out to Conlon and asked about topics that I hope will be helpful for families struggling with HD. Excerpts, slightly edited for style, follow.
What is your role within the UPMC Neurology department?
UPMC’s Department of Neurology is one of 55 clinics in the United States designated as a Center of Excellence by the Huntington’s Disease Society of America (HDSA). I first joined the Center of Excellence staff as an occupational therapist in 2015.
Soon after, I was attracted to the Research Coordinator position and wanted to be directly involved in making the research program offered in Pittsburgh more active and comprehensive. I continue to fulfill both roles in my position, but most of my time is spent on research.
At a recent HD support group meeting, you talked about a program called Enroll-HD. What is the objective ?
Enroll-HD is a long-term, global observational study looking at the effects of Huntington’s disease as people age. It is open to any family member with HD, whether or not they know their genetic status. To date, this study has recruited more than 20,000 subjects from Huntington’s families worldwide.
It acts as a database for initiating further research studies by gathering baseline and follow-up data and blood samples, which helps identify people interested in research studies and who match the criteria sought by each study.
I can personally say that when recruiting for incoming studies, my first place to look is in our local Enroll-HD database.
What demographic is Enroll-HD looking for?
Anyone from an HD family can be involved. They seek to recruit participants whether they are symptomatic or not, whether they have had genetic testing or not, and whether they are living at risk or have tested negative. The study has been particularly focused lately on younger, at-risk, tested or untested subjects with early or pre-manifested symptoms.
There is also a role in research for caregivers of people with Huntington’s disease. They can also enroll in the study and answer specific questionnaires about the effect that caring for someone with HD has had on their own health and well-being.
You are a study coordinator for clinical trials. How can people find clinical trials they may be eligible for?
There are a number of online resources to tell people about research studies they may be eligible for. The HDSA.org site contains a link to HD Trialfinder, which is a clinical trial matching service specifically for the HD community. This is a wonderful resource to sign up for if you want to be kept up to date with upcoming trials on offer.
Why are some people chosen for certain trials and others not?
As with many research programs, each clinical trial begins with a hypothesis or question that they seek to answer. In order to investigate this question, those running a trial try to line up a number of subjects who are similar in their disease stages, symptoms, other medications they are currently taking or have taken previously, and conditions medical conditions that could affect their performance or safety in the trial. This way, the study is structured to say with fairly high confidence that any difference between the treatment group and the placebo group is due to the study drug.
What gives you hope for the future in terms of clinical trials?
The current rate of new studies being proposed for the treatment of HD is higher in the past five years than ever before. With each study, there is a wealth of knowledge to be gained, not only about what works, but also what doesn’t. I hope this information will lead us to a treatment that will eliminate the threat of HD in our lifetime.
The course of Huntington’s disease is already remarkably different from what it was years ago. Symptom management has become more reliable and there are many other treatment options for individualized care. I love being on the front line of this battle. Our families and future generations rely on what we do today.
To note: Huntington’s disease news is strictly a disease news and information site. It does not provide medical advice, diagnosis or treatment. This content is not intended to replace professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay seeking it because of anything you read on this website. The opinions expressed in this column are not those of Huntington’s disease news or its parent company, BioNews, and are intended to spark discussion about Huntington’s disease issues.