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NewsHSG UPDATES – September 2023

September 2023

  1. CEO’s Insights
  2. New Options for Patients: FDA Approval of Valbenazine
  3. Lowering Research Participation Barriers: vUHDRS® Topline Results
  4. Facilitating HD Research Collaboration: CHDI
  5. Forging More Diversity and Representation in Research: myHDstory® Results
  6. Creating Opportunities for Learning, Engaging, and Connecting: HSG 2023 Annual Meeting
  7. Building a Stronger HSG: ENGAGE-HD + New Members + Process for New Ideas and Projects
  8. Reflections on an Impactful Life: Remembering Christine Hunter
  9. Catalysts for the Future: HSG Education and Training
  10. In the Know

1.   CEO’s Insights

In memory of Christine Hunter, someone who was devoted to making an impact on the Huntington’s disease community with her skills, talent, and heart, we are dedicating this issue of HSG Updates to the theme of impact. While we have had some big wins, we recognize these are some battles won while a war rages on. No one is giving up. In this issue we explore some of the areas in which HSG has recently driven progress, spurred potential, and helped positively impact the acceleration of treatments for HD.

– Shari Kinel, JD

2.   New Options for Patients: FDA Approval of Valbenazine 

We are thrilled with the recent approval of valbenazine for chorea in adults with HD. This approval was based off the positive, highly statistically significant results from the KINECT-HD trial and the ongoing KINECT-HD2 trial led by the HSG and NBI. While this is not the first VMAT2 inhibitor approved for chorea associated with HD, this is still a welcome and important win for those living with or at-risk for HD.

Evaluation of the ENROLL-HD database demonstrates that chorea may be undertreated. The reasons for this finding are multifactorial but this reminds us that we still have work to do in the realm of symptomatic treatment for HD. Chorea is certainly not the only symptom our patients struggle with; however, it is an important symptom that can negatively impact quality of life, functional independence and increase caregiver burden.

A positive trial not only gives our patients another symptomatic treatment option, but also provides opportunities to raise awareness and in doing so, hopefully indirectly improves care and access to HD patients and families. In addition, the availability of multiple treatment options encourages ongoing research and innovation in the field of HD.

We remain optimistic about and focused on an approved disease modifying therapy for HD hopefully sooner rather than later, but in the interim, we hope that additional effective symptomatic treatments can help our HD patients and families throughout their journey.

– Erin Furr Stimming, Study Principal Investigator of KINECT®-HD/HD-2 Studies

3.   Lowering Research Participation Barriers: vUHDRS® Topline Results

The Huntington Study Group (HSG) has revolutionized research methodologies by introducing innovative approaches. Our participant-centric strategies, coupled with advancements in technology, aim to deliver clinical trials to participants, eliminating the need for participants to travel to trial sites. This approach mitigates recruitment and participation barriers, including unforeseen circumstances, geographical constraints, evolving participant preferences, and time constraints. HSG has a longstanding commitment to addressing these challenges, consistently achieving study enrollment and retention objectives while conducting high-quality clinical trials.

In 2010, HSG’s Project AWARE was a comprehensive survey of over 600 individuals and families impacted by Huntington’s disease (HD). This observational study aimed to enhance awareness, willingness, and ability to participate in clinical trials and observational studies, contributing to the design of future trials. HSG has diligently applied the insights gained from Project AWARE to initiatives aimed at facilitating access to research centers and formulating study protocols that incorporate the input of HD patients, thereby enhancing the quality of clinical trials.

In keeping with the HSG’s philosophy of bringing innovative methods to clinical research, the HSG successfully embarked on developing a virtual version of the gold standard assessment tool, the Unified Huntington’s Disease Rating Scale (UHDRS®) developed in 1996 (HSG et al 1996). The results of the virtual Unified Huntington’s Disease Rating Scale (vUHDRS®) showed that virtual implementation of the vUHDRS® was reliable and feasible including assessment of motor, Total Functional Capacity and all other functional sections, cognitive and behavioral sections. The HSG is looking to the vUHDRS® as the next research study tool to assist with the development of hybrid clinical trials combining remote and in-person visits to significantly reduce participant burden and increase research.

The vUHDRS® results were presented at the International Congress of Parkinson’s Disease and Movement Disorders® in Copenhagen, Denmark in August and will be presented at HSG’s annual meeting in Phoenix.

– Samuel Frank, MD, vUHDRS® Study Principal Investigator
– Jody Goldstein, vUHDRS® Study Co-Principal Investigator

4.   Facilitating HD Research Collaboration: CHDI

For 30 years, HSG has been working to improve the lives of people impacted by Huntington’s disease through research, education, and collaboration. By building collaborative relationships and credibility with key HD stakeholders, we are better positioned to understand perspectives and fulfill the needs of research site personnel, sponsors, and people impacted by HD.

In July of this year, we announced that our clinical research organization (CRO), HSG Clinical Research, Inc. (HSGCR), began providing services for CHDI Foundation’s Enroll-HD observational study and clinical research platform in the US and Canada. Enroll-HD is currently the world’s largest observational study for Huntington’s disease families, providing an important resource for HD families, clinicians, researchers, advocates, pharma, and biotech companies.

The melding of the operational and clinical experience and knowledge of CHDI and HSG will enable the teams to mold the next generation of Enroll-HD by enhancing operational efficiency and quality and potentially creating new ways to recruit study participants, collect study data, and ensure data integrity.

Combining the strengths of both organizations allows us to more collaboratively accelerate treatments that make a difference and make a greater impact for the entire HD community. 

– Elise Kayson, HSG VP of Clinical Operations 

If you’re interested in collaborating with HSG, send us a note at info@hsglimited.org

5.   Forging More Diversity and Representation in Research: myHDstory® Results

One of the main goals at HSG is to improve diversity in research by reaching underrepresented populations. We know from the HSG Care Improvement Project surveys and other work that 70% of people with HD do not access specialty care. Because the US is such a large country, and specialty care and research are concentrated in big cities, many groups, particularly those in rural and less populous areas, are not able to join clinical or observational studies.  It can also be burdensome to travel to a site for research visits. Those with fewer economic advantages are therefore underrepresented in many studies.

Most clinical trials are 90% or more Caucasian, but the overall population is much more racially diverse, and HD affects all races and ethnic groups. At HSG, we have several initiatives to make research more accessible and inclusive. This includes the myHDstory® online, direct-to-participant research platform. We recognize that research cannot be representative of the entire HD population if it does not include perspectives from people of all backgrounds.  With the myHDstory® platform, people with HD can participate in an observational study from the comfort of their own home or other private location.

HD presents many challenges to patients and their families. HSG offers online participation through the myHDstory® platform to lessen this burden with respect to research participation. It is our hope that these efforts will make research more representative of the HD population as a whole and help us to understand how people from differing economic and racial/ethnic backgrounds experience HD symptoms.

More studies are currently in the works for the myHDstory® platform, including new PROs, longitudinal capabilities, and reaching even more participants. We hope to have some exciting announcements

– Karen Anderson, MD, Study Principal Investigator of ‘Making HD Voices Heard’

6.   Creating Opportunities for Learning, Engaging, and Connecting:
HSG 2023 Annual Meeting

Among the not-so-hidden benefits of the HSG meeting is the opportunity to meet, re-meet, learn from, and connect with colleagues who share a passion for care of HD families and research in HD. For a young clinician, investigator, or coordinator, this is the best opportunity you will have all year to discuss difficult cases, think about different ways of managing patients, learn about new and upcoming research studies, and share HD-related experiences. You can join a working group, volunteer for a committee, learn from a seasoned investigator how to conduct a study more efficiently, and test out your bright idea on an interested audience. Submit a poster describing your work! New in 2023 is a Saturday training program called ENGAGE-HD, specifically directed towards the HD experts of the future.   

Even if your hair has begun to turn gray, you will still benefit from hearing about the successes and challenges that your colleagues have faced, while inspiring and encouraging the younger attendees. Become an HSG leader! Give a presentation, create a new working group, lead a clinical trial!   

The relationships you develop and nurture at the HSG 2023 meeting will last throughout your professional lifetime—and beyond! 

– Martha Nance, MD, HSG Executive Membership Committee and Chair of HSG Family Education Committee

If you’re planning to attend this year’s annual meeting, here are a few useful links:

7.   Building a Stronger HSG: ENGAGE-HD + New Members + Process for New Ideas & Projects

In the realm of scientific progress, one fundamental truth stands out: progress requires people. The pursuit of groundbreaking research is inherently dependent on the collaborative efforts of dedicated individuals. But despite the increasing prevalence of progressive neurodegenerative diseases, the relatively stagnant numbers of medical students opting to specialize in neurology creates a concerning gap. The future of HD research and patient care is at risk. HD is a scientifically fascinating and clinically significant disease that has the potential to attract the intellectual curiosity of the next generation of medical practitioners and researchers. To galvanize this interest and nurture a fascination with HD as a career path, we need to offer HD-specific education created and curated by international experts.

The acronym ENGAGE-HD means to Educate, Nurture, Gather, Acquire knowledge, Grow, and Excite professionals, both experienced and new, about HD. The HSG ENGAGE-HD program is an initiative to attract, foster, and educate new members and researchers and to create educational programs and events exclusively centered on Huntington’s disease for those with an interest in neurology and movement disorders. Through this initiative, we hope to strengthen the impact HSG has on the world of research and treatment. 

Our overarching goal is to orchestrate a transformation: expand the pool of professionals dedicated to the pursuit of movement disorders (particularly HD), bolster the knowledge of people committed to research and treatment of HD, and add meaningfully to the body of HD-related publications. By cultivating the growth of researchers and practitioners passionate about Huntington’s disease, HSG becomes a stronger organization, better poised to enhance patient care and uncover scientific breakthroughs.

– Jee Bang, MD, MPH, HSG Executive Membership Committee and Chair, HSG Provider Education Committee

Over the past few months, HSG’s membership has continued to grow. There are several new faces as well as some people with whom HSG has been working for quite some time and are now official. We welcome all of you to our network and look forward to everything we will accomplish together. You are the future of HSG.

If you are already an HSG member, please login here to ensure all of your information is up to date.

Process for New Ideas and Projects
At HSG, we’re continually impressed by our members and the education, research, and care initiatives you develop. As our network continues to grow and thrive, we want to ensure that we have the proper support in place to help ensure efficiency, effectiveness, and collaborative success. Please take the time to familiarize yourselves with the related policies and processes below, and don’t hesitate to reach out with any questions.

Preliminary Proposal Form
Are you interested in starting an education or care initiative or a research project under HSG? If so, start by filling out our Preliminary Proposal Form. This is a short form intended to provide leadership with high-level details so they can ensure it is in line with HSG’s mission and is feasible from a resource perspective. If your preliminary form is approved, you will be asked to submit a more detailed proposal through either the HSG Education or Care Initiative Formal Proposal Form or the HSG Investigator-Initiated Clinical Research Formal Proposal Form. If your preliminary form is not approved, you will be provided with feedback and suggestions for a one-time resubmission.

Policy for Proposals Utilizing HSG Resources
If you are working on an HSG-approved project and are seeking funding from an external company (i.e., not HSG), please complete the Policy for Proposals Utilizing HSG Resources form prior to contacting any potential funders.

8.   Reflections on an Impactful Life: Remembering Christine Hunter

When we think of dedicated professionals, few names shine as brightly as Christine Hunter’s. With a heavy heart, we reflect on her life, one that left an indelible mark on our hearts and the world of medical research. Christine’s journey was nothing short of extraordinary, and her legacy continues to resonate within the Huntington’s and Parkinson’s Disease and communities.

A Passionate Advocate
Christine’s involvement in the Parkinson’s community was profound. She served as a coordinator representative on the Executive Committee at PSG (Parkinson Study Group) and, beyond retirement, remained actively engaged with the Parkinson’s Foundation and the Michael J. Fox Foundation. Her dedication was unwavering, a testament to her heart of gold and a smile that radiated love and kindness to all fortunate enough to know her.

Karen Williams, from Northwestern, eloquently captured Christine’s essence when she said, “Christine always made time for everyone, especially her fellow coordinators. She served as a mentor (officially and unofficially) to so many people over the years and was always available to lend an ear or give her opinion, she was a wealth of knowledge.”

Patients Were Her Priority
Throughout her illustrious career, Christine never lost sight of her patients. Whether speaking at support groups, volunteering at Tourette’s camps, or offering one-on-one support to patients and their families, she embodied the true spirit of compassionate healthcare. ZsaZsa Brown, also from Northwestern, aptly noted, “She was an advocate for the HD community as well as a great role model for us Coordinators. She will truly be missed!” 

A Remarkable Leader and Friend
Joseph Jankovic, MD, a close colleague and friend, beautifully encapsulated Christine’s remarkable contributions. He described her as “a beacon of hope for countless patients with Parkinson’s disease, Huntington’s disease, Tourette syndrome, and many other movement disorders.” Christine’s dedication went beyond borders, as she volunteered on medical missions in underserved areas and touched lives worldwide.

In a poignant personal note, Dr. Jankovic shared, “Christine was my most trusting professional partner, advisor, and confidant as well as a close friend. Because of her unique combination of warm empathy and inspiring enforcer of discipline, I often referred to her as a hybrid between Mother Theresa and General Patton.”

Christine’s sudden and tragic passing on July 26, 2023, during a family vacation in Branson, Missouri, has left a void in our lives and the broader medical community. She is survived by her loving husband, two sons, and four sisters.

As we reflect on Christine’s impactful life, let us honor her memory by carrying forward her passion for our work and striving to make our patients’ lives better. Her kindness, generosity, and wisdom will continue to inspire us and future generations, ensuring her legacy lives on.

9.   Catalysts for the Future: HSG Education and Training

Preparing investigators and coordinators for clinical trial conduct is at the core of the HSG Site Readiness Training Program. The HSG training and development programs serve as a catalyst to ensure ample support and clear guidance to all HSG members. For years, our Site Readiness Training Program (formerly known as “Member Education”) has served as the foundation for advancing clinical trial knowledge and developing what we refer to as “Tiger Teams” – an elite set of sites that can rapidly deploy a clinical study.

Our training programs include a comprehensive collection of tools and resources that enables HSG members to execute clinical trials in rapid time to provide consistent, timely, high-quality data. Providing education and training on innovative assessment tools, new methodologies, and changes in established techniques are key to preparing the next generation of researchers, clinical trial success, and ultimately our ability to make an impact for the HD community.

– Samuel Frank, MD, Co-Chair of HSG Executive Membership Committee

– Jody Goldstein, HSG Director of Clinical Operations


Grant Opportunity
The Hereditary Disease Foundation provides Postdoctoral Fellowships and Grants to researchers around the world to advance the discovery and development of treatments for Huntington’s disease and other brain disorders. They began accepting Letters of Intent for Postdoctoral Fellowships and Grants on September 15, 2023 with a due date of November 1, 2023. Click here to learn more.

Upcoming Events
Help4HD International Symposium – October 13-14 in San Diego, CA
If you’re attending this meeting and would like to connect with HSG’s Director of Clinical Operations, Jody Goldstein, send us a note at info@hsglimited.org.

European Huntington’s Association Conference – October 19-22 in Belgium

Quick Links
HSG Website
HD Trial Finder – US & Canada
HD Trial Finder – Europe

If you would like to write an article for the HSG Updates newsletter or have your meeting or event featured, contact kristin.keyes@hsglimited.org