Press Releases

Huntington Study Group® Holds 30th Annual Meeting 

Rochester, NY — November 20, 2023 — The Huntington Study Group® (HSG®), a world leader in conducting clinical trials and providing educational programming for Huntington’s disease (HD), recently held its 30th annual meeting, HSG 2023, November 2-4 in Phoenix, AZ. Hundreds of scientists, industry partners, HSG research sites and members, advocacy groups, staff, HD patients, families, and caregivers from around the world convened to hear the latest in HD research and advancements, network with colleagues, and connect with a dedicated HD community.   

This year’s sessions focused on 30 years of learnings, progress, and accomplishments since HSG’s founding and the discovery of the HD gene 1993. Presentations also discussed the changing future of clinical trials, emphasizing how the HSG is developing innovative approaches to conducting clinical trials to lessen the burden of participation. As the lead clinical research organization for industry sponsored multi-center global studies, the Huntington Study Group shared how HSG is developing novel approaches in clinical trials, including remote visits, wearable sensors, direct-to-participant virtual visits, and virtual assessment tools such as the vUHDRS™, a key assessment tool in HD trials. These approaches will enhance enrollment and participation in clinical research and ultimately accelerate treatments for those affected by Huntington’s disease.  

“It was inspirational to gather for HSG’s 30th annual meeting, reflect on our accomplishments, and discuss the important work we’re focusing on for the HD community. For three decades, HSG has been the leader in convening its global network of researchers focused in HD. The power and impact of having so many dedicated people coming together to focus on improving the lives of patients with HD continues to inspire me,” said Samuel Frank, MD, Co-Chair of HSG’s Executive Membership Committee.

This year’s meeting highlights included a record-breaking number of submitted abstracts which are published in the Journal of Huntington’s Disease. In addition, two of HSG’s founders, Drs. Ira Shoulson and Karl Kieburtz, provided a 30-year view of the accomplishments in research and discovery for Huntington’s disease which laid the foundation for the current pipeline of investigational research including novel gene therapies. Attendees had the opportunity to learn about HSG’s comprehensive education center, the “HSG Brainery,” which will streamline the education, certification, and credentialing opportunities the organization provides to researchers, clinicians, healthcare partners, community members, and persons impacted by HD.

The inaugural session of ENGAGE-HD (Educate, Nurture, Gather, Acquire knowledge, Grow, and Excite professionals about Huntington’s disease), a groundbreaking program committed to finding and educating the next generation of HD researchers, was held on Saturday. This effort was developed and is being led by longtime HSG members Erin Furr Stimming, MD, Jee Bang, MD, MPH, and Jody Corey-Bloom, MD, PhD.

Saturday also marked the return of Family Day, an annual day-long program dedicated to patients, families, caregivers, and the local HD community. This year’s sessions were led and developed by Martha Nance, MD, Chair of HSG’s Family Education Committee, along with a team of Phoenix-area healthcare providers from Barrow Neurological Institute. One of this year’s keynote speakers at Family Day was Erin Paterson, author of the memoir, All Good Things. Erin spoke about her journey with genetic testing, infertility, and the search for happiness. Allie LaForce, TNT Sports reporter and one of the founders of HelpCureHD, a not-for-profit organization dedicated to providing grant funding for PGT-IVF (pre-implantation genetic testing in-vitro fertilization), shared her experience with genetic testing and PGT-IVF during this year’s Spotlight on Genetics panel session.

“HSG is dedicated to accelerating treatments that make a difference, and the efforts we were able to showcase this year in support of our mission are a testament to the strength of our network,” said Shari Kinel, CEO of HSG. “We are so proud of our accomplishments and are hopeful that we’ll have even more successes to share at next year’s meeting. Until then, we will continue doing everything we can to improve the lives of people impacted by HD through research, education, and collaboration.”

Planning for HSG 2024 is underway and a location will be announced soon.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.   

About Huntington Study Group / HSG Clinical Research, Inc.
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network with thousands of members at more than 130 HSG credentialed research sites worldwide. HSG conducted all three pivotal clinical trials that led to the only FDA-approved medications for Huntington’s disease associated chorea. The organization is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

To read the full press release, click here.

Huntington Study Group® Observational Pilot Study Shows HSG’s myHDstory®  Platform Reaches the Unreachable

Rochester, NY, October 11, 2023 – The Huntington Study Group® (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc. (HSGCR), a world leader in conducting clinical trials for Huntington’s disease (HD), today announces that the pilot observational study on their innovative online direct-to-patient research platform, myHDstory®, is complete with unprecedented topline results.

The pilot study, Making HD Voices Heard, not only achieved its goal of capturing the patient voice to allow for a better understanding of how HD patients experience their symptoms and how the disease affects their wellbeing, but also indicates that HSG’s myHDstory® platform successfully reaches participants who don’t usually take part in research. The results show that most participants in the study had never participated in any trials or studies. In addition, there was much higher racial and geographical diversity seen in participants than in any other HD study.

Karen Anderson, MD, Professor of Psychiatry & Neurology at Georgetown University and longtime HSG member, was the study’s Principal Investigator. Dr. Anderson stated, “By allowing participation in research anytime, anywhere, HSG has developed a tool that breaks down some of the barriers to research participation, allowing for a better understanding of HD and the potential for improved data from patient reported outcome measures. The high participation rate, the racial and ethnic diversity, and the relatively young age of myHDstory® participants are meaningful.”

Making HD Voices Heard successfully reached over 400 participants in the US reporting their lived HD experience using the HD-Patient Report of Problems (HD-PROPTM), a tool created by Ira Shoulson, MD and developed by Grey Matter Technologies and Modality.ai to interpret and classify symptoms reported by patients in their own words.  Dr. Shoulson, HSG founder, also served as the study Co-Principal Investigator.

“HSG is thrilled by the results of the pilot study and the team is working diligently on developing the next two studies for the myHDstory® platform. These will include longitudinal capabilities so we can measure changes over time. We’re grateful to the study participants who devoted their time and energy to this study and whose dedication supports our shared mission of accelerating treatments that make a difference,” said Shari Kinel, HSG CEO.

A conference call is scheduled in mid-October to provide updates to the Making HD Voices Heard study participants.

An abstract detailing the pilot study’s results has been accepted for a poster presentation at Huntington Study Group’s annual meeting in Phoenix, AZ this November. To learn more or register for the meeting, visit huntingtonstudygroup.org/hsg-2023/. For more information about myHDstory® or to sign up to receive updates about future studies, visit huntingtonstudygroup.org/myHDstory/.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent.  Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered. 

About Huntington Study Group / HSG Clinical Research, Inc.
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network with thousands of members at more than 130 HSG credentialed research sites worldwide. HSG conducted all three pivotal clinical trials that led to the only FDA-approved medications for Huntington’s disease associated chorea. The organization is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit huntingtonstudygroup.org.

To view the full Press Release, click here

 Huntington Study Group Welcomes FDA Approval of New Drug for Chorea in Huntington’s Disease 

Rochester, New York, August 18, 2023 – The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., a world leader in clinical trials for Huntington’s disease (HD), today shares that the Phase 3 pivotal KINECT®-HD study conducted by HSG in collaboration with Neurocrine Biosciences, Inc. resulted in the U.S. Food and Drug Administration’s (FDA) approval of Neurocrine’s drug, valbenazine, for the treatment of chorea associated with HD. HSG has been instrumental in all three pivotal clinical trials that have led to FDA approved medications for Huntington’s disease.

Chorea, the hallmark motor symptom of HD, described as irregular, excessive involuntary movements can severely negatively impact day-to-day life. Dr. Erin Furr Stimming, Neurologist at UTHealth Houston, longtime HSG member, and Principal Investigator of the KINECT-HD study said, “It is incredibly important to have additional therapeutic options for people with HD, and we are extremely grateful to our HD study participants for helping us make this new treatment available.”

The KINECT-HD study met primary and secondary endpoints, showing that valbenazine, a novel vesicular monoamine transporter 2 (VMAT2) inhibitor, reduced HD-related chorea symptoms as early as two weeks after the initial dose. In KINECT-HD, valbenazine was also well tolerated. Andrew Feigin, MD, Chief Medical Officer of HSG said, “We are immensely proud of our collaboration with Neurocrine that led to this important study.”

KINECT-HD was a randomized double-blind, placebo-controlled study with 128 participants at 46 HSG sites across North America conducted by Huntington Study Group in collaboration with Neurocrine Biosciences, Inc. and Clinical Trials Coordination Center (CTCC)/Center for Health + Technology (CHeT) at the University of Rochester Medical Center. The study also included the use of a novel patient reported outcome measure, Huntington’s Disease Health Index (HD-HI), and a sensor sub study. The complete results of the study were published in The Lancet Neurology in June 2023.

Shari Kinel, CEO of HSG said, “We would like to thank the study participants, their families, and HSG sites for their support of KINECT-HD.”

Neurocrine issued a press release today announcing the FDA approval. The press release can be found on their website at https://www.neurocrine.com/news-and-media/.

Neurocrine Biosciences, Inc. will be providing an update on the KINECT-HD studies at HSG’s Annual Meeting on November 2-4, 2023, in Phoenix, Arizona. KINECT®-HD2 is an ongoing open-label study to evaluate the long-term safety and tolerability, as well as the maintenance of effects of INGREZZA in patients with HD chorea.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network with thousands of members at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

About Clinical Trials Coordination Center at University of Rochester Medical Center
CTCC specializes in the development, management, and conduct of clinical research studies and provides a full range of research and clinical trial management support services that facilitate the conduct of clinical research from study concept through data analysis, publication, and FDA approval. Over the past 30 years, the CTCC has managed the conduct of more than 135 clinical research studies with 50 sponsors (government, industry & private) that enrolled over 40,000 research participants in US, Canada, Europe, New Zealand, and Australia.

To read the full press release, click here.

HSG and HD-CAB Collaborate to Provide Global Patient Advisory Board

Rochester, NY, March 30, 2023 — The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., a world leader in conducting clinical trials for Huntington’s disease (HD), recently collaborated with Huntington’s Disease – Community Advisory Board (HD-CAB) to provide a Global Patient Advisory Board on behalf of Annexon Biosciences, Inc. Annexon is a clinical-stage biopharmaceutical company whose focus is on developing a pipeline of novel therapies for patients with disorders of the body, brain, and eye that are caused by improper regulation of the body’s complement system1.

The Advisory Board (Ad Board), held on November 30, 2022 in Jersey City, New Jersey, USA, was a six-hour session that included eleven HD-CAB advocate members from the US, Canada, Scotland, France, Italy, Sweden, Pakistan, and India, who convened to share with Annexon their stories and viewpoints and to discuss the challenges HD trial participants and their families face. One of the goals of the Ad Board was to encourage open communication about how trial sponsors and Clinical Research Organizations (CROs) can better support HD families before, during, and after clinical trials. This conversation was led by Astri Arnesen, Chair of HD-CAB and President of the European Huntington Association. Dr. Christopher Ross, HSG’s Chief Scientific Officer and Dr. Jody Corey-Bloom, Neurologist at UC San Diego and longtime HSG member, facilitated a discussion regarding Annexon’s scientific rationale in HD and planned trial design.

“HD-CAB represents the voice of the global HD community. Consisting of HD family members from six continents and 20 countries, our aim is to ensure that critical factors aren’t overlooked in the design and implementation of clinical trials. It was a pleasure to work with HSG and Annexon as they actively listened to the real-world experience of HD-CAB advocates, adjusted course as appropriate and committed to doing so on an ongoing basis,” said Astri Arnesen.

“I would like to thank the HD-CAB advocates as well as the HSG and HD-CAB management teams for the expertise and actionable next steps shared at the Global Patient Advisory Board,” said Peter Collins, VP, Global Program Team Lead of the Neurodegeneration Franchise at Annexon Biosciences. “We look forward to continued collaborations with leading patient organizations, such as HD-CAB and HSG, to ensure Annexon consistently engages with, and incorporates feedback from, the patients and families we seek to serve.”

HD-CAB recently completed a follow-up International Ad Board on behalf of Annexon Biosciences at the HYDO International Congress in Glasgow, Scotland in March 2023. HSG, HD-CAB, and Annexon Biosciences are continuing to discuss opportunities for future collaborations.

Dr. Ross noted, “HSG is always looking for innovative ways to support and engage the HD community and to make sure their voices are heard. We found this collaborative effort to be truly meaningful and hope this opportunity is just one of many that we will have the privilege of participating in. Our team is grateful to HD-CAB for their invaluable contributions and to Annexon for enabling our organizations to make this important meeting come to fruition. It was a great success.”

If you’re interested in learning more about collaboration opportunities, please contact info@hsglimited.org.

1 The body’s complement system is part of the immune system. It is responsible for defending cells and tissues from infections and diseases.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network of over 800 experts in Huntington’s disease at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

About HD-Community Advisory Board
HD-Community Advisory Board (HD-CAB) is a coalition of partners from the European Huntington Association, the International Huntington Association, and the Huntington’s Disease Youth Organization whose mission is to represent the voice of the global HD community and provide HD community experience to regulators, industry, researchers, and governing bodies. HD-CAB provides the unique expertise of living with HD to stakeholders relevant for therapeutic development. For more information, visit www.hd-cab.org.

Click here to view the full press release.

Huntington Study Group and CHDI Foundation Collaborate on Enroll-HD

Rochester, NY and New York City, NY, July 25, 2023 — HSG Clinical Research, Inc. — the Huntington Study Group’s clinical research organization (CRO) and wholly owned subsidiary (collectively referred to here as HSG) — has begun providing CRO services for CHDI Foundation’s Enroll-HD observational study and clinical research platform in the USA and Canada. 

“CHDI has long supported and worked with the HSG as a clinical investigator-facing network of healthcare professionals dedicated to Huntington’s disease,” said Robi Blumenstein, President of CHDI Management. “HSG is very familiar with the HD clinical sites throughout North America and, with the establishment of its clinical research arm, HSG Clinical Research, it was only natural that we would turn to HSG as a CRO for Enroll-HD. HSG brings exceptional domain knowledge and continuity to Enroll-HD and the studies and trials it supports, and we look forward to the continued expansion of this relationship over time.”

Enroll-HD is a clinical research platform and the world’s largest observational study for Huntington’s disease families that serves as a resource for the entire HD community, including families, clinicians, researchers, advocates, and the vitally important engagement of pharma and biotech companies developing novel therapeutics. Currently, the Enroll-HD clinical database comprises more than 30,000 participants from around the world at more than 155 clinical sites in 23 nations.

CHDI and HSG will leverage their expertise and innovation to continue gaining knowledge and insight into the natural history of HD, combine their strengths in research, clinical expertise, and study execution to ensure the ongoing success of Enroll-HD, and work together to accelerate therapeutic development to make a difference for those affected by Huntington’s disease.

Shari Kinel, CEO of Huntington Study Group, noted, “CHDI is an incredible organization and HSG could not be more excited to work with them on this important study. Our organizations have a shared commitment to improving the lives of individuals and families affected by HD, and we are confident that our joint efforts will lead to meaningful outcomes. We are already looking forward to future collaboration opportunities.”

Learn more about Enroll-HD, including how to participate and how it can help your research. 

If you’re interested in learning more about opportunities to collaborate with HSG, please contact info@hsglimited.org.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent.  Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

To read the full press release, click here.

Huntington Study Group Announces Positive Topline Results for Virtual Unified Huntington’s Disease Rating Scale® (vUHDRS®) Study

The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., a world leader in conducting clinical trials for Huntington’s disease (HD), today announces positive topline results from its Virtual Unified Huntington’s Disease Rating Scale® (vUHDRS®) observational study. This novel, innovative study assessed the reliability of virtual use of the HSG’s standard assessment tool, the Unified Huntington’s Disease Rating Scale® (UHDRS®).

The analysis of the data was consistent with key portions of the scale showing excellent reliability. Specifically, the modified motor portion and the primary outcome measure of stage, the Total Functional Capacity, had high intraclass correlation coefficients (ICCs). In addition, all other functional, cognitive, and behavioral sections had ICCs consistent with excellent reliability. The vUHDRS® study may assist with the development of remote and hybrid trials thereby reducing participant burden and increasing availability of data.

The vUHDRS® study, funded and sponsored by Huntington Study Group, was inspired by the necessity to break down barriers, and allow remote assessments in HD trials. The COVID-19 pandemic accelerated vUHDRS® development. The study was conducted at 16 HSG sites in the United States. Fifty-nine participants completed the study with about half using their own devices and the other half using HSG-provided tablets and portable Wi-Fi connections.

“These results are exciting for clinical research and patient communities. Clinical research in Huntington’s disease is one step closer to being more accessible to a larger population and offers the opportunity for more access to remote care in HD,” said Samuel Frank, MD, Associate Professor of Neurology and Director of the HDSA Center of Excellence at Beth Israel Deaconess Medical Center, and Principal Investigator of the vUHDRS® study. “The lessons learned and data from this study supports our belief that this key endpoint for HD studies can be effectively delivered in a virtual setting, thereby improving overall care and development of treatments for our patients and families impacted by HD.”

The vUHDRS® results will be presented at the International Congress of Parkinson’s Disease and Movement Disorders® in Copenhagen, Denmark, August 27-31, 2023, and at HSG’s annual meeting in Phoenix, AZ, November 2-4, 2023.

About the Unified Huntington’s Disease Rating Scale (UHDRS®)
The HSG developed and owns the UHDRS® which was established in 1996 to assess motor, cognition, behavior, and function in patients with Huntington’s disease in a standardized manner. The UHDRS® in part or in whole is commonly used as the primary endpoint in many HD trials. To learn more about the tool including licensing information, visit https://huntingtonstudygroup.org/uhdrs/.

About Huntington’s Disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network with thousands of members at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

To read the full press release, click here.

HSG UPDATES – September 2023

September 2023
MAKING AN IMPACT

  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

ENGAGE-HD
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

10.   IN THE KNOW

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
clinicaltrials.gov

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 

Huntington Study Group Achieves Last Patient Last Visit for Its Observational Study to Test Virtual Use of the Unified Huntington’s Disease Rating Scale®

Rochester, NY – May 16, 2023 – The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., announces an important milestone in the novel observational study Virtual Unified Huntington’s Disease Rating Scale (vUHDRS™). The last participant completed the last visit, marking study completion.

The purpose of this study is to determine the reliability of administering all sections of the Unified Huntington’s Disease Rating Scale (UHDRS®) virtually compared to in-person for clinical and clinical trial use. The UHDRS® is the gold-standard in assessing motor function, cognitive function, behavioral abnormalities, and functional capacity in Huntington’s disease (HD). Since the tool was originally developed in 1993, the HSG-owned instrument has been used in part or in total as the primary endpoint in many HD trials. The changing face of clinical trials is prompting the need for reliable tools for assessing and interacting with patients and research participants remotely. This need became clearer and more urgent during the COVID-19 pandemic, accelerating HSG’s development of this study.

vUHDRS™ has the potential to positively impact care delivery and clinical trial operations as part of remote and hybrid trials, effectively removing some of the barriers people face when participating in research studies. This study could also increase data availability, frequency of data collection and could enhance clinical care.

Samuel Frank, MD, Associate Professor of Neurology, Director of the Huntington’s Disease Society of America Center of Excellence at Beth Israel Deaconess Medical Center, and Principal Investigator of the vUHDRS™ study, said “It’s exciting to be part of this critical study that will enhance future clinical trials in HD. We couldn’t do any of this without an incredible team and the help of our dedicated participants for whom we are so thankful.”

The vUHDRS™ study was conducted at 16 HSG sites across the US, where 60 subjects with motor manifest HD were followed over approximately six weeks through a combination of in-person and remote visits. The study team is beginning to analyze the data and hopes to announce results in third quarter 2023.

More information about this study will also be presented at HSG’s 30th Annual Meeting in November. To learn more about the meeting, including sponsorship opportunities, click here.  
 
About Huntington’s disease 
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent.  Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.  
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network of over 800 experts in Huntington’s disease at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org. 

To read the full press release, click here.

Huntington Study Group Announces Call for Abstracts for the 30th Annual Meeting

Rochester, NY – June 1, 2023 – The Huntington Study Group (HSG), with a mission of accelerating treatments that make a difference for those impacted by Huntington’s disease, together with its wholly owned subsidiary, HSG Clinical Research, Inc., a world leader in conducting clinical trials for Huntington’s disease (HD), today announces that the submission period has begun for abstracts to be considered for presentation at their 30th annual meeting in November (“HSG 2023”).

For decades at their Annual Meeting, HSG has provided an opportunity for researchers to present valuable findings and information about HD clinical research to event attendees through poster sessions and platform presentations during the Peter Como HD Research Symposium. Dr. Como, one of the founders of HSG, dedicated his life to advancing research and promoting the sharing of new knowledge about Huntington’s disease.

All abstracts accepted by HSG’s Publications Committee will be presented by the authors at HSG 2023 during multiple poster viewing sessions throughout the meeting, printed in a program provided to all meeting attendees, and published in the Journal of Huntington Disease (JHD). In addition, two abstract authors will be selected to formally present their research during a plenary session.

Dr. Erin Furr-Stimming, Co-Chair of HSG’s Publications Committee, said, “I have served on the Publications Committee since 2016 and have been incredibly impressed with the quality of the abstracts submitted. I always look forward to reviewing the exceptional science and innovation focused on advancing our understanding and treatment options for HD.”

The deadline for abstract submission is July 31, 2023. Eligible abstracts may include data that has been presented previously, though it should still be of public and scientific interest. Authors will be asked to disclose relationships with funding sources and manufacturers of commercial products discussed in the presentations. Abstract authors do not need to be HSG members. To learn more or submit your research for consideration, visit https://www.judgify.me/HSG2023.

If you would like to sponsor this year’s poster sessions, please contact us at info@hsglimited.org. For general sponsorship opportunities for the HSG 2023 annual meeting, please click here.

About Huntington’s disease

Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent.  Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.

The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network with thousands of members at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

To read the full press release, click here.

Neurocrine Biosciences Announces Publication of Full KINECT™-HD Phase 3 Study Results of Valbenazine for the Treatment of Chorea Associated with Huntington’s Disease in The Lancet Neurology 

  • Statistically Significant Improvement in Chorea Associated with Huntington’s Disease Seen as Early as Week 2
  • Chorea Improvement Supported by Statistically Significant Clinical Global Impression of Change (CGI-C) Response Status and Patient Global Impression of Change (PGI-C) Response Status Scores at Week 12
  • First Phase 3 Study to Implement Huntington’s Disease Health Index (HD-HI), a Patient-Reported Outcome Measure, Showed Reduced Disease Burden as Reported by Patients Receiving Valbenazine versus Placebo
  • Supplemental New Drug Application (sNDA) Filed, with Prescription Drug User Fee Act (PDUFA) Target Date Set for August 20, 2023

Neurocrine Biosciences, Inc. today announced that the complete study results from its Phase 3 KINECT™-HD study investigating valbenazine for the treatment of chorea associated with Huntington’s disease (HD) has been published in The Lancet Neurology online edition and will appear in the June 2023 print issue. The study met its primary and secondary endpoints, demonstrating a reduction in chorea symptoms associated with HD and improvement of overall chorea severity as noticed by clinicians and patients, with improvement seen as early as Week 2 of the initial dose in the 12-week study.
 
The KINECT-HD randomized double-blind, placebo-controlled Phase 3 study was conducted with 128 participants at 46 Huntington Study Group (HSG)–credentialed sites in North America.

The primary endpoint was a reduction in severity of chorea, the cardinal motor feature in HD, as measured by change in the HSG-developed Unified Huntington’s Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score from baseline to the average score at weeks 10 and 12. The TMC score is part of the motor assessment of the UHDRS that measures chorea. 
 
To read the full press release, click here.

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