News

HD Insights – Autumn Issue

The latest issue, Volume 20 of HD Insights has arrived and available online for viewing/downloading. This first edition of HD Insights version 2.0 includes several visual, style and content updates from editor Daniel Claassen, MD and deputy editor Sara LaJeunesse.

In this issue:

HSG Scholarship Winners Receive Funding to Attend Premier Huntington’s Disease Conference

The Huntington Study Group (HSG) announces the 2018 CME4HD Online scholarship winners who have been awarded funding to attend its renowned annual meeting, HSG 2018: Unlocking HD.

Maryluz Camargo Mendoza, Ph.D

Addie Patterson, D.O

Maryluz Camargo Mendoza, Ph.D, associate professor at the National University of Colombia, Bogota, has received a scholarship for referring 49 people who successfully completed five CME4HD Online modules. Addie Patterson, D.O., associate professor at the University of Gainesville won the random drawing of 133 participants who completed five modules successfully. Both scholarship winners receive airfare, hotel and a travel stipend to attend the conference, held at ZaZa Hotel in Houston in November.

CME4HD Online is a continuing medical education program designed to teach healthcare providers how to care for and manage individuals with Huntington disease (HD). Participants gain the knowledge, tools,and resources needed to provide quality care to families impacted by HD, while learning from worldwide HD experts who teach the course. Topics and discussion include symptoms and diagnosis, HD treatments, ethical dilemmas in genetic testing, strategies for disease management, translation of complex clinical information for families and the role of ancillary services in the management of HD.

The CME4HD referral program encourages sharing and completion of the program modules to help educate medical professionals about Huntington’s Disease in order to help patients receive better care between sub-specialist appointments.

CME4HD Online program was first shared at HSG’s annual event and is now available online free of charge. The program was planned and implemented by HSG in partnership with the North American Center for Continuing Education, LLC (NACCME). This program was made possible, in part, by an independent educational grant from TEVA Pharmaceuticals.

The Huntington Study Group was formed in 1993 and is the world’s first HD cooperative therapeutic research organization. Twenty five years later, HSG is a world leader in facilitating high quality clinical research trials and studies that work toward finding effective treatments for HD. HSG has the first and largest HD clinical research network of more than 400 active and compassionate investigators, coordinators, scientists and HD experts at more than 120 credentialed research sites around the globe. HSG members are world experts in caring for individuals and families impacted by Huntington disease. Visit https://huntingtonstudygroup.org/ for more information.

Dr. Nicolò Zarotti Named Recipient of the Peter Como Scholarship

The Huntington Study Group (HSG) is pleased to announce the that Dr. Nicolò Zarotti has been named recipient of the Peter Como Scholarship to attend the renowned annual meeting.

Dr. Zarotti obtained his Bachelor’s and Master’s degree in Psychology in Italy, and later joined Lancaster University (UK), where he completed his PhD focused on emotional processing and communication in Huntington disease (HD). Dr. Zarotti is currently part of a team collaborating with the British Psychological Society to produce new guidance on psychological approaches to the understanding and treatment of psychological difficulties in people with motor neurodegenerative conditions such as HD.

The Peter Como Scholarship, named in memory of one of HSG’s founders and biggest supporters, is awarded annually for a professional in the mental health field to attend HSG 2018: Unlocking HD. The Como Scholarship includes two nights’ lodging, travel funding, and a modest stipend.

The Huntington Study Group was formed in 1993 and is the world’s first HD cooperative therapeutic research organization. Twenty-five years later, HSG is a world leader in facilitating high quality clinical research trials and studies that work toward finding effective treatments for HD. HSG has the first and largest HD clinical research network of more than 400 active and compassionate investigators, coordinators, scientists and HD experts at more than 120 credentialed research sites around the globe. HSG members are world experts in caring for individuals and families impacted by Huntington disease. Visit https://huntingtonstudygroup.org/ for more information.

Show off your credentials

HSG credentialed research site sealHSG is now offering credentialed sites the opportunity to display their ability to participate in clinical research by putting an electronic badge on their website. Northwestern University Parkinson’s Disease & Movement Disorders Center was the first to add the badge to their site. Check it out here.

The badge tells families, staff and applicants to your program that you have received all necessary training and are participating in clinical research for Huntington disease.

If you would like to put the badge on your website too, please contact HSG at +1 800-487-7671 or info@hsglimited.org.

FDA Requests More Data on Potential New Treatment for Huntington Disease

tevahomeTeva Pharmaceuticals Industries announced yesterday that the U.S. Food and Drug Administration (FDA) has asked for more data on SD-809 (deutetrabenazine), which is currently under review to treat Huntington disease (HD), a rare, inherited neurodegenerative disorder.

The request for more data is common when the FDA is asked to approve new medications, and this is the first deuterated compound to be reviewed by the FDA. Michael Hayden, M.D., Ph.D., Teva’s president of Global R&D and chief scientific officer said Teva plans to respond to the request in the third quarter of 2016.

Deutetrabenazine was investigated in the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Group (HSG) on behalf of Teva Pharmaceutical Industries. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience.

“We are grateful to the patients and families who have participated in First-HD and helped us get to this point. HSG will continue its role in the clinical development of this product with TEVA,” said Samuel Frank,Sam Frank resized M.D., Huntington Study Group’s principal investigator for First-HD and a movement disorders specialist at Beth Israel Deaconess Medical Center. Huntington Study Group’s co-principal investor is Claudia Testa, M.D., Ph.D., associate professor of Neurology at Virginia Commonwealth University.

Most individuals with HD experience chorea during the long course of the disease, which averages 15-20 years. Huntington disease is an autosomal-dominant, inherited disease that usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others don’t experience the diseases symptoms until much later in life. The disease is caused by the death of brain cells known as medium spiny neurons, which are killed off by a mutant protein. The disease brings with it an array of symptoms besides chorea, including cognitive problems, changes in personality, and psychiatric problems like depression. Because HD is autosomal dominant, each child of a person with HD has a 50 percent chance of inheriting the disease. For more information about HD, visit www.huntingtonstudygroup.org.

Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. Much of the work that led to the completion of the First-HD trial was carried out by the Huntington Study Group, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, and Australia who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information about the Huntington Study Group, visit www.huntingtonstudygroup.org.

“While disappointed with the delay, we remain hopeful and optimistic that the FDA will soon approve the second treatment for HD,” said Ray Dorsey, M.D., chair of the Huntington Study Group and director of the University of Rochester’s Center for Human Experimental Therapeutics (CHET).Ray Dorsey

First-HD was conducted at 34 Huntington Study Group sites across the United States and Canada, enrolling 90 participants over 14 months, in the 13-week double-blind, placebo-controlled trial. Scientific, technical, logistical, and analytical support for the study was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders. For more information about the Clinical Trials Coordination Center, visit www.ctcc.rochester.edu.

Teva Pharmaceutical owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals last year. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.

A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This trial, which includes participants from First-HD, is also being led by the Huntington Study Group and the Clinical Trials Coordination Center for Teva Pharmaceutical Industries. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.

For media inquiries, contact contact HSG at +1 800-487-7671 or info@hsglimited.org.

World Experts in HD to Convene in Nashville

HSG 2016 with dateTraining opportunities, family education available at HSG 2016

The Huntington Study Group (HSG), an international, non-profit network of more than 400 researchers and clinicians dedicated to seeking treatments that make a difference for Huntington disease (HD), is hosting its annual forum, HSG 2016: Discovering Our Future, Nov. 2-5 in Nashville for training and education and for presentation of new research findings and treatments to the worldwide community. The event, which is expected to draw over 300 attendees, is being held at the Gaylord Opryland Resort and Conference Center.

Among the highlights of the event are:

  • UHDRS Symposium, which focuses on a proposed modified Unified Huntington Disease Rating Scale and its use in clinical trials as an efficacy endpoint.
  • HD Innovators Forum, which includes presentations from leading HD industry partners working on developing new HD treatments.
  • CME4HD, a day-long, in-person continuing medical education course for healthcare providers hosted by HSG and Vanderbilt University School of Medicine. The course will teach providers how to care for and manage individuals with HD. Participants will have the opportunity to earn 8.75 AMA PRA Category 1 CME CreditsTM.
  • Keynote speech by Dr. Bob Beall, former CEO of the Cystic Fibrosis Foundation, who will talk about how CFF developed its care and research models in a non-profit setting through partnering with families, care providers, researchers, and biotech.
  • HD Research Symposium, highly acclaimed research presentations drawing worldwide recognition.
  • HD Family Education Day, designed for the local HD community and family members. The day starts with hearing about the latest in HD research, followed by interactive workshops and breakout sessions.

Registration costs vary on participation, but HD Family Education Day is free for HD community members. Please visit www.huntingtonstudygroup.org for more information and to register.

For more information:
+1 800-487-7671 or info@hsglimited.org

# # #

FDA Approves Second Drug for Huntington Disease Symptom

Frank

The U.S. Food and Drug Administration (FDA) today approved SD-809 (deutetrabenazine), the second drug approved for use in the United States to treat chorea in Huntington disease (HD), a rare, inherited neurodegenerative disorder.

The approval was based on positive results from the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Group (HSG) on behalf of Teva Pharmaceuticals. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience. The results were published in JAMA, July 2016.

“We are so grateful to the patients and families who made this development possible by participating in this ground-breaking trial. Trial participants are the key to bringing new treatments to the entire HD community,” said Samuel Frank, MD, Huntington Study Group’s principal investigator for First-HD and associate professor of Neurology at Beth Israel Deaconess Medical Center/Harvard Medical School. Claudia Testa, MD, PhD, associate professor of Neurology at Virginia Commonwealth University served as the co-principal investigator.

Testa

“It’s exciting to offer a new treatment,” Testa said. “Trials like these give patients, families, and care providers more options to effectively manage HD symptoms and improve quality of life.”

Most individuals with HD experience chorea during the course of the disease. Huntington disease is an autosomal-dominant, inherited disease that usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others experience disease symptoms much later in life. The disease brings with it an array of symptoms besides chorea, including dystonia, cognitive problems, changes in personality, and psychiatric problems like depression. Because HD is autosomal dominant, each child of a person with HD has a 50 percent chance of inheriting the genetic change that causes the disease from their affected parent, whether that parent is their mother or father. For more information about HD, visit www.huntingtonstudygroup.org.

Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. Deutetrabenazine is the first FDA approved compound with deuterium substitution. Much of the clinical work that led to the approval of deutetrabenazine was carried out by the Huntington Study Group, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, and Australia who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information, visit www.huntingtonstudygroup.org.

“This is a great day for the HD community,” said Ray Dorsey, MD, chair of the Huntington Study Group and director of the University of Rochester’s Center for Human Experimental Therapeutics (CHET). “The unmet need for therapeutics for individuals with HD is immense, and this approval brings us closer to making HD an increasingly treatable condition.”

First-HD was conducted at 34 Huntington Study Group sites across the United States and Canada, enrolling 90 participants over 14 months, in the 13-week double-blind, placebo-controlled trial. Scientific, technical, logistical, and analytical support for the study was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders.  For more information about the Clinical Trials Coordination Center, visit www.ctcc.rochester.edu.

Teva Pharmaceuticals owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals in 2015. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.

A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This open-label trial is also being led by the Huntington Study Group and the Clinical Trials Coordination Center for Teva Pharmaceuticals. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.

Media inquiries:

Huntington Study Group — +1 800-487-7671 or info@hsglimited.org

 

2018 HSG Election

Dear HSG Members,

Below is the content of an email that all HSG members should have received today. If you didn’t receive it, please check your spam folder and mark it a “safe sender” so you receive future important communications from HSG. If you have any trouble logging in, please contact HSG at +1 800-487-7671 or info@hsglimited.org.

Huntington Study Group is honored to announce the Chair and Co-Chair candidates for the 2018 HSG election. It’s our 25th anniversary, and the candidates represent the best of what our growing and active membership has to offer. They are world experts in Huntington disease care and research. They are leaders and collaborators. They are the future of HSG.

Voting begins Jan. 26.

Each nominated pair was approved by the nominating committee and has submitted a Joint Vision Statement and CVs for voter consideration. To read them, click on the candidate pairs below (Chair/Co-Chair):

In addition, the candidate pairs will present their vision and take questions from the membership during webinar forums at the times listed below:

  • 9 am ET 1/15/18: Webinar Forum with Andrew Feigin and Elise Kayson (Click here for details)
  • 12 noon ET 1/15/18: Webinar Forum with Claudia Testa and Samuel Frank (Click here for details.)
  • 9 am ET 1/16/18: Webinar Forum with Blair Leavitt and Christopher Ross (Click here for details.)
  • 12 noon ET 1/17/18: Webinar Forum with Blair Leavitt and Christopher Ross (Click here for details.)
  • 9 am ET 1/18/18: Webinar Forum with Ralf Reilmann and Bonnie Hennig-Trestman (Click here for details.)
  • 9 am ET 1/19/18: Webinar Forum with Claudia Testa and Samuel Frank (Click here for details.)
  • 12 noon ET 1/19/18: Webinar Forum with Andrew Feigin and Elise Kayson (Click here for details.)
  • 9 am ET 1/22/17: Webinar Forum with Martha Nance and Jody Corey-Bloom (Click here for details.)
  • 12 noon ET 1/22/18: Webinar Forum with Ralf Reilmann and Bonnie Hennig-Trestman (Click here for details.)
  • 1 pm ET 1/23/18: Webinar Forum with Martha Nance and Jody Corey-Bloom (Click here for details.)

If you’d like to submit your questions for candidates ahead of time, please contact Heather Hare: heather.hare@hsglimited.org or +1 (585) 242-0277. Your questions can be asked of all candidates or specific candidates, on your behalf or anonymously.

PLEASE NOTE: You may only vote once and cannot change your vote once it is submitted.

Important Dates:

  • 12:01 am 1/26/18: Voting begins
  • 12:01 pm 2/9/18: Voting ends

Elections for the Chair and Co-Chair take place every four years by electronic ballot, with the majority of total votes received from HSG members required for election. The Chair and co-Chair team will be entitled to compensation for their time and effort.

  • Click here to read the Chair job description.
  • Click here to read the Co-Chair job description.

If no pair of candidates earns a majority of the votes in the election, HSG will hold a run off election in February between the two pairs of candidates who received the most number of votes.

Huntington Study Group Welcomes New Leadership May 1

The Huntington Study Group (HSG) is pleased to announce that Andrew Feigin, MD, and Elise Kayson, MS, ANP, have been elected as the chair and co-chair, respectively, of HSG, a world-wide network of Huntington disease (HD) researchers. Both Feigin and Kayson have dedicated their careers to the clinical care of patients and families and research in HD.

Feigin and Kayson will begin their four-year term as chair and co-chair May 1, 2018, succeeding Ray Dorsey and Blair Leavitt, who along with Julie Stout, Joni Steinman, and Shari Kinel, expertly led HSG through the last four years. They were democratically elected by HSG’s world-wide membership of more than 500 investigators, coordinators, and other researchers and care providers.

Feigin, a Professor of Neurology at NYU Langone Health and co-director of the Marlene and Paolo Fresco Institute for Parkinson’s and Movement Disorders, has been involved in the care of HD patients and research since his participation in the Venezuela Collaborative Research Group, which isolated the HD gene 25 years ago. Kayson, Director of Clinical and Strategic Initiatives at the University of Rochester’s Center for Health + Technology (CHeT), has been involved in the care of HD patients and research since the inception of the HSG and was one of the founders of the organization.

Feigin’s independent research has focused on the development of novel imaging biomarkers that could be used as outcome measures for HD clinical trials. He has served in many HSG leadership positions over the past 20 years, including as a member of the Executive Committee, chair of the Program Committee for the HD Clinical Research Symposium for five years, and currently chairs the Clinical Research Advisory Committee. Feigin is the principal investigator (PI) of the SIGNAL trial and the co-PI of LEGATO-HD, and has served as a site PI on numerous other HD trials. “I am honored and excited to serve as the Chair of this amazing organization as the most exciting new potential therapies for HD are now entering human trials,” Feigin said.

Prior to leading CHeT’s Clinical and Strategic Initiatives, Kayson was the Director of Project Management for the Clinical Trials Coordination Center (CTCC) at the University of Rochester and previously worked in industry. In addition, Kayson’s long involvement in all aspects of more than 50 clinical trials and the FDA approval of the only two drugs for HD gives her a deep understanding of clinical trial design, organization and conduct, and insights into and appreciation of HD clinical trials from the perspective of study participants to coordinators, investigators, CRO functions, and sponsors. She has served in many leadership positions in HSG, including as a member of the Executive Committee, co-chair of the HSG Credentials Committee, and co-chair of the HSG Educational Committee. “It is exciting to be part of the momentum of research in HD. I am honored to serve as the HSG Co-Chair and look forward to reaching the goal of finding treatments that make a difference for our patients and families,” said Kayson.

HSG appreciates all the candidates who ran for these positions and salutes their enthusiasm and commitment to the entire election process. In addition, we thank each of our members who attended the webinars, viewed the webpages, and took the time to cast votes for this important election.

As HSG begins our 25th year of seeking treatments that make a difference, let’s join in congratulating our new leadership and wishing them the very best as they pursue their vision of partnerships, innovation and education to bring an exciting future to HSG.

New HD Insights Is Out (Vol. 19)

 

The new issue of HD Insights (Vol. 19) is out. Click here to read the latest news in HD research.

In this issue:

1 2 3 4 5