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:

HSG Leadership Election Results

Andrew Feigin, Chair-Elect

Elise Kayson, Co-Chair-Elect

On behalf of the HSG Nominating Committee, it is with great pleasure that we announce that Andrew Feigin and Elise Kayson have won the run-off election for the HSG Chair and Co-Chair positions. We thank all of the candidates who ran for these positions and salute their enthusiasm and commitment to the entire election process. In addition, we thank each of you who attended the webinars, viewed the webpages, and took the time to cast your vote 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 outline an exciting future.

Andy and Elise 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.

Sincerely,

Anne Young

Chair, HSG Nominating Committee

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.

Ionis and Roche announce promising news on the Ionis-HTTRx trial

Ionis and Roche announced Monday, Dec. 11, that the Phase 1/2a Ionis-HTTRx trial reported no significant safety issues among participants. In addition, cerebral spinal fluid collected from participants during the trial showed a dose-dependent reduction in mutant huntingtin proteins in the fluid.

For more information, read the HD Buzz article about the announcement. More details about this early phase trial is lower in the article.

Click here to read the Ionis press release.

Click here to read an FAQ from HDSA

Click here to read the Washington Posts‘s coverage.

Click here to read an informative Q&A with Ed Wild.

Welcome new staff member, members of leadership team

The Huntington Study Group is pleased to announce a new staff member and new members of the leadership team. Anne Van Dusen, CHC, is HSG’s new Director of Education & Special Programs, Sarah Noonberg, MD, PhD, replaces Mike Poole, MD, on the executive committee, and Christopher Beck, PhD, replaces David Oakes, PhD, as HSG’s Director of Biostatistics/executive committee member.

“We are extremely fortunate to have Sarah, Chris, and Anne join our team,” said Ray Dorsey, MD, chair of HSG. “Sarah and Chris have a long-standing interest and record of service in HD. Anne brings much-needed expertise in training to help drive our educational efforts.”

Anne Van Dusen, CHC, Director of Education & Special Programs

Anne joined the HSG team in June of 2017 as the Director of Education & Special Programs. Anne is part of the Executive team and will develop and implement both educational and program plans that will sustain and help HSG to maintain its standing as a proven world leader in facilitating high quality clinical research trials and studies in Huntington Disease (HD). Anne holds a BS from the University of Binghamton School of Management and is certified in healthcare compliance by the Compliance Certification Board. Prior to joining HSG, Anne held several leadership roles in compliance, quality and finance, most recently at CCSI of Rochester, ACM Medical Laboratory and Lifetime Care Home Care and Hospice. Anne is passionate about trail running and is very active in the local running community.

Christopher Beck, PhD, HSG Director of Biostatistics

Chris is an Associate Professor of Biostatistics and Orthopaedics at the University of Rochester Medical Center. As a member of several national and international collaborative groups conducting research in areas including Huntington disease, Parkinson disease, Batten disease, bone repair, rheumatoid arthritis, and cardiomyopathy, Chris has extensive experience and a great deal of expertise in the biostatistical aspects of basic and clinical research. He joined HSG in 2005 while serving as a biostatistican for the TREND-HD study, and he has continued in that role for a total of eight HSG studies. Chris is honored to become only the second Director of Biostatistics in the history of HSG.

Sarah Noonberg, MD, PhD, Executive Committee Member

Sarah is currently the Chief Medical Officer at Prothena, a biotechnology company focused on the discovery, development, and commercialization of novel immunotherapies for diseases involving protein misfolding. Prior to joining Prothena, she served as Group Vice President and Head of Global Clinical Development at BioMarin Pharmaceuticals Inc., where she was responsible for clinical development programs using gene therapies, oligonucleotides, and recombinant proteins to address a diverse range of neurodegenerative, neuromuscular, metabolic, and hematologic diseases. Earlier in her career she led clinical studies in Huntington disease in partnership with the Huntington Study Group, as well as clinical studies in Parkinson’s disease, Alzheimer’s disease, prostate cancer, and cystic fibrosis. Sarah earned her MD at the University of California, San Francisco, her PhD in Bioengineering at the University of California, Berkeley, and her BS at Dartmouth College. She is a board-certified internist and works part-time as a hospitalist physician treating a broad range of inpatient and critical care patients.

Overnight Switch from Tetrabenazine to Deutetrabenazine Safe, Trial Shows

JAMA Neurology paper published today from ARC-HD Trial

People with Huntington disease-associated chorea can safely convert overnight from tetrabenazine to deutetrabenazine (brand name: Austedo), according to the results of the Alternatives for Reducing Chorea in Huntington Disease (ARC-HD) trial published yesterday in JAMA Neurology. The Phase III open-label, single-arm switch cohort of the trial was led by the Huntington Study Group (HSG) and the University of Rochester’s Clinical Trials Coordination Center (CTCC) on behalf of Teva Pharmaceutical Industries Ltd.

Although the topline results of the trial have been released previously, the peer-reviewed publication about the switch arm of ARC-HD clinical trial is now published in a premier neurological medical journal.

“This trial provides more good news for our patients who need options for medication to control their chorea,” said Samuel Frank, MD, HSG’s principal investigator of First-HD and director of the HDSA Center of Excellence at Beth Israel Deaconess Medical Center in Boston. “We are grateful to the people who volunteered in this trial and their families who supported their participation.”

The ARC-HD trial enrolled 37 patients who were on a stable dose of tetrabenazine for 8 weeks or longer. Participants converted from tetrabenazine to deutetrabenazine at a dose that was half of their original total daily tetrabenazine dose. After one week, investigators began weekly dose adjustments, if needed, to achieve optimal chorea control. This study was focused on safety. In addition, Total Maximal Chorea Score and Total Motor Score were evaluated as efficacy endpoints.

In April, the U.S. Food and Drug Administration (FDA) approved deutetrabenazine, the second drug approved for use in the United States to treat chorea in HD. The approval was based on positive results from the First-HD study, a Phase 3 clinical trial which was also led by HSG and CTCC on behalf of Teva Pharmaceuticals. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea. The results were published in JAMA, July 2016.

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.

“The deuterium chemistry can provide effective chorea control with fewer daily doses and with lower peak doses, potentially improving medication tolerance. These are both big benefits for our patients,” said Claudia Testa, MD, PhD, HSG’s co-principal investigator for ARC-HD and director of the HDSA Center of Excellence at Virginia Commonwealth University.

“It’s gratifying to see the current progress in treatments for people with Huntington disease. In addition to being grateful to the research participants who are a major driver in that progress, we are grateful to the sites and site staff whose dedication to seeking treatments that make a difference is unparalleled,” Testa added.

Much of the work that led to the completion of the ARC-HD trial was carried out by HSG, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, Australia, New Zealand, and South America 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.

Scientific, technical, logistical, and analytical support for ARC-HD 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.

# # #

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

Seeking Survey Participants

If you are someone with HD, or a family member, friend or caregiver of a person living with HD, please take a moment to complete this brief survey. The survey represents a collaboration between the Griffin Foundation, the Huntington Study Group (HSG) and the Huntington’s Disease Society of America (HDSA) in partnership with a broad group of organizations. We would like to understand YOUR experience – YOUR journey – and YOUR knowledge to help us improve access to quality care for HD patients and families in the United States. Your feedback is invited and welcomed!

Click here to complete the survey.

Help4HD Blog Radio features I am the DIFFERENCE campaign

Help4HD gave the Huntington Study Group the opportunity on its radio show to update the community on our many current projects, including the I am the DIFFERENCE campaign. Katrina Hamel, VP of Help4HD, hosted the show with guests Ann Nelson, an HD community member and participant in the campaign, and Heather Hare, HSG’s director of Communications & Outreach. Listen to the show by clicking here.

To learn more about HSG 2017: Elevating HD, click here.

To participate in the HD-HI survey mentioned on the show, click here.

Updates on the HDCIP survey are forthcoming.

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