News

JAMA publishes results of First-HD trial

FirstHD_Horizontal sm screen resIn case you missed the news last month, the Journal of the American Medical Association (JAMA) published the results from First-HD, an HSG trial run by co-PIs Dr. Samuel Frank and Dr. Claudia Testa at 34 HSG sites around the country.

The JAMA abstract is available here (the paper is available with a subscription). HSG’s announcement is available here.

Frank spoke with several medical reporters about the findings, including at Medscape, which published this piece about the findings.

Thank you to Frank, Testa, Elise Kayson, Jody Goldstein and Jacqueline Whaley and the rest of team at CTCC, our site investigators and coordinators, and especially to the trial participants and their caregivers.

Vaccinex Receives FDA Fast Track Designation for VX15 Antibody for the Treatment of Huntington’s Disease

vaccinex_logoVaccinex, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VX15 as a potential treatment for Huntington’s disease (HD).  VX15 is the Company’s novel clinical stage monoclonal antibody that blocks the activity of semaphorin 4D (SEMA4D), a molecule that is believed to promote chronic inflammatory responses in the brain.

Read the company’s press release.

Chorea Reduced by Deutetrabenazine in Study led by HSG


JAMA publishes First-HD study

People with Huntington disease experienced improvements in chorea while taking deutetrabenazine (SD-809) compared to placebo, according to a paper published today in the Journal of the American Medical Association (JAMA). Although the topline results of the trial have been released previously, the complete peer-reviewed publication about the First-HD clinical trial is now published in a premier medical journal.FirstHD_Horizontal sm screen res

Deutetrabenazine was investigated in the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Groupteva_cns_logo (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.

“Patients’ chorea and motor scores improved compared to placebo over the course of 12 weeks,” 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. “In addition, both the participants and their study physicians reported overall improvement.”

First-HD enrolled 90 patients at 34 HSG research sites between August 2013 and August 2014. The trial followed patients for 12 weeks on the medication and measured their chorea, as well as patients’ and clinicians’ impression of improvement.

Sam Frank resized

Frank

“As a physician who cares for people with HD, it’s gratifying to see positive results from a well-designed, fully enrolled trial. Until we find a cure, we aim to bring our patients more treatment options to relieve symptoms,” Frank said. “We are grateful to the people who participated in this trial and their families and support systems that made their participation possible. Research in the HD community depends on volunteers enrolling in trials.”

At the end of May, Teva Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) asked for more data on deutetrabenazine, which had been under review to treat chorea associated with HD. 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.

There is only one drug currently approved to treat chorea associated with Huntington disease: tetrabenazine. 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. This can translate into effective symptom control with fewer medication doses a day, lower total daily doses, and improved tolerance. In First-HD, both patient and clinician overall assessments were significantly better in the deutetrabenazine treated group compared to placebo after 3 months. The deutetrabenazine group improved in a quality of life measure while the placebo group worsened.

testa_VCU

Testa

“Overall status and quality of life measures are especially relevant in chorea, where no single number captures what is clinically meaningful to patients themselves,” said Claudia Testa, MD, PhD, HSG’s co-principal investigator for First-HD and director of the HDSA Center of Excellence at Virginia Commonwealth University. “It’s exciting to see how treating an HD symptom can make a real-life positive impact.”

Much of the work that led to the completion of the First-HD trial was carried out by the 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 First-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 http://www.ctcc.rochester.edu.

Teva Pharmaceutical acquired deutetrabenazine through 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 who completed First-HD, is also being led by the HSG 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.

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 Heather Hare, director of Communications & Outreach, at heather.hare@hsglimited.org.

What is HSG?

For HD Awareness Month, the Huntington Study Group is launching an education campaign, aimed at helping health care providers who are less experienced with Huntington disease (HD) to learn more about how to care for their patients and families with HD. HSG wants to share insights of our experienced HSG members with less experienced providers to help them better understand issues faced by HD patients and families. So, HSG is launching a new education section of our website with tools to help new HD providers.

 

  • There, you will find:
    • Videos addressing common questions providers new to caring for families with HD may have
    • A page of useful resources for providers, including care guides, referral resources, and links to many advocacy organizations
    • Annual Event education: HSG 2015 educational and research presentations
    • The underlying message is that HD is a treatable disease
    • A video explaining what HSG is
Please share this new section of our website with the providers who call you for help and let us know if you have more ideas for resources we can add. If you are on social media, we encourage you to share all of this information with your networks to ensure the word gets out there. If you’re a hashtag user, we’ll be using both #hdisatreatabledisease and #letstalkabouthd (HDSA’s hashtag) throughout May, HD Awareness Month.

HSG adds board member Joni Steinman

Joni SteinmanAs our newest board member, Joni M. Steinman brings 35 years of health care management consulting experience to advancing the goals of the HSG. As the co-founder and managing principal of AUSMS Healthcare Consultants, Steinman served the U.S. health care industry, advising policy makers, administrators and practitioners on strategic, facility, organizational and business planning and development matters, including performance improvement initiatives for hospitals, surgery providers and practitioners, hospice and home care agencies, medical groups and governmental agencies, regarding the development of many facets of acute, sub-acute, ambulatory, home and long-term care.  In 2012, after completing several consulting engagements in Toronto, Canada, Steinman affiliated with Strategic Interests, LLC, a Rochester, NY-based firm dedicated to guiding health care organizations nationwide to grow through improved performance and the innovative use of information technology and business intelligence.

With educational achievements in both political and medical sociology (Brandeis University, cum laude) and social work administration (San Diego State University), Steinman has devoted her career, as AUSMS’ motto indicates, to guiding clients and colleagues alike ‘From Ideas to Results’ in health care and human services. And as the sister of a Nobel Laureate in Physiology and Medicine, Steinman grew up as a close and ever-fascinated observer to the critical impact that the pursuit of and commitment to scientific research can have on some of our most vexing medical challenges.

Steinman has always given back to her communities through voluntarism and philanthropy. Examples of her commitment including serving as President of, respectively, the Health Systems Agency of San Diego and Imperial Counties, the Jewish National Fund’s San Diego Region and San Diego Women in Health Administration.  Today, in addition to her new commitment to HSG, she is a member of the Rochester Women’s Giving Circle, a community of philanthropic women who combine their financial strength to support area women and girls on their journey to economic self-sufficiency.

 

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 Heather Hare, director of Communications & Outreach, at heather.hare@hsglimited.org or 585-242-0277.

SIGNAL completes enrollment in cohort A

vaccinex_logoVaccinex Inc. completed enrollment of cohort A in its SIGNAL Clinical Trial, a randomized Phase 2 study to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503 in subjects at risk for or with early signs of Huntington’s Disease. Read the press release.

FDA wants to hear from you!

FDAThere’s still time and opportunity to tell the FDA your thoughts on drug development for Huntington disease!

The FDA focused on hearing from individuals with HD, family members and caregivers at its Sept. 22 Patient Focused Drug Development hearing. But from now until Nov. 23, the FDA is taking input from the entire HD community, including care providers and researchers. Specifically the FDA wants to know: At all stages of HD/JHD, if a treatment could be developed, what symptom would you most like to stop or what ability would you like to regain – what is most important to you?

The other topics about which the FDA is gathering input include the symptoms that affect daily life, how the disease affects social interactions and relationships, and how treatments have changed over time. To see the full list of topics, click here.

This is your chance to advocate on behalf of HD families and underscore with our country’s drug development process how imperative it is to address the many symptoms of HD. Please take advantage of this opportunity.

To submit your comments, click here and then click on the green “Submit a Formal Comment” button on the right side of the screen.

 

Regional Health Care Practitioners Invited to Learn More about Huntington Disease Oct. 23

Day-long course offering continuing education credits through USF

The Huntington Study Group (HSG), in support of the goal to make care for Huntington disease (HD) patients more available, is holding a day-long education opportunity for health care providers in Tampa on Friday, Oct. 23. “Practical Pointers and Perspectives on Huntington Disease for Local Practitioners” is part of HSG’s annual research and educational event, which, this year, is called HSG 2015: Building Our Future.

HD is uncommon, but not all that rare. Clinicians, nurses, social workers, etc., specializing in neurology, movement disorders, mental health, genetics and palliative care, as well as primary care providers, are likely to see HD patients and families who need their guidance through the complex and progressive course of the disease. This course seeks to empower providers with the knowledge and resources they need to care for HD patients.

The course, provided jointly by USF Health and HSG, is designed for health care providers in neurology and general clinicians, which has been approved for continuing education credits. The fee is $100 per registrant and includes CE credits.

Attendees will leave with:

  • An up-to-date understanding of the diagnosis, staging and typical symptoms of HD
  • Skills for expert approaches to treatment
  • Understand the role of allied health in the management of HD
  • Awareness of the available medical, education and support resources

Register here!

More details can be found here.

For more information:

Heather Hare

(585) 242-0277

Heather.Hare@hsglimited.org

 

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