On a Mission to Cure Huntington’s Disease: The Huntington Study Group’s 127 Investigator Site Network

Jan 1, 2020 | HD, Huntington Disease, Huntington Study Group, Research Network, Research Site

On a Mission to Cure Huntington’s Disease: The Huntington Study Group’s 127 Investigator Site Network

Huntington’s disease, an inherited disorder, results in the death of brain cells. Symptoms typically start between 30 and 50 years of age, but it can begin at any age. The disease may develop earlier in life in each successive generation. About 8% of the cases start before the age of 20, and often those with HD frequently underestimate the degree and severity of their problems. There is no cure. The mutation causing the disease lies in the HTT gene—representing the instructions for making a protein called huntingtin. Although scientists are not certain as to all of the protein’s roles, they know its somehow associated with brain development. A mutation in the gene causes the protein to clump together and somehow kill off neurons. A worldwide network was formed nearly two decades ago to combat this inherited disease. The Huntington Study Group (HSG) was formed in 1993 and represented the world’s first HD cooperative research organization.

Huntington Study Group (HSG) Background

Formed in 1993, HSG today positions itself as a world leader in facilitating high-quality clinical research trials and studies that bring hope closer to those struggling with HD to find an effective treatment that makes a difference—offering rigorous care initiatives and working to improving the quality of life and outcomes for HD families. With a vision “to be an invaluable research partner to families, HSG members, sponsors, and partners,” HSG is made up of families, HSG members, sponsors, and partners. It forms a network aligned worldwide to bring dedication, passion, and talent to find a cure to HD.

Key Accomplishments

Key HSG accomplishments include more than 30 clinical trials and studies since its inception. HSG developed the Unified Huntington’s Disease Rating Scale (UHDRS)—an important tool offering a uniform assessment of the clinical features and course of HD. Recently, HSG participated in a clinical trial that led to the first, and to-date the only FDA-approved drug (tetrabenazine) for the treatment of HD.


This drug was approved back in 2008 by the FDA to treat chorea associated with HD. Although some drugs had been used “off label,” tetrabenazine was the first approved such drug for Huntington’s disease in the United States. The drug is a treatment for chorea and not a cure for HD.

Current HSG Clinical Trials

HSG currently runs several clinical trials—some representing therapeutic trials (experimental or known compounds/drugs used), and some are observational studies (no drugs used). HSG stands ready to support potential participants to learn more. See the call to action later below. Studies include:

· Kinect HD



· The Pride-HD Study

· First-HD

· Arc-HD







HSG research publications can be accessed here.

HSG Clinical Investigational Sites

HSG credentials clinical research sites and can be termed Credentialed Research Sites, which are vetted by their HSG Credentials Committee. Presently, a total of 127 sites are in the HSG site network. They seek to ensure that these sites are qualified to perform Huntington disease clinical trials and studies in accordance with the organization’s high ethical and medical standards. Their value proposition—among others—is that the world’s leading HD experts and researchers participate. Their network of sites is worldwide with locations in America, Canada, Europe, and the UK, Australia, New Zealand, and Chile.

Site Qualification

New research centers and sites can join the HSG network and become a Credentialed Research Site. There is a site credentialing process and a two-step application process. Interested sites can learn more here

HSG HD Insights

A newsletter is accessible to all, simply register. HD Insights is a semiannual publication highlighting the latest HD research. The editors work to bring timely research in an accessible manner. The Editorial Board includes prominent HD-focused clinical investigators from Vanderbilt, Stanford, and others.

Lead Contact

Shari Kinel, JD, Executive Director

Call to Action: Interested in learning more about HSG? See the contact page.