H.S.G
HuntingtonStudyGroup

The Huntington Study Group (HSG) is an international consortium of scientific investigators from academic and research centers who are committed to the cooperative planning, implementation, analysis and reporting of controlled clinical trials and of other therapeutic research for Huntington's disease (HD) and related disorders. The HSG aims to advance knowledge about the cause, progression and clinical impact of HD and related disorders and to develop and implement scientific strategies to examine promising therapeutic interventions. The HSG is committed to the principles of open scientific communication, peer review, full disclosure of potential conflicts of interest, and democratic governance of its organization and activities.

Through the support of non-profit HD organizations (Huntington's Disease Society of America, Foundation for the Care and Cure of Huntington's Disease, Huntington Society of Canada, Hereditary Disease Foundation) and the University of Rochester and other HSG sites, the HSG has continued to advance the experimental therapeutics for HD to develop multi-center controlled clinical trials and other research studies in keeping with its scientific standards and therapeutic mission.

The research activities and objectives of the HSG provide an opportunity to train investigators and staff in experimental therapeutics and the conduct of controlled clinical trials related to HD. Efforts have been made to include young investigators in the planning and conduct of clinical trials and other research studies, thereby fostering the development of the necessary interest and investigative skills to sustain a successful research program.

As of May 1999, there are 55 HSG research sites representing more than 200 clinical investigators, coordinators, and scientists in the U.S., Canada, Europe, and Australia. Participation in the HSG is based primarily on the contribution of investigators and coordinators to the UHDRS database and participation in multi-center trials. Through the commitment of its participants and the support of their institutions and the HD organizations, the HSG has enjoyed continuing achievements and growth with plans to launch major research initiatives in 1999 and into the new millennium.

Expansion of the HSG

Based on the growing international interest in the research activities of the HSG and the launching of the PHAROS and PREDICT-HD studies, it is anticipated that the consortium will continue to expand in 1999 to include additional sites and investigators. The HSG Executive Committee will continue to oversee its activities and projects. Steering committees, under the leadership of principal investigators and with the backing of their institutions, will continue to be responsible for the development and conduct of controlled clinical trials. The HSG anticipates that the HD organizations will continue their strong commitment to and support of the HSG.

Three Year Plan (1999-2001)

Over the next three years, the HSG intends to expand its core efforts in the conduct of multi-center therapeutic trials. The planning of a trial with riluzole is well underway. Trials of other agents that selectively retard glutamatergic neurotransmission or enhance bioenergic functions are being developed. The development and implementation of the PHAROS and PREDICT-HD projects emphasize our commitment to develop preventative therapeutic trials aimed at postponing the clinical onset of HD in healthy gene carriers.

The centerpiece of our three-year plan is implementation of the PHAROS and PREDICT-HD studies to measure the diagnostic accuracy and neurobiologic correlates of the clinical onset of HD. These studies are groundbreaking, involving prospective and objective assessment, unprecedented measures to protect confidentiality, well-controlled (double-concealed) conditions, and correlative DNA analyses. These studies are a necessary prelude for ultimately developing neuroprotective interventions aimed at postponing or preventing the onset of HD in healthy HD gene carriers.

In addition, the HSG Scientific Advisory Committee has recommended that potentially neuroprotective therapies for HD be tested systematically in genetic and animal models in order to identify the best therapeutic candidates to clinical trials. The HSG is eager to make these animal models available to scientists who are capable of carrying out experimental therapeutic studies. The HSG study proposal "Preclinical Assessments of Potential Therapeutic Agents in Huntington's Disease", supported by the Hereditary Disease Foundation (Santa Monica, CA) under the direction of Anne Young, MD, PhD (principal investigator), involves preclinical studies of experimental drugs in animal models of HD. This study is focused on therapeutic agents that are not currently under development by the pharmaceutical industry.

The amassed clinical and scientific expertise in the HSG bring an unparalleled, scientifically-based rationale for new therapies for HD. Our investigators, coordinators, and Coordination/Biostatistics Centers also provide streamlined and experienced resources for the efficient assessment of promising therapeutic interactions. In the long run, we remain confident that these human and scientific resources will help to develop more substantive therapies for individuals who carry the HD gene.

Investigator/Coordinator/Consultant Network

More than 200 clinical investigators, coordinators, scientists and staff make up the consortium of HSG sites. The HSG is guided by an Executive Committee and input from site participants, a Scientific Advisory Committee and the HD organizations.

The HSG membership includes experienced and well-regarded clinical investigators, coordinators and basic scientists who have provided their professional time and institutional support to the success of the HSG and its research aims. In 1997, the HSG began to develop a close working relationship with the Coalition for the Cure, a research consortium of basic scientists supported by the Huntington's Disease Society of America (HDSA) who work to advance our fundamental knowledge base of HD therapeutics.

The office of the HSG is located at the institution of the chair, Ira Shoulson, MD, of the HSG Executive Committee, which is currently at the University of Rochester, 1325 Mt. Hope Avenue, Suite 160, Rochester, NY 14620. Aileen Shinaman, JD, serves as the HSG Executive Director and Elise Kayson, MS, RNC, serves as the HSG Senior Project Coordinator.

Coordination Center

Since 1993, the HSG Coordination Center at the University of Rochester has been responsible for the overall coordination of the research activities including data management for HSG clinical trials. Directed by Cornelia Kamp, MBA, the Coordination Center's research operations involve about 45 individuals, including postdoctoral fellows in experimental therapeutics, who help oversee implementation of study protocols, site communications, and data management.

Biostatistics Center

The Biostatistics Center of the HSG is comprised of individuals from the Department of Biostatistics (Chair, David Oakes) at the University of Rochester Medical Center. David Oakes, PhD, Michael McDermott, PhD, and Hongwei Zhao, DSc, serve as the chief biostatisticians of HSG trials. In addition to an 11-member faculty, a group of 12 programmers and research associates are available to assist in the analysis of the HSG databases. Like the Coordination Center, the Biostatistics Center participates in the training of pre- and post-doctoral students interested in controlled clinical trials.