Request for Information: Soliciting Input on Huntington’s Disease Best Practice Guidelines for Therapeutic Target Validation

Notice Number: NOT-NS-10-005

Key Dates
Release Date:    December 9, 2009
Response Due By: January 29, 2010

Issued by
National Institute of Neurological Disorders and Stroke (NINDS)


This is a time-sensitive Request for Information (RFI) issued by the National Institute of Neurological Disorders and Stroke. The purpose of this RFI is to solicit recommendations on the best practices for animal studies on Huntington’s disease that could advance knowledge and validation of therapeutic targets for disease treatment. The Institute envisions that standardization of animal studies through identification of common protocols, outcome measures and breeding practices will enable comparison of results across studies and targets. Best practice guidelines should be inclusive of in vivo target manipulation through either genetic or pharmacological interventions. The responses to the RFI will be collected and evaluated by a group of Institute staff to assess how best to meet the needs of researchers in the field. Results of that evaluation will be made available to the public and will be part of a larger effort by NINDS to develop best practice guidelines for target discovery and validation for neurological diseases.


Huntington’s disease is a rare neurodegenerative disorder, which like many neurodegenerative diseases, currently has no treatment options to modify disease progression. The gene for Huntington’s disease was discovered in 1993, and genetic testing can now identify individuals carrying the pathological expansion of the CAG repeat in exon1 of the gene, prior to the onset of motor symptoms. Knowledge of the genetic basis for the disease enabled the development of numerous animal models for Huntington’s disease and the discovery of multiple cellular pathways associated with disease pathogenesis.  The field currently faces the challenge of determining effective criteria to identify the best targets for therapy development, taking into consideration the number of potential targets, the expense associated with preclinical drug development, and the limited patient population available for clinical trials.

This RFI is meant to solicit input from the extramural research community, scientific and medical organizations, Federal Agencies, and other interested parties well-versed in Huntington’s disease and the challenges facing therapeutic target identification and validation for this disease. Responses to this RFI will be reviewed by NINDS staff and will help inform and complement their assessment of best practice guidelines that will be utilized in the development of initiatives and review criteria involving standardized animal studies for Huntington’s disease therapeutic target validation. Following publication of the results of this RFI, and through ongoing interactions with the Huntington’s disease research community, it is anticipated that development of these guidelines will help to advance and transform the field overall. Best practice guidelines are not meant to limit the number of outcome measures studied, but rather to standardize a core set of outcome measures that can be applied to all studies focused on therapeutic target validation for Huntington’s disease. This RFI is for planning purposes only, and should not be construed as a solicitation for applications or proposals and/or as an obligation in any way on the part of the United States Federal Government.

Information Requested

The mission of NIH is to support biomedical discoveries that will improve health and save lives. To further the development of therapeutic target validation for Huntington’s disease, the NINDS would like input on the following questions:

Best Practice Guidelines

 Animal Models

Study Design

Outcome measures

Standardized protocols

General Information


Responses must be submitted electronically using the web-based format (, and will be accepted through January 29, 2010.  Replies to individual questions are optional, and the site will permit anonymous responses. The information provided will be analyzed and may appear in various reports.


Specific questions about this RFI should be directed to the contact listed below:

Margaret Sutherland, PhD
Program Director, Neurodegeneration
6001 Executive Blvd., Rm 2222
Bethesda, MD 20892
Phone: 301-496-5680
Fax: 301-480-1080


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