NINDS Neurodegeneration Study

NINDS Neurodegeneration Study

From the Spring 2003 issue of Hopes & Dreams, newsletter of the Illinois Chapter, Huntington's Disease Society of America. Reprinted with permission from the HDA-UK Newsletter. Edited by S. Clingerman, Ph.D.

For the past year, some 30 investigators have been screening a library of approved drugs for agents that are effective against neurodegenerative diseases, thanks to a joint program of drug discovery sponsored by the National Institute of Neurological Diseases and Stroke (NINDS), the Hereditary Disease Foundation (HDF), the Huntington's Disease Society of America (HDSA), and the Amyotrophic Lateral Sclerosis Association (ALSA). Of the 30 investigators funded, 15 are working on Huntington's disease.

This unusual collaborative research initiative, announced on May 8, 2001, was developed in response to recommendations of the NINDS Strategic Planning Panel on neurodegeneration, which was chaired by HDF scientific Director Allan Tobin and with HDF President Nancy Wexler as a member. The initiative provided supplemental funding for scientists already funded by NINDSD grants Approximately $1.3 million was committed by the four organizations for this program. Dr.Jill Heemskerk, program director of neurodegeneration at NINDS, said it was "tremendously valuable" to have the voluntary agencies participating in this program, because they contributed additional funds and because they permitted the participation of investigators not currently funded by NIH grants. These supplemental grants facilitated the testing of a pre-defined set of compounds in models of Huntington's Disease, ALS, and other neuro-degenerative diseases.

One of the unique aspects of this initiative was a requirement that scientists share their results. Data are being entered into a database maintained by NINDS. A workshop held in April, 2002 enabled investigators to compare their results with those obtained using other models and assays and to evaluate and prioritize the effectiveness of tested compounds
for eventual inclusion in clinical trials.

An article about the workshop can be found in the May 9, 2002 issue of the journal Nature (online at NINDS has been excited by the positive response of investigators to this aspect of the program, said Heemskerk. “Hereditary Disease Foundation has a good history of getting people to share information, and we are pleased to see that this spirit is shared by the broader community."

Approximately 1,000 drugs were tested, about three-quarters of which are already approved by the US Food and Drug Administration (FDA). The others were chosen because they showed some influence on brain activity. A number of drug leads have , arisen from this collaboration and, according to Heemskerk, “some (compounds) are already being prepared for follow-up studies in mice." The goal of this collaboration is to move therapies into clinical trials based on the "hits" from this screen. "We would like to push that through clinical trials in an active way, and see that it gets put into patients as soon as possible," added Heemskerk.

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Created: Aug. 11, 2003 Last Updated: Nov. 14, 2010