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The research news is very encouraging. Although there are no results yet from ongoing clinical trials, there have been continued developments in basic science and animal research. - Marsha Miller, Ph.D. 
Dr.Christopher Ross remarked in his workshop "From Mouse to Man, "The research is moving so fast because unexpected things keep happening." Although the HDSA and other research groups have planned research, they also want to be flexible to foster serendipity. Dr. Ross noted that there are a variety of organisms now being used in HD research, including e. coli, yeast, drosophilia, c. elegans, and the mouse. There are ten mouse models of HD, four with part of the HD gene and six which use the whole gene. One way to measure progression in the HD mice is to look at abnormal rotarod performance. In this test, mice must move quickly to stay on rotating rods. They try to do this because once they fall off they receive a mild shock. Lifespan is also an outcome measure. Dr. Ross showed slides which demonstrated the improvement of motor performance in HD mice with creatine. The use of creatine also prolonged life span, not dramatically but significantly. He cautioned that results with the HD mice may or may not translate to humans. There is a need to test toxicity, figure out dosage, and find out effectiveness. He described the drug discovery pipeline. At the opening of the 'pipeline', are the high throughput assays in which the results of thousands of compounds can be analyzed fairly quickly. As we move to more complex organisms, the cost of the research increase. Mostly costly are clinical trials with human beings. And in addition, as we move through the pipeline there are more 'failures' as promising compounds fail to yield good results. Major HDSA research efforts are varied:
Jill Heemskerk of the NINDS (National Institute of Neurological Disorders and Stroke) has funded the screening of existing drug compounds which have already been approved for other illnesses. This is particularly exiting because if one or more prove to be effective, the path to FDA approval will be considerable shortened. Dr. Ross noted that over twenty mouse studies have been completed within the last year, with twenty more ongoing. Therapeutic agents being studied include:
He talked about current human trials. There is a double blind, randomized, placebo Phase III clinical trial involving 150 patients, some of whom will receie LAX 101 which is ethyl eicosapeninoate, a purified form of EPA. The data from this study should be ready for publication next year at this time. [One of the Laxdale researchers, a delightful man from Scotland, attended the conference.] Phase 1 or 2 clinical trials in humans are ongoing for
There are also observational studies to help researchers and clinicians better understand what goes on in HD: Predict HD and PHAROS. HDSA funding priorities, as established by the Board of Directors are 40 percent research towards a cure, 40 percent towards care, and 20 percent administrative costs. Expenditures under care include the centers for excellence, the chapters, education, and advocacy. Dr. Ross's vision for the future is:
Of course the question on everyone's mind is when. No one knows the future but Dr. Ross says, "Most of us believe that this is a solvable problem and that it can be solved within a reasonable period of time." That time can be shortened by devoting increased resources towards research. He concluded his talk by stating, "I think it's reasonable to believe that an effective treatment will be available within ten years." |
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Prepared for Huntington Disease Advocacy Center, Copyright © 2002.