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The Research Pipeline: Overview
Basic research into what goes wrong in the brain in Huntington's continues to provide insights into how the disease might be treated.
The Huntington's Disease Society of American funds basic research through its Coalition for the Cure and its Research Grants and Fellowship Program. Follow the progress of HD researchers through our Research News Update and in our Research Reports archive.
Translational research: drug discovery, development, and design
As targets are identified, existing drugs and supplements known to address that target are reviewed and drug development efforts are instituted.
Translational research: preclinical testing
Promising drugs are tested in animal models such as drosophilia (fruitflies) and mice engineered to get Huntington's Disease. If the results are positive, a drug will be rigorously tested in one or more of the mouse models of HD. If a drug is effective and side effects are tolerable, toxicology studies are done and the drug can proceed to clinical trials.
Clinical and observational trials
Once a drug is ready for testing in human patients, it must proceed though three phases of clinical trials. A drug must be shown to be safe, well-tolerated, and effective before the FDA will approve it for human use. Visit our clinical trials page for more information about how trials work and to find a trial in your area.
Observational trials are also important. By observing and testing people with the gene over time (the longitudinal approach) and groups of people at different stages of the disease (the cross sectional approach), researchers can discover and validate surrogate measures of disease progression. Surrogate measures can reduce the time needed to conduct a Phase III clinical trial and get treatments to HD patients. HD family members - gene positive, at risk, gene negative, and caregivers - can volunteer to participate in an observational trial like COHORT.
HDSA supports both kinds of trials through its grants to the Huntington Project (HP) and its assistance in the recruiting process. The Huntington Project works through the Huntington Study Group (HSG), an affiliation of approximately 62 clinical centers that plan and conduct trials aimed at treating HD.
HDSA has continued to increase its financial commitment to clinical trials and provide $550,000 annually to the Huntington Project. This funding enables HP to bring projects to the stage where they can apply for funding from the National Institutes of Health (NIH) to carry out these important trials.
A dynamic process
Although the research pipeline appears to proceed smoothly from basic research to clinical trials, in practice it is much more dynamic. Translational researchers may need to go back to basic researchers to have questions answered. A drug may show clear signs of efficacy in preclinical testing but have serious side effects or poor bioavailability requiring overly large doses to work. Depending on its potential, there may be further efforts at developing the drug. A drug that is successfully treating similar symptoms in another neurological disorder such as Alzheimer's may move directly into clinical trials for HD patients.
It is important for the HD community to know that Huntington's research is going on in parallel. Basic research, translational research, and clinical trials are all in progress. There are more than two dozen drugs and supplements actively moving through the pipeline with dozens of others nominated for consideration. When one critical question is answered, efforts turn to other questions. When one drug fails, resources are quickly redirected to researching more promising ones.
To find out about the drugs and supplements currently moving through the research pipeline, visit our section Therapies in the Pipeline.