Arches. Photo by Daniel Chia
HOPES: Huntington's Outreach Project for Education, at Stanford
Jun
30
2011

About Disease Mechanisms

Welcome to the “Drugs and Supplements” section of the HOPES website! Articles within this section will frequently use the terms “treatment” and “cure.” Please note, though, that the word “treatment” must not be confused with the word “cure,” for there is currently no medical cure for Huntington’s Disease (HD). However, while no existing drugs can actually stop or reverse the neurological degeneration that HD causes, most if not all HD patients can benefit from the management of certain symptoms associated with HD, such as chorea, psychosis, and depression. There are well-tested drugs that have been developed to treat these symptoms, and such therapies may help ease the frustration, embarrassment, and emotional pain that often accompanies HD. In general, these drugs can greatly enhance the quality of life for HD patients and their families. Information on such symptoms and their treatments can be found by clicking here.

The “Drugs and Supplements” section of the website focuses on identifying and explaining the current research on various potential treatments aimed at stopping the progression of HD. We must make it clear that HOPES does not advocate the use of any of the therapies described without the consent or approval of a doctor, and cannot provide medical advice of any kind. However, we do hope that patients with HD, their friends, and their families will ask doctors and HD specialists about both the possibilities presented here and any new developments in this active area of investigation. In the past decade, intensive research efforts have given scientists a much better understanding of how HD damages the brain. With this improved insight, researchers throughout the world are actively investigating and testing the efficacy of drugs that target one or more of these mechanisms of pathology. Their hope is that interfering with HD’s damaging pathways can help delay, stop, or reverse the course of the disease.

This section is organized by disease mechanism. A potential contributor to HD’s damaging effects is introduced, followed by the drugs and supplements currently being studied to counter a given mechanism. For your convenience, a simple alphabetical list of these drugs can also be found by clicking here. Each drug profile also has a short drug summary that avoids potentially difficult and confusing details.

The therapies introduced in this section are at various stages of investigation. Some have been at the heart of well-controlled experiments on mouse or fruit fly models of Huntington’s disease, and others have shown some efficacy in treating disorders with disease mechanisms similar to HD’s such as Alzheimer’s Disease or Spinobulbar Muscular Atrophy (SBMA). There are also many that are being studied simply for their theoretical potential in alleviating HD’s degenerative damage. Because many of the treatments profiled here have not yet passed or even begun clinical trials, and many affect broad biological systems, HOPES must again stress that unsupervised testing could be potentially hazardous. None of these therapies should be tried without exclusive consultation with an HD specialist.

Most scientists believe that a combination of treatments, rather than a single treatment, will be needed in order to cure HD. The different therapies could work together to combat different aspects of HD’s damage mechanisms, and hopefully halt or reverse its progression. Similar therapeutic approaches are currently being used with varying levels of success to control cancer and AIDS.

While this section focuses on the potential drug and supplement candidates for treating HD, various potential treatment options beyond drugs also exist. Recent advances in gene therapy, stem cell research, and neurosurgery are some of the potential long-term solutions being studied. Some of these therapies are becoming available for clinical trials, while others still require further development. (For more information on Huntington’s Disease research in America and in your region, click here.)