Arches. Photo by Daniel Chia
HOPES: Huntington's Outreach Project for Education, at Stanford
Jul
16
2011

Pridopidine (Huntexil, ACR-16)

Drug Summary: Pridopidine, also known as Huntexil or ACR-16, is a dopamine stabilizer intended to improve voluntary movements and reduce chorea. Initial clinical trials – the MermaiHD and HART studies – show promising results, but drug regulation agencies have requested another trial before pridopidine can be sold to the general public. control relevant M. Hedlin, [...]

Jul
06
2010

XIAP Gene Therapy

The X-Linked Inhibitor of Apoptosis Protein (XIAP) gene is a gene present in normal body cells that inhibits the activity of caspases 9, 3, and 7. A caspase is an enzyme that degrades proteins and is involved in certain types of cell death, also known as apoptosis.

Jun
30
2010

Caspase-6 inhibition

There is ample evidence that Huntington’s disease is associated with a specific genetic mutation that produces an expanded polyglutamine chain in the huntingtin protein. This mutation causes huntingtin to become a misfolded protein with an altered shape. One of the hallmarks of HD is the build-up of short, broken fragments of the altered huntingtin protein [...]

Jun
26
2010

Tetrabenazine

Tetrabenazine Chorea is one of the most common and debilitating motor symptoms experienced by people with Huntington’s Disease. Tetrabenazine (TBZ) has been the drug of choice for treating chorea in 10 countries for more than a decade. However, TBZ has not been widely used in the U.S. because, until recently, the drug had not been [...]

Jun
26
2010

Other Neuroprotective Compounds

Scientists are currently investigating various compounds that are known to have neuroprotective effects. These compounds exert their effects through various mechanisms, often through a combination of the mechanisms discussed above. While most of these compounds are not currently being investigated as treatments for HD specifically, they are believed to have beneficial effects on people with various neurological diseases such as stroke, Alzheimer’s Disease, Parkinson’s Disease, etc. Many neurological diseases seem to have similar disease mechanisms, thus giving rise to the possibility that these compounds might eventually be potential HD treatments as well.

Jun
26
2010

Ethyl-EPA (Miraxion, LAX-101)

medium Ethyl-EPA is made from eicosapentaenoic acid (EPA), an omega-3 fatty acid. (For information on omega-3 fatty acids, click here.) Ethyl-EPA, also known as Miraxion or LAX-101, is a novel compound that may function as a neuroprotectant by preventing the degradation of brain tissue through a variety of proposed mechanisms. The compound might inhibit harmful [...]

Jun
26
2010

The Heat-Shock Response

Cells of all kinds are often exposed to sudden changes in their environment that cause stress. They often respond to stress by making different sets of proteins that protect the cell and return it to a healthy balanced state called homeostasis. These stress responses work together to make sure that cells and tissues are protected [...]