Huntington's Disease Research Reports

Hello and welcome to the new HD Lighthouse Research Portal!

The odds are very good that you were directed here from the the original URL. You've been redirected to our "focus on research" page found at

All of the HDlighthouse articles that were posted over the last few years can be found here. If there is an older HDlighthouse page that you cannot find but would like us to try to locate and set up on the new site please let us know and we'll be happy to chase it down. Just send an email to

2CARE Study Ends Early

The Huntington Study Group has ended its Phase III trial of CoQ10 early because of futility.  The study has been going on for a number of years now and early analysis of the data indicates that it is very unlikely to prove to be effective.

It’s time to discontinue CoQ10 as a supplement.   Mouse studies discussed here at the Lighthouse have had inconsistent results which is why we were not hopeful that this supplement would work.

Why Mechanism Matters

The failure of the Dimebon trial prompted me to think about how best to select drugs to move through the pipeline from preclinical research to clinical trials.  It seems to me that knowing how the drug works, how it acts on a therapeutic target, is an important consideration in prioritizing potential treatments.

HORIZON results are in: Dimebon unsuccessful in Phase III trial

Dimebon was not successful in its Phase III trial in Huntington's disease patients. These results are disappointing but not surprising after the unsuccessful trial in Alzheimer's patients.

The take home message here is that in choosing drugs from the pipeline to move into clinical trials, we should give our strongest consideration to drugs where the mechanism is known.  It was never clear how Dimebon might work.

Exercise Not Beneficial in an HD Mouse Model

People at risk for Huntington’s Disease are interested in research which suggests how to live a healthy lifestyle that postpones disease onset as long as possible. One of the most common recommendations and the most solidly researched has been exercising to fitness. There are two reasons for this. The first is that the overall health benefits to the general population are widely known.

Grape Derived Polyphenols

Researchers at the Mount Sinai School of Medicine in New York have investigated grape derived polyphenol extract (GDPE) as a potential treatment for Huntington's disease. They found promising results in a cell model, a drosophila model, and the R6/2 mice.

In previous studies, the authors investigated the bioavailability of GDPE and found that although it is metabolized in the gut, it does cross the blood brain barrier and can be detected in the brain.

DRP1 is Identified as a Promising Therapeutic Target

Impaired energy metabolism has been shown to be a major pathology in  Huntington's disease.  The mitochondria, the cell's energy factories,  have not been found to be  intrinsically defective but are thought to be  mismanaged in some way.  A University of Central Florida research team  lead by Dr.

A New Drug Enhances Neural Growth Factor

In the press release below, CeNeRx BioPharma, Inc. announces that their new drug CXB909 will go into clinical trials for peripheral neuropathy.  This is of interest to the HD community since the drug enhances the effects of neural growth factor (NGF) and may be neuroprotective in Huntington's Disease.  Preclinical studies of the effects on this drug on neurodegeneration have been done but do not appear to have been published so I do not know which disease models were used.  Still, the Lighthouse will follow this company's efforts.

Novel Benzoxazine Compounds

Researchers from Southern Methodist University and the University of Texas at Dallas have collaborated to develop new benzoxazine compounds to prevent neurodegeneration. Similar indolone compounds had been found to inhibit cell death but were toxic at higher concentrations. The benzoxazine compounds were synthesized by altering the ring core structure, preventing toxicity even at high doses. Benzoxazines are the reaction products of an amine, a phenol and formaldehyde.