Since April 2000 the mission of the HDLighthouse Families Web site is to present and explain the latest research findings so that families afflicted by Huntington's disease can become proactive in their care, have hope for the future, and make good decisions in the present. Additionally we provide information that is vital to the the support of HD families. Where possible we will direct you to specialists in the your area of concern, but if we are able to provide better or more current information then you'll find it here.

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.

The Effects of Huntington's

Having HD can and does make my life extremely complicated. Everyday things that can come easily to other people can become a huge struggle for us. Sometimes I feel like I am somewhat of a ticking time bomb because I have done incredibly well to make it as many years as I have with this disease. I know without a doubt that I am on borrowed time right now. The need to make every single day count before I progress too far is ever present. The fact that I am very easily confused add a lot

Everolimus Not Neuroprotective in the R6/2 mice

Here's another major article that adds to our information about autophagy.

Dr. Steven Hersch and colleagues have investigated the effect of the rapamycin derivative, everolimus, on the R6/2 mice.  While there was improvement in rotarod performance and the drug did penetrate the brain, it failed to protect neurons.  Everolimus reduced the HD protein in skeletal muscle tissue but not in the brain where it did not activate autophagy.


A lot of good research is coming from the Buck Institute.  Dr. Lisa Ellerby, Dr. Robert Hughes, and colleagues at the Buck Institute for Age Research have identified a group of proteinases as promising new targets for treatment. Proteinases are enzymes which cleave proteins.

The approach was based on the toxic fragment hypothesis that has received much support in previous research studies.  The idea is that a key event in the development of Huntington's Disease is the cleavage of the HD protein into fragments which then enter the nucleus of the cell and cause damage.

Autophagy and Antioxidants

Because HD is associated with oxidative stress, some Lighthouse readers are taking antioxidants.  A study at the University of Cambridge raises the issue of whether this is a good strategy since some antioxidants seem to interfere with autophagy.  However, other studies have suggested that autophagy is already impaired in HD ( see: cargo recognition is impaired in HD ).

2010 HDSA Covention Research Forum

The attendees were welcomed by Louise Vetter who urged people to answer thequestionnaire from Project Aware which was available in the exhibition hall oncomputers and on paper. Without clinical trials, we will not have treatments andresearchers need to know why potential participants choose to participate or not.

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