Posted January 5, 2009 by LaVonne Goodman M.D.

It's that time of year again: New Years Resolution time.  How about making a personal vow to get off the couch and into better physical condition?  Physical exercise is important for everyone's general health -- but in Huntington's which is known to cause muscle abnormalities and weakness -- it is especially important for maintaining muscle strength and function.

Over the last few years there have been small studies in Huntington's people showing that physical activity programs give functional benefit and can raise motor scores on measures of motor function. And recently a new study gives evidence that a more active lifestyle (as has been shown in mouse studies too) may slow progression of disease before symptom onset.  For those with premanifest Huntington's, higher levels of physical activity correlated with delay of onset for as much as 4 years.

Yes indeed: the New Year is a perfect time -- for us and our children -- to get off the couch.

posted December 3, 2008 by LaVonne Goodman M.D. and Jean Miller

The Huntington Study Group (HSG) held its annual meeting in St. Petersburg, Florida November 12-14 2008 for clinical investigators and invited guests. These meetings culminated in the 2nd annual  HUNTINGTON DISEASE CLINICAL RESEARCH SYMPOSIUM.  The HSG meetings and the Symposium are unique research events because the single focus is clinical research, or that which is limited to human study.

The HSG Symposium is not only unique; it is -- by design -- a remarkably inclusive event. HSG leader Ira Shoulson purposely planned the symposium to be a place where researchers and patient families can sit side-by-side to learn from presentations and each other. As in the previous year, Huntington family representatives were included all the way from the beginning planning stages, to participation of more than 100 families in this year's final event.

In the July, 2008 Journal of Experimental Medicine Dr M. Bjorquist, S. Tabrizi and collaborators reported on their findings of immune activation and inflammation in Huntington's.  They showed that several inflammatory markers called cytokines are found very early in the disease process and strongly correlate with disease progression. These findings are valuable to Huntington families because cytokines may become important biomarkers; and even more importantly this work adds to the growing evidence that inflammation is a highly important disease mechanism, which in turn makes it a key pathway for drug targeting.

Posted October 1, 2008 by LaVonne Veatch Goodman M.D.

The full article detailing results of Repligen Corporation's HDAC 4 inhibitor drug candidate was published in the jounal Proceedings of the National Academy of Sciences (PNAS).  Scientists from Repligen and the Scripps Institute in California detail exciting results from study of their drug candidate in the R6-2 mouse model.  So far, it is looking good . . 

Posted November 30, 2008, by LaVonne Veatch Goodman M.D.

In the previous article (Neuroinflamation Part 1) we reviewed evidence reported by Bjorquist and colleagues for neuroinflammation in Huntington's, where the authors found elevated cytokine levels which increased with disease stage. We also reported on other studies and observations showing that inflammation --and correlating cytokine levels -- form a major component of progression in neurodegenerative diseases.

Building on this work, scientists in academia, CHDI, and other pharmaceutical companies are hard at work on drugs that target inflammation, and several candidate molecules have progressed to testing in mouse models.  However, most of these candidate drugs -- whose safety is unknown -- target downstream mechanisms in the inflammatory process, or those that occur after cytokine release.  Trehalose -- whose safety is well known -- works in a very early part of the process by inhibiting cytokine release.  Might it be time to reconsider this agent for therapeutic development?