What is Cysteine? And what does it have to do with Huntington's Disease?

Posted March 27, 2014 by LaVonne Goodman, M.D.

In a paper published this week in the prestigious journal Nature, Dr. Solomon Snyder and colleagues reported promising results in cell and mouse models of Huntington's disease (HD).  They showed that these models were deficient in both (1) cysteine, one of the amino acid building blocks in proteins, and (2) an enzyme molecule involved in making cysteine.  And more exciting, they showed that adding cysteine was protective in these models.

Might cysteine help people with Huntington's disease?  Preliminary results reported in Raptor Pharmaceutical's recent press release suggests it may.

Raptor's Clinical Trial for Cysteamine in Huntington's: Cause for Optimism?

Posted March 1, 2014 by LaVonne Goodman, M.D

Raptor Pharmaceutical Corp. announced some positive (though preliminary) results from the 18-month review of their CYST-HD  Phase 2/3 clinical trial testing a cysteamine drug for Huntington's disease (HD).  They reported slower progression of motor impairment in those participants who had received drug compared to those who had received placebo.  If these positive results hold up on further analysis and completion of the remaining 18 months of trial, this author believes we have very good reasons to be optimistic about this drug for HD.

What is this Raptor drug?  Why try this drug in an HD clinical trial?  And -- why might we be optimistic now? 

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Treatment Guidelines for Huntington's: Who needs them?

Posted by LaVonne Goodman M.D.

During my years as an internal medicine physician, I have used standard of care guidelines for my patients with conditions like diabetes and heart disease. Guidelines are developed by experts in each disease who translate clinical trial evidence and/or expert experience into recommended care patterns for use in medical offices or at the bedside. When followed, guidelines have been central to improving the quality of care provided by all physicians whether they are specialists or generalists.   


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