In a new laboratory study from Italy, investigators have reported a very exciting result for Huntington's (HD). Pridopidine, the drug presently in clinical trial for HD was shown -- not just to treat motor symptoms -- but also to provide neuroprotective benefit in a genetic mouse model of Huntington's.

Though the important pridopidine human study, the PRIDE-HD trial has another year to be completed, and more time needed to complete analysis, this mouse study suggests we might get more than symptom benefit from this new drug. Exciting indeed that it might give neuroprotection too.

Care is described as informal when provided by unpaid family members or friends, and formal when caretakers are paid. Though a small subset of individuals with HD are cared for in the home through end of life, formal paid home healthcare services are frequently utilized. However the vast majority of individuals with HD, as the intensity of care needs increase will transition to long-term care facilities. In this situation, many family caretakers continue to advocate for and provide care.

What is known about informal care in HD?

In a recent New York Times article, Dr. Siddhartha Mukherjee, a cancer expert from Columbia University wisely recommended that potential clinical trial participants ask their investigators "Why is the trial being done?" And "What were the data that led to the clinical trial in the first place?"

So, what about LEGATO and laquinimod? What are the data that led to this drug being tested in HD?

Recruitment is not going well for PRIDE-HD, an important clinical trial now enrolling for Huntington's disease (HD). Why is this? Some think "why bother", particularly when testing a drug they may perceive as a failure in earlier trials. For some others, the trials may seem to too hard, too long, or not permit other drugs they are taking. Still others are waiting for the gene therapy magic bullet they believe is just around the corner.

Maybe we should look a little deeper?

A recent article reports that aggression is common in individuals with Huntington's disease. Authors report rates of aggressive behaviors between 22% (for clinic patients) and 66% (for hospitalized patients) among individuals with HD. What do they mean by aggression, and how should we interpret these numbers? It is important to remember that aggression (as defined in this article) and violence are NOT the same thing.

However, the most important thing to learn is not how often it occurs, but how we can understand, cope with and treat aggressive behaviors in HD.

Auspex Pharmaceuticals announced quite positive results for their drug SD-109. This drug treats chorea with many fewer side effects associated with tetrabenazine. And most important it improved quality of life.

Particularly during this past year, living with Huntington's disease (HD) has been full of its ups and downs. First there was the hope in ongoing clinical trials of both coenzyme Q-10 and creatine. Then there was sadness and fear that came with their failures. And now hope is springing again for new drugs coming to clinical trials now that will take a few years to complete. No doubt this Yin-Yang cycle will continue for HD, just as it does for other diseases.

However, there may be reason to believe in the chance of greater success this next time around.

"Bummed" was what this author felt after the recent announcement of the failed trial of creatine for Huntington's disease (HD). We can put some positive spins on recent trial failures; we have learned how to run large clinical trials, we can learn from negative outcomes, and best of all hundreds of HD individuals are freed up to participate in other (potentially better) trials. But the reality is that this is one more disappointing failure in an increasingly long string of negative trials. The community is bummed. Saying it straight is better, helps us to get over it, get up and go on to what comes next.

A story comes to mind . .

Human study shows that inflammation in the esophagus (swallowing tube) and stomach is common and occurs more frequently in Huntington's disease (HD) as it progresses. Study also shows that individuals often do not complain. Lower GI function has not been investigated in individuals with HD, but if mouse models of HD are predictive, we can expect problems there too. But the biggest contributor to gut distress in HD is likely due to medication side effects, less intake of water and more sedentary lifestyle as the disease progresses.

Pathological laughter or crying (shortened to PLC) refers to laughter or crying that is deemed socially inappropriate for the situation. Often the individual will laugh or cry spontaneously for no apparent reason. The abnormal behavior is one of impaired emotional expression. Can this be a symptom of Huntington's disease; and if so how can it be treated?