Lithium is an old drug that was first used for manic depressive or bipolar disorder more than 60 years ago. Over the last decade this drug has also been considered as treatment for several neurodegenerative diseases including Huntington's disease (HD); but the problem is its high risk of toxicity, particularly for long term use. But now with the recent publication about NP03, a novel (or new) low-dose lithium reformulation showing neuroprotective benefit in the YAC128 mouse model of HD, it is back in the running as a promising potential HD therapeutic.

NP03 is a new drug from Medesis Pharma that delivers lithium in a water and oil microemulsion that allows for much lower (15X lower) therapeutic dosing. Barring future problems, this drug may be on its way to study in people.

The prestigious American Academy of Neurology (AAN) has published a new set of "evidence-based" guidelines for the treatment of chorea in Huntington's disease (HD). These guidelines are based on "best evidence", which uses only sets of data from clinical trials or studies testing drugs for chorea. But what if there isn't enough evidence to make sound medical recommendations -- which had been the conclusion drawn by the Cochrane Review and the survey of expert HD physician leaders from the European Huntington's Disease Network (EHDN) and the Huntington Study Group (HSG)?

Why be concerned about the AAN guidelines? At best they are inappropriate; at worst they have potential to harm.

At the recent Enroll-HD Global Investigator Meeting a week ago, we learned that this soon-to-launch study sponsored by CHDI is intended to be more than observing progression in Huntington's disease (HD). It will enable investigators to do "add-on" studies that focus on specific populations (like Juvenile onset), or on "cure" efforts like the search for biomarkers, or preparatory study of new drugs before launching large scale trials, and to identify participants for clinical trials, etc.

But we also learned about an exciting new focus: Enroll-HD data may be used to improve HD care at HD treatment centers. And in a presentation by Kathryn Sabadosa from the Cystic Fibrosis Foundation we learned how this might be done.

Over the past decade a number of over-the-counter (OTC) supplements and prescription drugs have been reported to slow down progression of impairment in animal models of Huntington's disease (HD). Whether Huntington's people "should" take supplements for potential human benefit is unknown. This author's opinion has evolved over the years and I am now more skeptical about using mouse model results than when we initiated the HDDW trials. What follows will be a supplement update.

But -- to keep this discussion in perspective -- it is much more likely that a healthy life style will give greater benefit than any present supplement.

Why is a healthy life style important in HD? A big part of the answer include factors that promote the process of neuroplasticity. The "plasticity" part of neuroplasticity refers to capacity to change, similar to how plastic is malleable and can be made into many shapes. Similarly, neuroplasticity describes the ability of nerve cells to change -- based on various forces -- by making new, or stronger connections to other nerve cells in the brain. Healthy life style factors promote these forces. And vitally important in brain diseases, healthy life style factors can help preserve brain function while damage occurs.

Lack of good sleep is a major problem in Huntington's disease (HD). Further, the degree of sleep disturbance correlates with levels of fatigue, depression, cognitive impairment, harmful metabolic or energy factors, and degree of damage seen on brain scans. Bad sleep is bad news. But the good news, at least in HD-mouse models, is that rigidly scheduled "environmental entrainment" can improve several of these factors. To put this in perspective: the level of benefit is far greater than that reported for any supplement or drug tested in mice.

And best news? HD individuals can do this too, and if the experts are correct we'll not only feel and think better, we may slow progression of the disease.

Huntington's disease (HD) is commonly characterized as a trilogy of overlapping disorders that include cognitive, motor, and psychiatric components. But this author suggests that we should add HD stigma to the list. The stigma part of the HD has great negative impact on many, but it is not well studied or recognized for the toxic part it plays through every stage of this disease.

Perhaps, if we thought of stigma as an official part of HD, it might get the attention it deserves.

Passionate responses came fast and furious from Huntington's disease (HD) facebook communities following publication of articles by Gene Veritas about real-life dilemmas of two HD families who have made difficult reproductive choices at the extreme ends of the choice spectrum. These two courageous and thoughtful reports hit an emotionally charged nerve: Part 1, a couple who chose to abort an expanded-gene fetus that was subsequently donated to research, and Part 2, an 18 year old woman with juvenile-onset disease who with the support of her family has chosen to continue her pregnancy, which if positive for the expanded gene, her child will likely have juvenile onset disease as well.

No question that HD families face hard reproductive choices. What is known about how families make these decisions?

Just published on his blog, Gene has addressed that there is some conflict within our Huntington's organizations. This is to be expected in any human venture where there are passionate differences of opinion. Though there can be negative consequences, As Gene points out in his linked article: when conflict spurs discussion, it can also be a good thing.

Lundbeck and the Hereditary Disease Foundation are partnering to support the Venezuelan families whose participation in clinical research led to the discovery of the gene responsible for Huntington's disease. The goal is to provide funds that will keep their treatment center open.

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