I liked that too. Once we can get a trial start the price should...

I liked that too. Once we can get a trial start the price should move up. The cost of the Huntingtons trial is only $2m which they already have. The Huntingtons should be interesting. They had very good results on the model and now it is just to get the trial started so wee can see if it is just as effective in humans. It is the same drug as they use on the Alzheimers, so they already have a lot of safety data with the phase1 and phase2a Alzheimers trials.
There is no effective treatment for Huntingtons. One drug was approved a while back after a short trial, TBZ, but to date there is not a real disease modifying drug available to patients.
TBZ has some safety issues
[The FDA had two sets of concerns. The first was the risk of depression and suicidal thoughts. The second was a trend toward a worsening of mood, cognition, and functional capacity. The trend was not statistically significant which means that the results might have occurred by chance. Still, it does suggest that the patient, family, and doctor should continue to monitor the patient's response to the drug on an ongoing basis.]

I hope Prana keep the family advocates up to speed on how things are progressing once the trial gets underway.


[There are some lessons for the HD community from the TBZ experience. First, we need to educate ourselves about any drug that is under review for approval. I've talked to some HD family members at recent HD related events who were confused about what this drug is being approved for. It has been approved as a treatment for chorea, a symptom of Huntington's Disease. It was not tested for nor approved as a treatment of the underlying disease itself. That needs to be understood as do the good results in reducing chorea and the potential side effects. It's also a good idea to look at the studies that were done, as we do here on the Lighthouse (see the references below). In educating ourselves, we need to know 1) what any drug is expected to do, 2) what the evidence is for its effectiveness and safety is, and 3) and what the potential side effects are.

Second, we need to make our voices heard during the review process. Huntington's has no approved treatments for the disease itself. TBZ is the first approved treatment for a symptom of the disease, chorea. Although other symptoms such as depression are treated, the drugs prescribed were tested in other patient populations and their use in HD is considered off-label. In the next few years, we are likely to see more new drug applications, some for symptoms, some for the disease. Drugs have side effects, even the safest drugs. Those side effects have to weighed against the potential benefit. We need to tell the FDA how we balance the equation.]

http://www.hdac.org/features/article.php?p_articleNumber=589