A little high level background first. Everybody has a certain amount of the a protein in the body called the huntingtin protein. It helps do a wide variety of things in the body. When the protein is in normal. health, there are no problems. However, if the body begins to produce a mutated form of this protein, the result is Huntington's Disease.
In different ways, both of these studies aimed to change the makeup of the gene that causes Huntington's Disease by decreasing the amount of the harmful protein created by the body. Each study developed antisense oligonucleotides or ASO's. These are a type of gene silencing treatment the has DNA molecules designed specifically to alter the functionality of that gene.
The first study - Wave's Precession HD-1 and 2 - was designed to target the message the body creates to produce the mutated and harmful version of the protein. The idea was to eliminate the harmful version of this protein so that it couldn't cause harm to the body. In other words, eliminate the problem child, eliminate the problem. Unfortunately, it appears there was no compelling evidence to show a change in the amount of the harmful protein when compared to those treated with the placebo. As a result, Wave announced on March 30th, they have decided to halt the study and begin work on the next treatment.
The second study done by Roche and Ionis. This study developed an ASO aimed at silencing the entire huntingtin protein. In fact, during Phase I and II of this study, it appeared to have a lowering effect on the protein. Phases I and II are an assessment to see if there are any safety or harmful side effects and not designed to monitor the overall results the drug has on the body. It looked so promising. Then, on March 22nd, they announced they were halting the study.
Every study has a committee of independent data experts who periodically review the data of the trial and determine if the study should continue. They usually ask two question about the data as they review it.
Are there any new safety concerns that are emerging that did not show up during Phase I or II?
Does this ongoing trial show unlikely beneficial results?
In other words, if participants are showing some new strange or unfavorable side effect, the committee would order the trail be halted. If the symptoms do not appear to improve or it does not seem to show a likely benefit or improvement in a person's condition, they committee would recommend halting the trail. It appears based on the announcement from Roche, it appears that there are no safety concerns resulting in the halting of the study. We don't really know as not much information has been given. Hopefully, over the next few months, more data will be released and we might learn more.
Looking at the bright side, both studies have provided valuable information that will be helpful as they move forward to develop and test the next drug that might be the cure. My favorite website for all things HD news related (HDBuzz) said it best. "We shake ourselves off and do it again"
While this is a setback, I have not lost hope. I still believe the cure is out there and one day we will find it.
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