The day began with some very dry, factual information of the studies at hand on TBZ. This included speakers for the sponsoring company of Prestwick as well as various doctors looking at and analyzing those studies. The information and numbers presented left some questions, that due to the size of studies, the length of them, and the methodology, left some room for interpretation. There was a long list of adverse effects of TBZ but none reached the level that was of major concern. Combined however, a high percentage of users did experience some adverse reaction. Of course some were not alarming. They just have to be reported. You see these listed on every drug released such as sleepiness, nausea, etc. Depression was of course a concern as we have known about that. Characterizing this portion of the meeting is difficult other than to say it was what one would expect, interesting to a point but technical.
What was immensely more interesting but semi-discouraging was that while the drug was consistently cited as being "robust' in the control of chorea, the test subjects found no corresponding benefit in quality of life. This seemed to be a focal point and worrisome in the panel's thinking about TBZ. As presented, I myself was seeing what the committee was concerned over. If people have chorea controlled but really get no benefit from chorea control then why medicate for it? I was surprised this could actually be the case. Dispite concerns, it seemed there was no overall feeling that the drug would not be approved.
We broke for lunch and I sidled up to a couple of people outside who were more in the know over this process than I was and they confirmed that things were unreadable. They pointed out the problems that had been cited and the general problem with money available to study this sort of drug with this kind of disease -- the lack of NIH money to fund a large comprehensive study, for instance. Discussion of the topic during lunch was prohibited, but I skipped lunch and hung outside and talked to the smokers. And I broke the rules, like we smokers tend to do. I was reassured that the testimonies may help.
After lunch the testimonies began. WOW! The speakers laid themselves bare before that committee and bluntly begged them to understand chorea, the heartbreak of the disease, the true care and concern not for just loved ones, but for you, me and anyone who is dealing with HD. It was beyond moving. More than a dozen speakers hit every example of concern with respect to chorea. From the official hopes of the various organizations, to the heartbreak of carers from facilities, to the family careers, to sufferers themselves, no stone was left unturned. To cite any one as more pertinent than another would be an injustice. The body of speakers flowed as if scripted to follow one another with hopes of relief from chorea. There were no false hopes of TBZ, but just stating the idea that someone who wants to try TBZ should be given the opportunity to do so. I am not overstating just how moving this was and yet the extreme dignity was also poignant. No one needed to go over the top; there was no over the top to go over. Every speaker helped the objective and no one said anything even marginally hurtful to HD or hurt the consideration of approving TBZ.
What I found striking as well was the speakers showing that they grasped the reality that TBZ is not the answer to HD. It isn't perfect but we have nothing perfect with HD and this drug even at it's worst is basically harmless to those who wish to try it. The committee could see that the HD community won't allow unreasonable harm to happen to us and will be extremely responsible with TBZ. This was not said in those words but conveyed in that all the testimonies exhibited that responsibility of sufferers and carers, in every example of all aspects of the disease. So if TBZ were to be approved it will not be used simply because it's there but will be considered an option that may or may not work.
A well deserved fifteen minute break was needed. The lumps in peoples throats needed time to subside and running makeup need to be reapplied as tears had been flowing throughout the testimonies.
Following the personal testimony was a question and answer period between the participants. During this question and answer period it was evident that the personal testimony had its desired effect on the panel in many ways. Several members of the panel in there discussion quoted almost verbatim personal testimony in there responses to questions. Also up until this point we had heard that the study did not prove any benefit to the taker except less chorea. In fact the taker of the drug was more likely to have more falls. The panel could not see the huge benefit of just having less chorea. The focus changed from the hard numbers on falls or help getting dressed to whether a sufferer who had less chorea was given more dignity simply because chorea was absent was emerging. They saw intangible benefits that a test can't measure -- Less bruises, fewer embarrassing moments like dropping food in a restaurant. They instead saw that the clinical assessments were not asking the correct questions perhaps or not in the correct way.
I must mention that the committee did an excellent job of looking at the the whole person who has HD. They DID consider the caregivers and the sufferers family. They looked beyond numbers. I quite frankly was surprised at the committee's grasp of HD and all it's relevant issues. They used reasonable compassion and seemed to me to forego worries about TBZ for the benefit of our community. They did not abandon their concerns but weighed them against the potential benefits of TBZ. They kept in mind the responsibility to the public as well. At the end of the day they were very real people who honestly wanted what was best. I was impressed. They didn't act like they knew what they didn't. Their questions were not accusatory or leading. The questions were simple fact finding. They stated concerns but didn't over emphasize them. They accepted if another member had a reasonable rebuttal to a point. No ego clashing was evident. It was very much a reasonable discussion whether you agreed with a point being made or not.
I think it would be nice to recognize our list of speakers on our behalf. The speakers were Nancy Wexler, Barbara Boyle, LaVonne Goodman, Maria Hardin, Katie Moser, Anne Pea, Gabrielle Hamilton, Barbara and Gary Parker, Wesley Johnson, Cindy Diogo, Ann Russo, Daniel Born, Jonathan Monkemeyer and Cheryl, and Debbie Fine. We could not have been better represented. White t-shirts were distributed to those of us supporting approval of TBZ and all the support was helpful as well. The testimonies were sited were in several cases word for word in the panel discussion. The panel also mentioned several times that they received, I think, 1500 letters and each one was read by every panel member.
Not that I haven't editorialized plenty, but I must say I now see where putting ourselves out in the public eye is of maximum benefit. Those few individuals who spoke made a very tangible difference. They may have literally made a difference on countless lives in the future. I am very proud of them that they did what I didn't. When given an opportunity to make a difference I hope that I and all the rest of us take advantage of them. There will be more drugs, more fund raisers, and more advocacy issues. We are not used to being heard in the HD community let alone winning many significant battles. This is a good example of both. It makes me hopeful that as issues arise that we will now begin mobilize to our own benefit. We have this win to point to, and those who did accept the responsibility to thank, in showing us that we can be heard and can win!