That really loooong winding thread really has me thinking about these things. I have the feeling that the research community has a little disconnect with the HD community. Or with how HD families work. Probably more the second thing. I am going to toss out some thoughts, observations, and opinions. Feel free to slice and dice them at will. If I am correct on the original premise, that there is a disconnect, then identifying some things that research community can focus on might be addressed.

First.. of all the reasons people test I have ever heard on here, very seldom is the want to be a trial participant one of them. If it is, it is secondary and far from a primary reason. I am not too sure there has been a compelling study or trial of the presymtomatic that warrants this to be part of the thinking. There are no measurable biomarkers so trials don't use tested but no symptoms people. Some studies do. But people are fairly warned as to the impact on their personal lives of testing and it's repercussions. Those are huge, affecting our daily lives and relationships, and possibly our futures in terms of choices we make based on the knowledge. I think that there is going to have to be some really compelling offerings for those things to move down the decision ladder and research participation to move up it. I don't know why this is difficult to understand. Maybe it is understood but not empathised with. That's one thought.

So the other trials that are offered are for the symptomatic folks. Symptoms and their progress is measurable, as is improvement. Once a person is symptomatic however, they are being treated for those symptoms in many cases. Much of the decision making is made at least in tandem with a caregiver, as well as doctors. Very few symptomatic people who see doctors are making decisions totally by themselves. Sometimes they are there to be treated just to remain in the home at all. We know of many people who have to tell their loved one to treat or we have to part ways. Any trial that interferes with the delicate balance of livability, and normalcy won't fly. A caregiver won't permit it. A pHD won't go for it either in many cases. You can't play with meds that are working. These aren't like cancer trials where the patient can make the decision .. and has options from a working treatment to a possibly better treatment..and can switch right back to the original. The trail for a cancer patient isn't going to upset the family who cares for that patient to the point of displacing the patient for life outside of the home. Changing a cancer treatment won't be changing their personality, there moods, their quality of life. Let alone the the supporting family members quality of life. Life before the working medications are often traumatic and dramatic. I don't think the research community gets this. As much asd I would love treatment options for my children.. getting them and me through today is just as important. Life can be THAT bad. I would never bypass risperdal for creatine trials. Never. Never. Never. They have to get this point. Reading the words depression, irritability, dementia, or psychosis, is far from living with it for years. If those symptoms are relieved, we won't upset the treatments that are working for them.

My other thought is this. It's not talked about too often but what constitutes a good treatment? Is a slightly moderate gain in balance that important? Is extending life by several years with a modest improvement a good deal or are you just prolonging the illness and corresponding suffering of life with HD? That's not a thrilling prospect in some respects. If you nuero-protect a little bit did you just give me two more years of late stage HD? That kind of thing to a researcher is a big deal and advancement.. and scientifically I would agree. It's morally, maybe, more ambiguous and hard to be excited about from this end. It may not be a conscious thought, but just a feeling that the difference between something and nothing are not always that important personally.

And lastly, while we really do want researcher to do the work they do, galloping in like the cavalry is a little unfamiliar. We have really been isolated people for a long time. Even from each other. We are barely warmed up to the concept of HD community among ourselves. For ten years I heard that some guy in the next county had HD too.. that's not community. If that's how close we feel to the next HD person.. imagine how far removed we feel from researchers, cures or treatments, and how are we supposed to care about some guy in the next county or he for us? HD is slooow. We move slooow with it. And by ourselves.. no cavalry. Actually not one foot soldier. I just have to submit that the research community just doesn't know us. I think that to get to the numbers that are desired it will take a more intimate knowledge of who they are trying to serve with this particular disease. In many ways I don't want to be singled out as special compared to other illnesses and we don't have to be sometimes. Other times we do.. especially the social and family aspects of it.

Please feel free to say anything good or bad.. nothing personal about this. This is going to be an upcoming challenge and this is the ground floor. I have mainly rattled this around in my own head and not solid on it by any means.

Steve likes this too

Wait ... this isn't Facebook ... sorry

Eric- I can't begin to tell you how let down I am by the HD research community. It all began two years ago when Allison was asked by her doctor at Albany Medical Center if she would like to participate in clinical trials. This offered her a glimmer of hope and she immediately said yes. She was ecstatic! When I called to enroll her in a trial which was being held in Albany I was told she was the perfect candidate until we got to her age. At the time she was 25 and I was told that the minimum was 28. The person told me she had no idea why there was a minimum age and that she would a.) ask the researcher why there was a minimum and b.) ask if an exception could be made for Allison. Of course, I never heard from her again. Subsequent conversations with HD research people have still not answered my questions. Allison has HD. Her symptoms are just like other phd's. Her symptoms mimic adult onset and not JHD. Her progression is the same as other phd's. We applied for a couple of other trials. Each time the minimum age just eluded Allison although it was different for each trial. Again, nobody could tell me the justification for the age limitation. When I explained to them how important the trials were to Allison, they just did not seem to get it. Finally hope arrived when Allison was deemed old enough for a trial, only to be rejected by the restriction that the participant could not live in a nursing home. This seemed ridiculous as she would be monitored more closely than someone living at home. Again, nobody could tell me the justification for this new limitation. Again Allison was devastated. A few months back, I expressed my sentiments on a thread that solicited concerns about clinical trials. I heard back from somebody who said that Allison was eligible for COHORT. Guess what? We knew this and she was already participating. I do not consider this a trial, it is simply a fact gathering symposium with observations which should be noted during regular examinations. To call it a trial is a misnomer as no treatment is being tried. We have been notified that Allison's neurologist in Albany is leaving after many years devoted to the study of HD. He has been to Venezeula often. We have taken this opportunity to reconnect with her original neurologist at Mass General (whom she left when we moved to Georgia). He was on the team that isolated the gene back in the 90's and is very HD savvy. Perhaps there is reason for hope again. I've now rambled long enough, but you did ask for it!



Edited 1 time(s). Last edit at 06/02/2009 05:07PM by howard.

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Edited 5 time(s). Last edit at 06/02/2009 09:12PM by Barb.

Good food for thought, Eric. You know, I think that you hit the nail on the head about people having other priorities. For many, that priority is LIFE. Some (like me) are single and have no choice but to attend to all of life's demands all by themselves. If there's no caregiver to shoulder some of the responsibilities - often the PHD will lack the time and/or energy for volunteer efforts beyond what is absolutely necessary.


Eric also said: "My other thought is this. It's not talked about too often but what constitutes a good treatment? Is a slightly moderate gain in balance that important?"

Perhaps. Balance IS one of the few symptoms I have. But I have learned to avoid losing it by watching my step. Since I seldom (in real life!) have to do the old heal-toe walk (which I always fail) - I manage to compensate for the balance thing fairly effectively.


Eric futher said, "Is extending life by several years with a modest improvement a good deal or are you just prolonging the illness and corresponding suffering of life with HD? "

I have no desire whatsoever to extend my life, without also improving my QUALITY of life.


Oh - and (based on my battle with traffic to get to doctor's appointments and HD Support Group) - I'd strongly suggest that test sites be in places where there's unconfusing traffic flow and adequate parking for those who drive. And good, close-by public transport, for those who don't.

jl

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Edited 4 time(s). Last edit at 06/02/2009 09:12PM by Barb.

Barb, I think you've misunderstood what Eric was saying. Given all the drugs in the pipeline and the need to prioritize the most promising drugs for trials, I think you'll agree with him that the drugs which extend both life AND quality of life are the best ones to select. That means that both those things need to be measured, even in the mice and we need a more sensitive way to measure this in the UHDRS which is just not sensitive enough to meaningful improvement in people. We don't want something that doesn't postpone onset or slow progression but just prolongs end stage HD.

This is why Dr. Hayden wants to see a clinical trial of memantine (there is one now but I think it may just be Phase II). Here's a drug which is neuroprotective in mice and which is improving quality of life in patients as well.



Edited 1 time(s). Last edit at 06/02/2009 08:21PM by Marsha.

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Edited 1 time(s). Last edit at 06/02/2009 09:11PM by Barb.

Then he's just not being clear because I have had this discussion with him and I feel certain that he meant to contrast extending life with improvement to extending it without.

You ARE doing good now, Barb. I can't imagine that you would be worse in the long run for being on memantine now. In fact I'm hoping that there will be more and more treatments available so that you have a long, good quality life. I want this for everyone - treatments to buy time until there's a real cure.

Well that would make more sense, thank you Marhsa, i hope so

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Edited 2 time(s). Last edit at 06/03/2009 05:30AM by JimmyPollard.

I had to jump ship today.. my dad fell very ill and I had to rush to the hospital. He seems to have stabilized. You didn't have to delete barb.. I said slice and dice..

I deleted cus i just wasnt making sense, it was 95 here today, and that was making me emotional, and it's gonna be 100 tomorrow, so i just decided to delete cus i wasnt in a good posting mood...hope your dad rests easy tonight, and you too...maybe i will slice and dice tomorrow

Jimmy why did you remove your post?

Barb...hope your feeling ok...i wish you didnt remove your posts too.

A little thought...i feel more and more that we need to be supporting our younger people, who may be the ones the researchers really want to target in 5 years or so, when there are more potential trials or research happening...our HD community wont be HD ready if we dont look after them from early on...

But maybe im way off track cause its late and im tired...lol



Edited 2 time(s). Last edit at 06/03/2009 06:45AM by Michelle.

I have not read the loooong winding thread that got Eric thinking so if I end up off base I apologize...

I was not tested so I could start being apart of the cure through research but have been very glad I got involved shortly after my testing.

The best thing that has come out of my being involved in research is almost since I first heard of HD 6 1/2 years ago, I have never had that feeling of isolation. I didn't have to deal w/believing we were the only family dealing w/HD. Through the researchers and our local HD counslor I was put in touch w/people going through the same or similar issues right away.

Secondly, I got involved in research about 5 1/2 years ago for my kids. I wanted to be involved in the process that could some day help them if need be. Then a couple years ago when I was at my yearly predict appt. I was told they were no longer just working on a treatments for my kids, they were getting closer to a treatment that could help ME!!!

I believe the researchers are trying to connect w/the HD community in the best way they can, to help those they can. I will be involved in any way I can to fight this disease for future generations.

Eric, I have never been asked by my fathers doctors to switch his Risperdal for creatine trials....but I have to agree if they did the answer would be NEVER ...I agree if you have something that works stick w/it....but if something isn't working it is great to see that new treatments are being sought out.

DCB
*Living for today*

We all have fear of the unknown. And that is reasonable. When I thought that Tony might want to be in the ACR16 Trial, I debated it in my own head for several weeks before I presented it to him. Would the possible benefit that he would have for a short time be worth the risk of some catastrophic reaction? I thought about our friend from OZ whose wife was given a high dose of Tetrabenazine and almost immediately developed suicidal tendencies and never recovered. The unknown is scary. Ultimately, the choice was Tony?s. He is logical and could make the choice for himself and he is more brave than I am. He immediately said that if he could help ?someone coming down the road behind me? that he wanted to participate in the research.

And we also considered the time and effort it would take. We thought driving would be better than flying but the drive is ten hours round trip. I worried that the trips might be too tiring for him. Jason goes with us (I lost Tony once in a German airport and I don?t want to go through that again. LOL) and we try to make each trip to Dallas a mini-vacation. We shop, we pick interesting restaurants and we visit notable sites. We just act like tourists. That would not be possible if Tony had severe behavioral symptoms. I think back to the days when Tony was raging and hostile. There is no way that he could have participated in a trial then.

Tony was not on any medication so that was not an issue for us. We were asked specifically about Tetrabenazine because it is a dopamine blocker whereas ACR16 is a stabilizer and the results would be faulty if both were taken during the trial.

Eric said, ?I have the feeling that the research community has a little disconnect with the HD community.? I have not felt that with the researchers that we see in Dallas. It has been very much a partnership, but, in general, I think researchers could be more open and available to the HD community. I have felt an aloofness from the researchers and a lack of trust from the HD community. A few times I have sensed what I considered to be animosity from some of the HD people. We have the same goal but we should learn to talk to each other

I know I should keep my mouth shut.I have never been able to get a handle on "Positive Thoughts" about HD. I can not speak as a Phd as I tested NEG, but I can speak as one who has Witnessed and continues to witness the Pain and Distruction of this Damdable Disease.Hope for a Cure is always lurking in the back of my mind right next to this Dragon HD,who never really Sleeps but is given respite in the form of Helpful Panacea"s. I want this Bastard Dead once and for all.

IMHO
This Rant is over
Ron C.

If no completed trials then no drugs. That is the bottom line.With encouragement from Barb and Maggie my wife is in the ACR 16 trial. Mark

Annihilated