Maggie, I followed your and Tony's ACR-16 era. Thank you.
Seven days back I contacted UT SW Med (amit Gode); stated I wished to enroll myself and my two daughters. I gave him our CAGs. He said he would talk to the doctor and get back with me. He has not. He has all my contact info.
How much time lapsed before you were contacted? I will contact him/them Monday. Bob

Hi Bob-

I would like to follow-up with you about this in the interest of making trials generally, and this trial in particular, go faster. Would you contact me on Monday after you speak to the UTSW study site and let me know what happened? I can be reached at robi.blumenstein@chdifoundation.org.

Thanks,

Robi

Robi Blumenstein
CHDI Management/CHDI Foundation
robi.blumenstein@chdifoundation.org
www.chdifoundation.org

Hi, Bob,
Amit is the right person to contact. I don?t know what is going on with them, but they have been nonresponsive to me lately as well.

During the trial Amit answered my phone calls on the day that I phoned him. He even called me about something that I posted here. And we talked about the discussion of ACR16 going on at HDAC. So maybe he is still reading this forum.

Even when I phoned him to say that our campaign to get NeuroSearch to push to get ?compassionate use? approved had worked and that they had made the approval a top priority at the company, he did not call me back. Once, when I needed to talk with him, I phoned another research assistant and was transferred to him. You might try that. If you don?t have a number, email me and I will look for it at the office on Monday.

The trial was very good for us and Tony?s quality of life improved. It has been extremely hard to watch him lose that ground again so fast. We had expected it but I am on the fence as to whether we would do another trial.

Robi, I would be happy to share Tony?s experience with you. You might also want to read our son?s blog about it at [atlasfolding.com] (Fighting Huntington?s Disease with Folding@Home)

How to help? Send an email or phone call to the FDA Office of Orphan Product Development and ask for compassionate use approval for Huntexil.
[www.fda.gov]

Maggie, thank you; good to know we are in the same boat.
Have you reconnected with NeuroSearch?
Robie, I shall update you.
Bob

The first step to getting the drug for the trial participants for compassionate use was to get NeuroSearch to agree to apply to the FDA for its approval. Jason and I made that a family project. We wrote emails and were joined by others from HDAC and Hunt-Dis. Then Jason phoned them. I copied Jason?s entry on his blog to tell you the story. A day or two later I got an answer to my email from the clinical project leader saying that because of the phone calls and emails requesting compassionate use that NeuroSearch had made getting its approval from the FDA a top priority.

The second step is to get the FDA to act quickly. So we are now emailing and phoning the Office of Orphan Product Development. I don?t know if NeuroSearch has finished writing its submission to request that the drug be allowed yet. We will see if we can find out this week.

You know what they say about squeaky wheels. Here is the blog entry.

from post of Aug 28....

Huntexil
It?s been hard for my family to watch my father?s decline since the end of the ACR16 trial (now named HuntexilTM). While my father is stoic, it is apparent even to people outside the family that his chorea has worsened without the drug. It often seems hard or even impossible for him to focus on a conversation now.

So it was very exciting for us to receive word through the HDAC forums that NeuroSearch is making the drug available for ?Compassionate Use? in Europe and the United States for persons that have gone through the trial. This basically means that anyone that has trialed the drug can continue to receive the drug until it is made available via prescription. It is up to the pharmaceutical company to decide whether to charge for it or not, but NeuroSearch is offering it free of charge to those qualified.

To get the details on the status of this availability here in the United States I phoned Denmark very early this morning and was forwarded to the Clinical Project Leader for the Huntexil trial. After calling me back an hour later I was pleased to find that she was very gracious; she spoke to me for several minutes regarding the status of Huntexil for Compassionate Use. I was told that NeuroSearch has never gone through the American process for getting a drug certified for Compassionate Use and that apparently there were multiple ways of getting it done; they were not yet sure of which was was best for this drug. It is unfortunate news that the process has not yet begun, but I was encouraged a bit by her saying that I was the first person to have contacted her from the United States and that it was significant to her and that she would redouble her efforts on getting the drug approved here in the USA.

If you are reading this and have Huntington?s disease or someone in your family does, I encourage you to go through the drug trial for ACR16 if you are able (especially now that you will soon be provided continued use after the trial under Compassionate Use). Slow enrollment in the trial is causing the American process for drug approval to drag out years longer than it needs to.

Here is the list of sites in the United States and Canada where the trial is taking place, and if you would like some help getting started with it just drop me a line, I?ll be glad to help.



Edited 1 time(s). Last edit at 10/11/2009 07:48AM by Maggie.

You know Maggie, i just feel so sorry and mad for you. It seems like the trial just used you for what they wanted, and now that you need them, they've just thrown you to the curb. So i dont really understand this, but it seems that someone says compassionate use is available, but whoever is saying this, is great that they're saying that, but that's as far as it goes? Lip service? Because you haven't been able to get it yet. Someone says it's available, yet you're having to pull teeth to actually try and get it. This seems like abuse of hd families at it's worst



Edited 1 time(s). Last edit at 10/11/2009 01:30PM by Barb.

Barb, I think the company wants to do this, but they have to wait until they have the go ahead from the FDA. I've met several of the people connected with the trial and they are really excited about this drug and hoping it will prove to be effective.

Thanks, Barb.
But it is not the clinic?s fault. The pharmaceutical company had to agree to provide the drug for compassionate use first, which is what we have accomplished because of our noise making with emails and phone calls. But they cannot give it out until they write a ?submission? to the FDA and get it approved for that purpose. It still would not be approved for general use in the HD population until the final approval for any doctor to write prescriptions for it. If we can convince the FDA to approve the drug for compassionate use we will be able to get it for Tony. But it would not be available for anyone who had not been in the trials.

The status is that compassionate use is approved by the drug maker but not the FDA.

I had felt a little like I had been thrown under the bus, but since they are not getting back to Bob, a new potential participant, I think they just have something else going on. I will try to find out.

Bob, keep trying if you want to try the drug. If all else fails perhaps you should contact NeuroSearch and find out if the University of Texas Southwestern Medical Center is still on the list for the trial.



Edited 1 time(s). Last edit at 10/11/2009 03:52PM by Maggie.

Thanks for explaining marsha and maggie. So the drug company wants to do compassionate use, but they haven't submitted a request to the FDA yet? Or they have submitted their request, and the FDA just hasnt approved it yet?

I still think we need to change the rules for human drug experiments.
All drugs trials must include placebo and the drug being tested so each person will know if the drug is effective for them.

If a drug is effective for anyone it will be supplied at no cost to the person until they die. If a drug company is allowed an out of saying " Oh it is too expensive to do that, then there should be no testing done on the drug and it will not ever be sold.

For each person in a study the drug company will pay hotel and meals costs and travel costs for two family members.(I travel about 350 miles to do the cohort study which is not done in Ontario.Hthe hospital gives us $12 for transportation. About the cost of breakfast at McDonalds.)

SOme people will say the rules are written in stone but actually people give the least they can. the see life from the other side, let me tell you I know a new neuro radiolgist. His contract includes 3 trips home with food transportation and hotels to his parents and three more all expenses paid to her parents.

so the sky is the limit if you get organized before Maggies heart breaking situation happens to anyone else.

It is hard enough to have HD without being victimized as a reasearch subject.

I wont do any drugs stuides until my offer includes my points.

Thanks, Dusty, but I don?t feel like a victim. We knew before we agreed to the trial that the drug would not be available for Tony after he finished the trial. We discussed it in the family and decided that three months of improvement was worth it. And it was.

I think about the electronic circuit that Tony designed for our company?s transmitter during the time of the trial and I smile. Our mechanical engineer called it ingenious and we have incorporated it into all of our line of transmitters. So, for a while we had part of our old Tony back.

I lived through the Thalidomide problems which caused deformities in babies and I understand why the system needs to move slowly.

But I am doing everything I know to do to get compassionate use.

Maggie, i'm glad we got your name back. i didn't recognize you when you were signed in as Margaret.

Thanks. I don?t understand what happened. I signed out as Margaret and asked for a new password with the user name as Maggie, but when it came back to me the user name had been changed to Margaret. I tried it over and over and finally the user name came back as Maggie. ???? I was not able to post once before as Maggie and so I registered as Margaret so that is why Margaret is in the system.

It's very strange, Maggie! Steve and I are still battling flu so all of the site work is still on hold.

The community has certainly come a long way. In 2005, I went to the HDSA convention where the HSG proudly announced that there would be a 'new' CoQ10 trial (which then didn't get off the ground for another three years) and that there was nothing else ready to go. I made Clinical Trials Now buttons, about 500 of them, and Lavonne and I gave them out (HDDW had a table). I had lots left over because many people were AFRAID TO WEAR THEM. They would say things like, "What if the researchers SEE ME with a button and get mad and Keep me out of clinical trials?" It was sad.

The same year, I remember a Lighthouse forum member calling a COE about the CoQ10 trial (uh you know, the one that didn't get off the ground for three years) and being told he'd better go off supplements including the BLUEBERRY CONCENTRATE the man was taking or he wouldn't be allowed in.

Things have definitely changed now that we're standing up for ourselves.

I spoke once to the entire World Congress last month. The issue was inclusion and exclusion criteria for clinical trials. I gave my age, positive gene status and lack of symptoms. Then I said, "I would be glad to participate in any clinical trial, but don't take away my blueberries and make me stop running."

That elicited a warm round of applause, so the researchers may be "getting it".

Will

Marsha Wrote:
-------------------------------------------------------
> The community has certainly come a long way.
>
> Things have definitely changed now that we're
> standing up for ourselves.


So true! We should all speak up for what we want. Together we can change things. Before we all emailed or phoned NeuroSearch they had no immediate plans to petition the FDA for approval of Huntexil for compassionate use. Within a couple of weeks after the communications from us started they had put the approval as a top priority with the company. They just needed to know that it was important to us.

I still say, this is abuse of hd families at it's worst. For some reason, in general, hd families, not you maggie, im talking in general, have a humble pie attitude towards their doctors and researchers, and have a, oh i should be so lucky to have a minute of your time attitude. Other diseases don't do humble pie, theyre out their fighting for their rights, they have attitude. When i go to my doctors, i have attitude, and i run the show, and they know it. I say what i expect and what i want, and they say ok. I'm sorry, but i said this before, but it wasnt taken well what i said, but i'm going to say it again and i don't care. People need attitude when going into a trial. I have said before, we need to make demands, demand the drug will be available after the trial, or we dont do the trial. And of course, everyone here says to me, oooooh, but we should be so lucky they want to do a trial for us, bull! They want to do the trials just as much as we want them. Where the hell is the attitude??? Why can other diseases get organized, and make their demands known? Why can't we get some friggin attitude on his???? Oh, because hd is so shameful and such a secret. Well let's start getting it out of the closet more and more, and get some attitude, and give hd a voice!

I agree with you there, Barb! We have been so pathetically grateful in the past to get medical care or participate in a trial that we've allowed ourselves to be patronized and decisions made for us without our input. We should have been chaining ourselves to buildings like AIDS patients did. You know how they used to do AIDS trials before activists brough about change? The endpoint was time to DEATH!!! So the treatment group got an experimental drug and the placebo group didn't and the placebo group had no chance of treatment EVER because the trial ran until the participants died! Activists refused to go along and the endpoints were changed to measurements of biomarkers of the disease. And when something was promising, trials were stopped early and everyone got the drug. So we know that change is possible!

The first thing that hit me when my MIL tested positive for the gene was how patronizing the medical community was to my ex and me. All of a sudden we were thought to be too dysfunctional for him to test and find out if he had the gene without jumping through hoops. Never mind that we had a stable 28 year marriage, had good jobs, were politically active, and taught Sunday School, all of a sudden we were poor pathetic souls who had to prove we could handle test results.

This whole attitude is carried throughout medical treatment. If a child has JHD symptoms, he has to get sicker and sicker (an average of nine years!) before anyone will test. I have actually heard doctors say that there is no point in even diagnosing JHD, that doctors should just deal with the symptoms!

If a family member is showing early signs of HD and making poor decisions and alienating friends, family, and employers he may or may not get a diagnosis. Again, I've heard doctors say, I won't diagnose HD until it's absolutely incontrovertible, the conditions are too stressful. Well guess what, it's a lot more stressful for the patient when he loses his job and can't get his disability or medical insurance, when everyone just thinks he's a jerk and turns their back on him and he winds up alone and homeless while thinking he's still healthy.

And doctors need to be consistent for crying out loud! When my ex behaved badly, I was told "It's not him, it's the disease!" True, I agree. But then when he I wanted guardianship to stop him from blowing money in the stock market and then later so he could go to a lovely assisted living facility that could deal with anger outbursts, I'm told, he's competent to make his own decisions. Pick one! Either it's the disease and we have to be understanding caregivers and intervene, or competent people are making bad choices and deserve to deal with the consequences. (and of course I think it's the former) Don't give us the responsibility without giving us the authority to carry it out! If I was trustworthy enough for my ex marry and if I was trustworthy enough for him to give me a springing power of attorney because as he told the attorney, there is no one in the world he trusts more than me, then doctors had no right to treat me as the enemy!

I'm on a soapbox today. The flu isn't any better, my mother is headed toward nursing home care because I can't take care of her at home while she is recuperating from her heart problems because I'm sick, and my daughter is dealing with the same problems I had to deal with with my ex with no resolution in sight.

The HD community needs to keep speaking up about unmet needs, about what we want in clinical trials, and about how we want to be treated. HD is hard enough without the people who are supposed to be helping us making life worse.



Edited 1 time(s). Last edit at 10/12/2009 02:30PM by Marsha.

This is Eric's post from another thread, i've copied it over to here, so we can talk about this on this thread. It's such a good post, i just wanted to be able to respond over here, so here is Eric's post:

Eric said:

Dr. Higgins who was at our state conference was going over available trials. It was a factual presentation. He went over what the compounds are, how they are suspected work, and the trial requirements. It was thorough enough, given the time he had, to pique interest.

When speaking of ACR16 he pointed out that the phase III trial in Europe was filled. Here it is not. And we need just 200 participants. He didn't comment more on that fact. I editorialized the "just" part.

Ok, now how can Europe fill their end of trial and we can't? Somehow, someone or something, has made it their job to fill the trial there. I am sure some countries there have many participants and others have few to none. So speaking of Europe as a whole is probably not fair.. and also I am sure there are cultural differences there from us. Doctors in some countries in Europe hold a different station there (in the doctor/patient relationship) than here for instance.

None the less something is lacking here in the process. I have no idea who is addressing this. Or even if it being addressed. If Europe is doing something right we are not, we need to look at that. If what they have done can't be duplicated here we have to come up with some other unique solutions.

Maggie's experience points out several things to me.

Firstly..her only vehicle of support is this forum. And on this forum, she has no piers which is sort of sad. So few have been through the process and watching her think through out loud the pros and cons of the trial, well that feels not right.

Secondly she is left to contact the company herself asking about compassionate use. The FDA as well. No FAQ's concerning trials in general or this one in particular. No contact person or persons.

I also am sorry to hear the Maggie is left dangling about enthusiasm about her trial experience even though she found it successful! To see improvement and then decline following the the drug stoppage is very hard to deal with. She had that part thought through.. but not she couldn't feel it ahead of time. We need to make sure these companies are pre-prepared for success and have the steps in place to push for compassionate use prior to starting the trial as standard procedure. Maggie and people like Maggie wouldn't be half soured over what should be hopeful and happy. It would also be a carrot to join a trial ... "if you and others are finding improvement... we will have the drug available to you after the end of the trial with your doctor's approval". Maggie doing the leg work for this is not right. I don't know how many people helped Maggie from here. I called the FDA on her behalf but I shouldn't have to do that. I am not the person to do that either. It was a cold call on a subject that I had very little knowledge about. But I knew there was no one else in an official capacity to do that.

Ok... Now we have two points of contact nationally between large groups of people with HD concerns in the US... the HDSA and the HDAC forum. I am not seeing a huge over lap of the two. This forum is inviting to everyone... and the best repository of general HD thought. I think the HDSA needs to begin extending itself. HD is developing issues and needs are surfacing that the HDSA is behind on.

I think the HDSA natl. board is way behind. I can only judge this by advancements and results and what conversation revolves around the HDSA. There is almost no conversation at all about the HDSA here. They don't participate here..where their audience is..and provide no interactive forum to discuss issues with them. Robi Bloomenstein seems able to communicate here. He just threw his e-mail addy out there. I very much have appreciated his participation. If there is a board member here besides Steve... I wouldn't know who that is. So if they don't read or communicate with us how do they know what's up? I am not even going to complain about the office..they move through the board. If Louise is the very best president to be had.. she can only do what the board allows. The chapters do what is asked. I won't hit them at all... the chapters don't dictate things. Maybe it's time to ask who the board is.. what they are doing... and see if they can actually be interactive in some way. All I see are names on a website. They mean nothing to me at all. They seem to be about as isolated as any HD family is. I know all are well meaning... and this isn't to beat them up. Just the dynamics are changing everywhere else and this one never changes. I have to question that. I don't even know who I am talking to here. Most people don't think about the HDSA here and I don't think the HDSA board is here to listen.

I just think that issues like trials should hinge on the HDSA and their active participation. Does that mean they will be dealing with people like me with opinions? Yes it does. Will I have completely unrealistic suggestions... probably. But the HDSA doesn't have act so fragile about that.

There is a community of all volunteers I am associated with... not like with HD... that acknowledged that improvements needed to made to their concerns. They started a thing asking the people what they saw as needing improving. The also spoke frankly to the volunteers... and said we can't spend a lot of money. We don't have it and won't have it soon. So they started a program called "100 papercuts". Identifying very small things that a person or two could fix for free or very cheaply. The idea was that fixing many small things equals a big thing. The community got very tight... the org can point to improvements... and more people were invited into the fold... making the org bigger and better. Rather than hide from problems they looked for them. They solved them. Not every single one. They were not looking to be perfect. No one expected perfection. And a few people did dissent sometimes. But outcome was that the org is stronger. It had vision. They didn't look for excuses to not be better and bigger based on money. We can't buy trial participants for instance. Money is not the answer to that. Top to bottom understanding and education can help that though. What are the paper cuts that make ACR16 not fill up? Can we fix those for the next trial? I think so. I don't think that anyone doesn't want things better. From the top or the bottom. But it's time for a vision.. more than make money, spend money, with no direction. Even if the ultimate goal is "the cure" how does that happen without people in the door to try it? Walks are not translating into trial participants..so what will? Does HDSA even want to be this kind of place? Or just remain status quo? Do we want to help the HDSA? I do. I think we need them. I think they can be what they want to be.

This forum can't be the everything of all things besides fund raising. What a tool this place could be for the HDSA though. I would love to get to know the board..the office.. the other chapter board members. Encourage their efforts, share ideas, complain sometimes... (Keeping it real on that one) Honestly, doesn't anyone not have a national vision? It doesn't have to mine... and if there is one..please share it. Anyone from the board..toss us a bone. There are some 7000 people hitting this site. We are not big meanies here..even if we are sometimes moody. But I am sure people would do a lot for HDSA if someone was here and asking consistently. Look at Tim using us... no one is complaining. People keep trying harder and harder just for the consistent effort he has used to encourage our help. The effort of the HDSA is appreciated..no one has said one negative thing. It's all positive. Why not this way with everything? Not everyone here is a political activist.. but this offers a thing for those who are or who would like to try to be. No one paid Tim, or me , or Patty, Now two Patty's.. Very costless. Is the board aware of this?..Do they understand this could be the norm? Do they see the value of this being the norm for the people they serve and for the HDSA? Learn and grow..grow and learn. Really... this could be a dawn for everyone. We have the possibility of connectiveness, drugs in the pipeline... more hope which could lead to action. I don't get why it's not being explored? I just don't.

To those who's names I used I hope I used them well. I meant to.