That's a good point rj, remember people in the US used to get TBZ from canada and other countries

WHOOOO WHOOOOO !!!!!!!!!

The drug is being made available by NeuroSearch free of charge to individuals who have completed the study. We are working now to get FDA approval for Tony to start it again. Below is the email from the doctor who is the clinical project leader for the trial. It explains how the drug will be given to pHDs after the trial.

I am finding waiting for the family doctor to do the paperwork is very hard especially after seeing the European results which were so much like the results that Tony had. Like the kid you take on a car trip who asks every ten minutes, ?Are we there yet?? I want to call the doctor?s office every day and ask, ?Have you filed the forms yet??

Off topic?..Tony and I will have our 48th anniversary on Friday!!! That?s a lotta years.

NeuroSearch email

Thank you for your e-mail.
NeuroSearch would be happy to provide pridopidine, free of charge, to your father under an expanded access program in the US. This requires involvement of your father?s treating physician. The rules for expanded access to investigational drugs for treatment use in the US were recently amended and NeuroSearch are willing to support your father's physician in meeting FDA's submission requirements for a "single patient IND". In essence this means that we will provide our permission for FDA to refer to any information in our IND - which is the assembled paperwork that we have submitted to the FDA for the development of pridopidine to treat Huntington's Disease - to support a single patient IND for your father. Your father's treating physician may contact me directly for further discussions about the possibility of a single patient IND.

An extension to the HART study is taking longer than expected and we are looking into alternatives. The clinical data from the European MermaiHD study will be available next month, and depending on those there may be possibilities for a broader program. However, that remains to be determined once we know what the data tell us.


Med venlig hilsen / Best regards,



Edited 1 time(s). Last edit at 02/04/2010 02:10AM by Maggie.

Oh i hope it happens quickly for you Maggie

If Marsha is excited then that means there is really alot to look forward to. I really wish my sister would try for this trial. I feel she would do very well. I have brought it up a couple of times but she just pretty much ignores the idea.

Stacy-NJ

Stacy there's no better time to give her a big loving kick up the arse than right now. The drug clearly works, get her into that trial

We need to get that trial started asap, I'm not even in the States, I'm in the UK. The quicker things happen world wide the quicker we stop this darn disease.

[www.hdyo.org]

Dr. LaVonne said on the HUNT-DIS list that only 30 more are needed for the US trial.

Will

OMG this is soooooooooo friken' exciting!!!

My hubby and I have an appointment already set up for next tues for my 4 month follow up to my Dr. I just printed everthing out. We will bring her the info on it.

THIS IS THE BEST WEBSITE EVER!!!!!!!
OMG!!!!!!!!!
Bridget

Maggie, I would SO be calling the doc office every day, asking about the paperwork! (Or have different family members take turns!)

Happy Anniversary!

Hi All - I am new to this forum although I have followed for a while. my husband was just about to start the HART study in Long Island New York but he is pretty early on (very recently diagnosed) and we were encouraged to deal with his psychiatric symptoms first with meds that were not allowed in the HART study... with these wonderful results I am wondering if we made the right decision... I hope that the drug will be available in US ASAP... also, I am assuming that the exclusion list of meds was just for the purpose of the trial and when it is on the market this won't be an issue - is that correct? Also, I want to say how ecstatic I am about this news for everyone out there affected!! Hope makes all the differece!!

The exclusion list of meds was just for the purpose of the study. The European study included a group of people on psychiatric meds to see if the drug was effective and that there weren't any additional side effects. Everything was fine!

I have a question here, relating to what Hope2. Maybe Marsha can answer.
NeuroSerch's announcement says "The use of antipsychotics showed no influence on the positive treatment effects of Huntexil". Then is says 40% of the volunteers were on antipsychotics. If I get it right, antiphychotics were an exlcusion criteria here in northamerica. My questions is: why??? And could they accept more people now, knowing what we know from the european study so we can finish the study here in Northamerica?

Ouch, too slow again... but still, could this change the exclusion criteria in Northamerica?

Good question. I suspect they won't take the time to apply for a change since there are only a few more enrollees needed.

Thanks Marsha. But then I hope this will be a lesson for the future.

Thanks Marsha! That is awesome news!

Way to go hope2.
I finished the study last oct- I did not know how impoortant it was until I read about it on this forum!
Take care
Bridget

Hi Maggie,

What approach did you take in order to get a response?

For example was it all done through email, phone calls etc...?

I am not interested in the contents of the message as this is personal to your family, however, would be interested in knowing the steps taken.

Thanks in advance for your time.

Kip Jones.
Ontario, Canada

Hi, Kip,
Actually, NeuroSearch was very responsive. My son, Jason, and I were determined to get the med approved under the compassionate use. Tony had done so well on it during the trial but I thought that it would take many, many months. I sent an email but did not know who to send it to and it was not answered. Jason called Sweden at 3:00 in the morning and was passed around in an effort to find someone who could speak to him in English. Finally, he was transferred to a woman who not only had good English but was very interested in Tony?s experience with the trial and how he had done. She was amazed that we drove so far to be in the trial. She gave Jason the name and phone number for the clinical project leader.

When we got the direct email address of the clinical project leader, our emails have been answered the following day. Remember they are having their work day while we sleep at night here because of the time difference. The following is from Jason?s HD blog and he tells the story.

It was because of Jason?s phone call that NeuroSearch put the effort to get the drug to people who had been in the trial as a priority in their company.
________________________________________
Aug 28 2009
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 one 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.

Does anyone know if the people that participated in phase 1 and 2 of this trial are being followed. I know they didn't want to take on too much in order to get the drug approved but I am curious if information is being gathered on long-term usage. Will Huntexil affect the disease itself - is there a reason to start using this med even if the patient has minimal movement issues.... Thanks!