As generic drugs all those have a clear indication. But put together in low-doses they have different indication. Pharnext suggests that low-dose combination could be a treatment for cmt1a. They are running their third phase this year.
At least someone is trying to 'do something' positive. Is seems so much worse when no one is bothered about your predicament. At my hospital the neurologist wanted to discharge me altogether. I insisted that I go for annual check ups in order to be a current patient. When they asked why, when there is no treatment available, I said; how will I know if there is ever a treatment in the future unless I am on your list of patients?
PCM, you are totally correct! Did they 'get it' then?
HM, I hope and pray this is the beginning of good things to come for everyone with all types of CMT!
SK I don't know if they 'got it' or not. All I got was "OK then". I am due my annual appointment next month. I expect it is just going to be the usual 'going through the motions' of seeing me again. However, I do ask, every year 'What have you personally done to help people with CMT in the last year? Their usual reply is 'there isn't much that can be done'. Not the answer I would like. My personal view of them is; they do just enough to keep their job and their pay packet and not a jot more. Am I just cynical?
Hey PCM,
No, I've never considered you to be cynical, but sometimes it doesn't take much to go there! Perhaps you should print out some info about the PXT3003, hand it to them and ask when they think it will be available to you in the UK! I'd challenge them to 'do their jobs' a bit more! See, I can go to cynical too!
Wouldn't it be nice if they had some good info for you that we didn't even know about! Cynical is okay for visit, just don't lose site of the hope, girlfriend! I'm still pulling for you all!
I've printed off some of the details, ready to take with me next month. So, wish me well.
When I found medical results about PXT3003 for phase two I went to see a doctor and asked for a prescription for low-dose Baclofen and Naltrexone. My general question to the doctor was that if it's allready known that this combinaison (BNS) has positive effect on cmt1a is it necessaire to wait many years untill it comes into the pharmacy (mixture). She understood my idea.
How does PXT3003 help CMT? What are side effects?
If anyone is interested I found this page today asking for people to register for research purposes.
Baclofen and Naltrexone are both old known generic medications, their side effects with normal doses are known. But how they work together (low-doses), that's just what Pharnext is testing at the moment. It's very personal what the side effect are f.ex. me, I am very sensible to medications. It wasn't easy in the beginning B and N i got some side effects but they vanished in a few weeks.
PXT3003 diminishes the abnormal growth of schwann cells if i know.... thereby the nerves electric velocity becomes better.
Carm said:
How does PXT3003 help CMT? What are side effects?
HM,
How long has it been since your Doctor prescribed this med combination for you? I would have also asked to be on this, the meds are not unusual, and not strong. I have great hope that you will notice a difference for the better any time now. I know that with some of the meds I have taken for autoimmune, especially the biologic Enbrel, it took months, but it was worth the wait when it finally 'kicked in'.
I send you my best, and hope that more members will take this info to their Doctors, like PCM is doing! Time to wake them up!
SK,
It's two months ago when my doctor prescribed for me Baclofen 5mg x 4/day, Naltrexone 1mg x 4/day and a "tea spoon" of sorbitol devided into 4 portions/day.
SK said:
HM,
How long has it been since your Doctor prescribed this med combination for you? I would have also asked to be on this, the meds are not unusual, and not strong. I have great hope that you will notice a difference for the better any time now. I know that with some of the meds I have taken for autoimmune, especially the biologic Enbrel, it took months, but it was worth the wait when it finally 'kicked in'.
I send you my best, and hope that more members will take this info to their Doctors, like PCM is doing! Time to wake them up!
Here is an article about pleotherapy:
On page 1168 you find this:
"PXT3003 is a combination of the generic
drugs baclofen, normally used to treat spasticity,
naltrexone, usually used to treat opiate and
alcohol addiction, and sorbitol, a laxative."
This information is very good and easy to understand. This is not only something great for CMT1A, but a glimpse into the new ingenious way that meds will be configured.
They proudly state that they have had positive results in improved motor functions and muscle strength in the rats tested, and they are always a favorite test animal. As promising as that is, it goes on to say that this is NOT the only combo med being tried for CMT1A.
Thank you so very much for this, not only is this happening now, it's a glimpse at the future of medicine!
SK said:
This information is very good and easy to understand. This is not only something great for CMT1A, but a glimpse into the new ingenious way that meds will be configured.
They proudly state that they have had positive results in improved motor functions and muscle strength in the rats tested, and they are always a favorite test animal. As promising as that is, it goes on to say that this is NOT the only combo med being tried for CMT1A.
Thank you so very much for this HM, not only is this happening now, it's a glimpse at the future of medicine!
This is the approval for the European and US markets, correct?
HI!
Thanks for all the cool links to Dr Cohen's research especially the youtube video. After some google stalking I found a couple of references containing dosage information for Naltrexone/Baclofen/Sorbitol in the trial. (See below) The first reference has the actual doses for the low/medium/high dose arms of the trial. The second reference makes it clear that patients were dosed twice daily. This makes sense to me as the half-life of naltrexone and baclofen are on the order of a few hours ( Wikipedia :-)
The highest dose values from the trial are:
Naltrexone = 0.35 mg
Baclofen = 3 mg
Sorbitol = 105 mg
http://apps.who.int/trialsearch/trial.aspx?trialid=EUCTR2010-023097-40-FR
http://clinicaltrials.gov/show/NCT01401257
Yes the doses are really small. My doctor was extremely open to prescribing me these drugs especially in these low doses maybe yours will be too. If not, shop for a new doctor :-) I had to go to a compounding pharmacist to get such a low dose of naltrexone though and a friend is sourcing the sorbitol from a reputable supplier.
Hopefully this time next week I will be on my own little trial. I will need a home test for how effective the treatment is as self reporting over such a long time can be difficult in such a slow moving disease. I think I will build myself a "9 hole peg board" and time how long it takes to add and remove the pegs. It is a standard neurological test I can easily do at home.
Wish me luck!
CHEERS to you all :-)
Hi Tina,
I am very excited that your Doctor responded in such a positive manner and is prescribing these for you! I am ever hopeful that this will make a substantial difference in your state of well being. Look forward to hear of your progress! Thanks for posting!