I asked my doctor and also in the pharmacy about those numbers what do they mean. But they didn't know what they ment.
Tina said:
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 :-)
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.
I asked my doctor and also in the pharmacy about those numbers what do they mean. But they didn't know what they ment.
Tina said:
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 :-)
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.
Goodluck! Please keep us posted. The doctor that is going ahead with this, is he a Neurologist? How would these three drugs help CMT1A? I need to research more...
Hi Tina
I asked my doctor and also in the pharmacy about those numbers what do they mean. But they didn't know what they ment.
Tina said:
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 :-)
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.
● for compound D from about 1 to about 20 mg per day if day if taken orally.
● for compound B from about 2 to about 20 mg per day if taken orally. The different doses may be suitable if administered in form of nanoparticles or similar formulations.
I would be intrested how do you understund those numbers. You think "concentration number" means dose in mg or how? This is important I think. My doctor gave me prescription for Baclofen 20mg/day and Naltrexone 4mg/day.
Product Name: non applicable Product Code: PXT3003 Pharmaceutical Form: Oral solution INN or Proposed INN: Baclofen CAS Number: 1134-47-0 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 3- INN or Proposed INN: Naltrexone CAS Number: 16676-29-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.35- INN or Proposed INN: Sorbitol CAS Number: 50-70-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 105-
Tina said:
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 :-)
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.
No, not yet. But pain is still mostly away which is a miracle. I’ve hard to believe it. If this state will continue it’s a good sign. I’m waiting if some senso-motoric improvements will appear which would be much more than a miracle.
SK said:
Hi HM,
Just checking in to see how you are doing? Any other subtle improvements noticed with the PXT3003?
Sorry it has taken me so long to reply, I have been on a course :-)
I think you're correct. I was mislead by the units being mg. If we assume that units should be per ml, combine with dose of 5ml , twice daily then dosage comes out as:
Sorbitol 525 mg Baclofen 15 mg Naltrexone 1.75 mg
twice daily. Which somewhat overlaps with your experience.
Interestingly the stage 3 trial has an arm with twice this dose. Will be interesting to see how that goes when they report back.
Hmmm... back to my friendly GP. Thanks for the headsup, I appreciate having someone to check against :-)
It may be that way, but if we take a look at the dosage for Baclofen, 30mg/per day is not anymore a low-dose, it’s a normal dose. And high-dose for Baclofen is about 75mg/per day if I know. All ingredients for PXT3003 are low-doses. Low-dose for Naltrexone is >5mg. The question is what a low- dose for Baclofen if 25mg is already “normal dose”?
Tina said:
Hi!
Sorry it has taken me so long to reply, I have been on a course :-)
I think you're correct. I was mislead by the units being mg. If we assume that units should be per ml, combine with dose of 5ml , twice daily then dosage comes out as:
Sorbitol 525 mg Baclofen 15 mg
Naltrexone 1.75 mg
twice daily. Which somewhat overlaps with your experience.
Interestingly the stage 3 trial has an arm with twice this dose. Will be interesting to see how that goes when they report back.
Hmmm... back to my friendly GP. Thanks for the headsup, I appreciate having someone to check against :-)
I reviewed the youtube video again, particularly the slide at about 14:09 minutes in. It's a bit low resolution but the relative amounts in the high dose group are baclofen = <1/10, Naltrexone = 1/100, and Sorbitol = 1/5. Can't quite read the doses listed bellow them, but the doses looks like baclofen = 6mg, Naltrexone = 0.7 mg, and Sorbitol = 20mg.
This would suggest that my original thoughts of baclofen = 3mg twice daily, and naltrexone 0.35mg twice daily are not far off the mark. Also reconciles to "normal" doseages being baclofen ~ 80mg/day, and naltrexone ~ 50 mg/day.
Howerver I can't reconcile the Sorbitol dose. If I allow for a mistake on the slide where 20 mg should be 200 mg then at least that would match the twice daily dose(2 x 105mg). However a "normal" dose is anywhere from 15 to 30 to 150 ml at 70% concentration. 1/5 of the minimum is 15/5*0.7 = 2.1 g which is 10 times the twice daily dose. This will only reconcile if there is a second mistake on the slide and 1/5th should be 1/50th.
I'm undecided on what dose to ask for. I might just keep going with my current dose and see what happens if anything. And maybe double the dose when I need to refill the prescription.
Hopefully the research group will publish their findings soon and put me out of my misery :-)
Naltrexone 4mg per day
Baclofen 20mg per day
Sorbitol 1-2g per day
It surely takes time to see the motoric improvements, maybe more that a year. It depends on how bad CMT1a one has.
Tina said:
HI!
I reviewed the youtube video again, particularly the slide at about 14:09 minutes in. It's a bit low resolution but the relative amounts in the high dose group are baclofen = <1/10, Naltrexone = 1/100, and Sorbitol = 1/5. Can't quite read the doses listed bellow them, but the doses looks like baclofen = 6mg, Naltrexone = 0.7 mg, and Sorbitol = 20mg.
This would suggest that my original thoughts of baclofen = 3mg twice daily, and naltrexone 0.35mg twice daily are not far off the mark. Also reconciles to "normal" doseages being baclofen ~ 80mg/day, and naltrexone ~ 50 mg/day.
Howerver I can't reconcile the Sorbitol dose. If I allow for a mistake on the slide where 20 mg should be 200 mg then at least that would match the twice daily dose(2 x 105mg). However a "normal" dose is anywhere from 15 to 30 to 150 ml at 70% concentration. 1/5 of the minimum is 15/5*0.7 = 2.1 g which is 10 times the twice daily dose. This will only reconcile if there is a second mistake on the slide and 1/5th should be 1/50th.
I'm undecided on what dose to ask for. I might just keep going with my current dose and see what happens if anything. And maybe double the dose when I need to refill the prescription.
Hopefully the research group will publish their findings soon and put me out of my misery :-)
I have asked my GP to prescribe Baclofen, Naltrexone hydrochloride, and D-sorbitol in the low doses discussed above. Problem is, in the UK, the dosage of Naltrexone is 50 mg and she feels unable to prescribe at 5 mg.
Any ideas or help would be massively appreciated as to how I can get a prescription for LDN here in the UK.
Hi everyone! Hope all of you are well...we were on this topic a while back with SK. Now its come up again on the forum. In reading all of the above about PTX3003, and any of you who are on it, in what way does this help CMT type1A???? Just had my appointment with a neurologist and totally forgot to ask about this. I am in Canada, so not sure if this is even available here. Also, has this been approved?
Hi mo, I’m on my iPhone and autocorrect won’t allow your screen name, so for now, you’re ‘Mo’. It is so freezing cold where my computer is from these Gale winds…
The best thing I can think to ask about getting LDN in 5 mg is if it’s available in Canada? Because it’s a land of the UK, it may be possible that with a prescription you can get it through the mail. It’s a question worth asking.
Our member HM is the only one I know of who has taken this, so rereading his entries on this would be the place to start. I’m sure that if you have further questions, he would be glad to answer them.
It's important to realize that these meds are not yet through clinical trials, so it is not something you will find easy to talk your Dr into, no matter how beneficial we may think it may be.
I think it is a good idea to follow up on the clinical trials and see if this actually hits the markets. It is perhaps a good idea to take all info you can find to your Dr, so that he is aware that this is being tried for some types of CMT.
I am ever hopeful that this is deemed safe and successful and becomes readily available by prescription.