pxt3003

General General CMT Discussion
1

Hi!

Pharnext is developping their pleotherapeutic medication PXT3003 for CMT1a. http://www.pharnext.com/pipeline/cmt-program

Phase II was positiv. Look key facts: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2014/05/human_orphan_001344.jsp&mid=WC0b01ac058001d12b

They are runing III phase now. This is very intresting because all ingredients for pxt3003 are generic medications allready used for other indication.

2

Oh this is tremendous!

Here are what the ingredients are

http://www.drugs.com/baclofen.html

http://www.drugs.com/ppa/naltrexone-hydrochloride.html

http://www.wisegeek.org/what-is-sorbitol.htm

https://answers.yahoo.com/question/index?qid=20080918152348AA0PN0l

3

Seems that you are already on the right track with the LDN! I hope this is a giant success for all CMT1a's!

4

So do I, SK. Thanks for posting this, HM!

5

What are the ingredients?

6

Baclofen, Naltrexone hydrochloride, and D-sorbitol, and the definitions are on my first post.

7

I thought sorbitol is just a sugar substitute, can't see how that can help. In fact, just the opposite.

8

Too much sorbitol causes diarhea. I don't take so much. Sorbitol decreases PMP22 levels as do Baclofen and Naltrexone. First idea is to use three medications which they all decrease PMP22 levels by different pathways but together are strong enough to lower PMP22 levels enough. The other idea is that this way low-doses can be used, so, side-effects are minimalised.

http://www.google.com/patents/EP2211846B1?cl=en look at point 0043, "Targeted metabolic pathway in the CMT1A disease", and points 0046 and 0052.

PCM said:

I thought sorbitol is just a sugar substitute, can't see how that can help. In fact, just the opposite.

9

So where does Pilocarpine, Methimazole, Mifepristone and Ketoprofen fit in? What effect do these have? Has anyone every tried any of these? Or Baclofen and Naltrexone?

10

Pilocarpine, Methimatzole and Mifepristone are much more "heavy" and dangerous medications than Baclofen and Naltrexone, so, I suppose this is why Pharnext chose NBS combination. Ketoprofen belongs to NSAID group... no good. My doctor gave me a prescription for low-dose Naltrexone, low-dose Baclofen a few months ago.

11

Here is Pr. Daniel Cohen's (Pharnext) presse conference https://www.youtube.com/watch?v=lJC95yCZ5wA At point 23:32 "Prèmieres Conclusions" for phase II are represented.

SK said:

12

Is this really true?!?

13


Carm, here is a summary of the trial from the Musclular Dystrophy Campaign:

http://www.muscular-dystrophy.org/research/clinical_trials/418/charcot-marie-tooth+disease


Carm said:

Is this really true?!?

14

Thanks dancermom! Wondering if this would be effective long into the disease? Damage is already done?

15

That I don't know, Carm. Hopefully, we will find out more soon...

16

yes, perhaps good news for my two boys! Can't help it but this CMT is really starting to bother me emotionally...trying to keep strong.

17

HM said:

Pilocarpine, Methimatzole and Mifepristone are much more "heavy" and dangerous medications than Baclofen and Naltrexone, so, I suppose this is why Pharnext chose NBS combination. Ketoprofen belongs to NSAID group... no good. My doctor gave me a prescription for low-dose Naltrexone, low-dose Baclofen a few months ago.

How are you finding it in helping you or is it too soon to know?

18

I also find that CMT bothers me emotionally. It's almost like I feel 'stuck'. Not moving forward in life generally, just trying to get through one day at a time etc..... I wonder if anyone else feels like this. Like my feet are set in concrete and I can't go anywhere.

19

I've been trying to figure out what they (Pharnext) mean with "Low-dose Baclofen" and "Low-dose Naltrexon" and what could be a good dose for sorbitol. I asked in the pharmacy about this subject. They told me that Baclofen 25mg/day is a normal dose, so, low dose is under it then.

Maybe this http://www.antiaging-systems.com/113-naltrexone gives a hint that low-dose naltrexone is around 4,5mg.

Sorbitol looks somewhat difficile but anyway it must be so little that it doesn't cause diarrhea. In my case it's 1-1,5 gramms /day devided into three portions.

20

Sorry, what are these drugs used for and what do they treat?