Stem cell treatment for CMT

[Etxalar]

There is a clinic in Cancun (world stem cells) that offers stem cell treatment for CMT sufferers. It is costly at 15000 US dollars. Has anyone got any views on stem cell treatment for CMT? I have CMT type 1A and I am in a position where I can afford the treatment. I am sceptical about the benefits and possible complications on this type of treatments. Any views on that?

[CMTUnitedKindom Partner]

There is NO viable treatment for CMT - for any type - available anywhere in the world, yet, least of all using stem cells.

Please don't waste your dollars on this - because it won't work. We're still at least three years, if not more, for a drug treatment for type 1a, assuming it works when the current research gets to human trials - and who can predict that?

Sorry, but it's probably a scam.

Karen

[Etxalar in reply to CMTUnitedKindom]

Thanks a lot for your reply Karen.

Could you please provide a link or information regarding this drug research?

[Stetson28 in reply to CMTUnitedKindom]

Hello what is the mfn2 gene 1 bad copy causing? Is that cmt type 2a or does that require 2 bad copies. Thank you

[SAMBS in reply to CMTUnitedKindom]

Is there any progress and advance on your reply above Karen regarding any drug or treatment for CMT that would alleviate the symptoms of numbness and nerve pain?

[spabbygirl]

It's really good to have that confirmed Karen. I had an email from CMT US wanting to raise money for research towards treatment but it said nothing about what sort of treatment so I found it a bit disappointing. I'd like to know if the research is to repair the nerve tissue, or the myelin sheath or just pain relief or something else.

Maybe I should email and ask or look at their website.

[CMTUnitedKindom Partner in reply to spabbygirl]

Research is for all sorts of things - much is still learning - what does this gene do, how does that protein work in nerves and that kind of thing.

But they are also trying to figure out how to reverse or control symptoms by finding out if any drugs will have an effect on those nerves.

It’s an incredibly long process and a difficult one. For instance, in type 1a, we know there is an extra gene of PMP22. PMP22 is a protein that is required for the health and maintenance of peripheral nerves, and we’re getting too much of it. So research is working on reducing this over-production. But if you reduce it too much, you cause another condition called HNPP!

And of course, any drug developed will have to be taken for the whole lifetime, probably from childhood to the end of life. So it must be incredibly safe and side effect free.

Other research is working on “here and now” stuff - like learning whether exercise helps slow progression (it does!) and what outcome measures are needed to measure success in drug trials - no easy task with a slowly progressive disease that may not change much over two or three years.

Does that help? If you want more information, go to the research section on our website.

[spabbygirl in reply to CMTUnitedKindom]

Thanks so much for those 2 replies Karen, I should look at our website more often! I will in future. I didn't know about PMP22, but yes the balance of biochemicals is very sensitive & vital to get accurate. I have been having trouble with my vision, which my new neurologist attributes to CMT in the form of optic neuritis, but more recently I have been having trouble with momentary loss of voice and difficulty swallowing. A vet friend & my dentist say its probably caused by CMT because the same nerve serves eyes and mouth. My consultant was less sure but I'm waiting for an appointment with him to find out his view. I have had other tests to rule out anything else so will wait and see!

[CMTUnitedKindom Partner]

CMTA are primarily researching drugs that will - in the case of type 1a, at least - stop the over expression of PMP22. As you know, in that particular type, we have an extra gene, which causes too much of the PMP22 protein to be produced. Why exactly this is bad, is still a bit of a mystery, but basically anything that upsets the delicate balance of the workings of the nerves is bad news - and all the types feature missing genes, or faulty genes leading to a variety of issues with that balance. So drugs are being tested in stem cells (in the laboratory) to see if any can stop this over-expression, which would arrest the condition. How exactly this would work in people with existing degeneration still remains to be seen, of course. And of course, as was found out with the Vitamin C trial - what works in the laboratory (in mice in that particular case) doesn't always translate to benefit in people, so there are many steps to go yet.

You can read more at cmtausa.org/index.php?optio...

We are having a meeting with Professor Mary Reilly at the end of February, at which we'll get all the latest up to date information on research (Mary is involved in the collaborative process with all the folk in the States being funded by CMTA), I'm also going to a scientific meeting in Oxford in March, where Dr Mike Shy, the leading light behind CMTA's programme will be talking about ; another meeting in Amsterdam at the end of April - that's focussing of specific types of type 2; and I'll also be attending the CMT International Consortium meeting of scientists in Antwerp in June. So over the next few months, our specific knowledge of what exactly is going on will increase dramatically!

Mary Reilly is also talking at our annual conference in April - I'll be uploading the paperwork to our website for members when I've finished answering this question (!) and will also be focussing on research in her lead talk.

So if you're not a member, think about joining at cmt.org.uk and get the latest information from the UK's leading expert in CMT!

Karen