Gene Therapy and remyelination as a cure - AMN EASIER


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Gene Therapy and remyelination as a cure

diderot555 profile image

Hello all,

A good friend of mine was diagnosed yesterday with AMN.

He already started having symptoms two years ago when he couldn't run more than a kilometer, and since then got a little worse. Today he can't run, and walks quite slowly with difficulties.

1) I have read in some researches that a successful Gene Therapy was conducted on two children with ALD and the levels of accumulated VLCFA were drastically reduced and the ALDP protein restored.

What's going on with that? seems pretty awesome. Do you think there is a chance in the near future that a gene therapy can treat this disease?

2) Also, I read that there is a process of remyelination, the can actually restore the damaged myelin and reverse the damage so that the person can actually walk normally.

Thank you all for the support and help.

11 Replies

I think successful gene therapy for adults is only a matter of time, though how much time? Who knows?

As for remyelination, there are plenty of contenders as far as drugs/supplements. I took CDP Choline for a while. Didn't work, then again I didn't take for that long.

Peptides, nerve growth factor, human growth hormone.

Check the website thisisms plenty there.

diderot555 profile image
diderot555 in reply to monkeybus

Hey monkeybus, thank you for the quick reply!

Is the gene therapy an actually possible cure? Will it force the body to create the missing protein completely?

Regarding remyelination, what is this drug you mentioned? Is there a drug the is our there to buy for remyelination?

Thank you!

Aaron98 profile image
Aaron98 in reply to diderot555

There needs to be more money going into gene therapy studies, just to make them happen. And then, a few men with AMN need to step up and get it done. I would absolutely considering doing it, if it could lead to a successful treatment.

monkeybus profile image
monkeybus in reply to Aaron98

I'll fight you for any position in a gene therapy trial.

monkeybus profile image
monkeybus in reply to diderot555

CDP Choline is a supplement. Healthy stuff anyway. Great for the brain.

Choline is cheap, but CDP Choline isn't.

You'll need to buy bulk powder to get any kind of discount. I bought several kilos a few years back. Still have it. I kept threatening to make a list of remyelination agents on here. I'll get back on it, but Google will see you right.

Try this, OTC Hay Fever medicine as remyelinator -

OTC Antihistamine May Repair Myelin Damage in Chronic MS

I just saw an article on The United leukodystrophy Facebook page discussing the possibility of a new treatment for rebuilding myelin in MS. We tend to try to catch the “run off” of MS research so this is good news for all!

diderot555 profile image
diderot555 in reply to tieaknot

I'm happy to hear that! Thanks.

SongStream profile image
SongStream in reply to tieaknot

During my last visit to Stanford for the clinical trial, my coordinator was saying that she will be assisting in a new clinical trial for MS involving stem cells. As previously said, our diseases mimics MS and I am excited of its application.

I just found out that the trial begins in June using one's own bone marrow stem cells, thus, eliminating a donor, radiation and chemo therapy.

This stem cell therapy is used for the lost myelin or for the fault gene?

I've included a link that has additional information from the National MS Society. From the site:

Repairing myelin, the coating that surrounds and protects axons (nerve wires) and which is damaged by MS, may represent the best strategy for protecting axons from injury and improving function for people with MS. Thanks in part to our pioneering funding, potential cell therapies and myelin repair strategies are now approaching or already in clinical trials.

There is exciting progress being made through innovative research related to the potential of many types of stem cells both for slowing MS disease activity and for repairing damage to the nervous system. In addition to cell therapy, finding ways to stimulate the body’s own stem cells inside the brain is another approach being actively investigated. Today the idea of nervous system repair holds significant promise as a strategy to restore the function that MS has taken from people; and reducing or stopping MS progression.

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