Cord Stem Cells and CMT 1A

[Guest guest]

Leena,

Below is the most recent abstract I have found relating specifically

to stem cells and CMT 1A. This is from our National Library of

Medicine. As you read, you'll see this is just now being experimented

in animal models, so IF this 'therapy' can one day benefit persons

with CMT (it will first benefit those with Type 1A, the most common

type), and since animal models are currently being used, this will

require repeated testing and randomized placebo controlled trials. In

other words, this will take some time - meaning my best guess here is

about 10 years away, UNLESS some amazing breakthrough happens, which

is indeed popular.

The diseases/disorders you mentioned will also most likely receive

first priority, simply because more people have the syndromes and are

affected, plus those organizations supporting the research for those

diseases/disorders have large amounts of money for this specific

research.

The CMT 'gene' has been evolving along with the 'human' for about 7

million years. It appears to be very strong and has been through its

own unique 'mutation', so simply " curing " CMT through only the stem

cell approach is far off. Remember, CMT is " genetic " . Symtoms of CMT

may in fact be treated with stem cell therapy one day, before a

complete arrestment of all types is made. (My last count was

something like 44 KNOWN 'types'). I'm sure you can imagine the

enormous amount of funding required and work by researchers to more

appropriately target genotype and phenotype and then get more help in

treating symptoms, while at the same time attempting to

completely " arrest " CMT.

The most promising research I have seen is the use of NT-3 (for CMT

1A) - there has only been ONE human clinical trial so far; and also

the animal studies of Vitamin C (Ascorbic Acid) which is now in the

first human trials in several European countries. You can find the

full text papers of the NT-3 trials and Vitamin C in our Files

section here at , in addition to PGD.

Pre-Implantation Genetic Diagnosis (PGD) is a type of in-vitro

fertilization in which the CMT gene marker can be completely removed,

thus resulting in CMT-free children from an affected CMT parent.

This, however, is only available for a small number of CMT types,

like 1A, 2E, etc.

I'd also suggest you contact the professional who presented the

lecture you heard and ask your questions.

~ Gretchen (read below)

From Methods Mol Biol. 2006;326:189-201.

Identification of transplanted human cells in animal tissues.

Benten D, Cheng K, Gupta S.

Department of Medicine, Albert Einstein College, Bronx, NY, USA.

The potential of cell and gene therapy has generated extensive

interest over the past several years. More recently, identification

of stem cells of various types, especially embryonic stem cells,

reinforced this interest. Systematic studies are now being launched

to define the biology of various stem cells, including after

transplantation of cells in mmunodeficient animals. This requires

robust and unequivocal means to identify transplanted cells. Ideally,

it should be possible to screen animal tissues for human cells with

relatively simpler methods, followed by more precise localization of

transplanted cells.

We describe the application of conserved primate Charcot-Marie-Tooth

disease type 1A repeat element for polymerase chain reaction-based

screening of animal tissues for human cells. Similarly, direct

polymerase chain reaction labeling of pancentromeric human alphoid

sequences with digoxigenin-UTP generates in situ hybridization probes

for identifying transplanted human cells. This pancentromeric probe

identifies human cells irrespective of the original tissue source and

can be combined with additional in situ methods to analyze cell

differentiation. Incorporation of these strategies will facilitate

translational studies aimed at understanding mechanisms concerning

the trafficking, engraftment, proliferation, differentiation and

function of human stem cells in animals.