NT-3: one more report

[Guest guest]

(This is old news for most of us - but found this at MDA's website

and wanted to post anyway. For any of our new members who want to

know more and read all our other NT-3 information, look in our Files

section in the NT-3 Folder. We have the entire NT-3 medical article

there written by Dr. Sahenk, and you are free to print it off and

take it to your doctor. ~ Gretchen)

Neurotrophin 3 Shows Promise

http://www.mdausa.org/research/050830CMT_NT3.htmle in CMT

A pilot study of eight people with Charcot-Marie-Tooth (CMT) disease,

a disorder in which signals in the peripheral nervous system are

impaired, has found that treatment with neurotrophin 3 (NT3) improved

sensory function and nerve regeneration.

Neurologist Zarife Sahenk at the Columbus Children's Research

Institute Neuromuscular Program at Ohio State University led the

study team, which received MDA funding. Jerry Mendell, a neurologist

and MDA clinic co-director at Ohio State University Hospitals, was

also an investigator.

NT3 is a natural substance that belongs to a group of compounds known

as " neurotrophic " (nerve-nourishing) factors.

The investigators, who published their findings online July 6 in

Neurology, studied eight people with CMT1A, a form of CMT that

results from an abnormally duplicated PMP22 gene on chromosome 17.

The function of the PMP22 protein, which normally contributes to an

insulating sheath that covers nerves running between muscles and the

spinal cord (peripheral nerves), is disrupted, impairing sensory

(related to temperature, pain, vibration and pressure) and motor

(related to voluntary movement) signals.

After establishing that NT3 was apparently effective in mice with

PMP22 defects, the research team randomly assigned four adults with

CMT1A to receive injections of NT3 three times a week for six months.

Another four people with CMT1A were assigned to receive an inert

substance (placebo) that looked exactly the same.

At the end of the study, the placebo group's scores on a standardized

scale of generalized neuropathy-related impairment had worsened,

while scores in the treated group had improved.

There were no significant changes in specific sensory or motor tests

in the placebo group at six months, but the NT3 group showed improved

vibratory sensation assessed by a tuning fork test. Their reflexes

also improved. Motor function did not improve in either group.

Biopsies of the sural nerve, located in the calf, showed some

regeneration of the nerve tissue in the NT3-treated participants.

" We hope that this approach with neurotrophic agents can be applied

to peripheral neuropathies, where there are few treatment options, "

Sahenk said.