Neurotrophin 3 Improved Sensation in CMT1A (Repost) Quest Article

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QUEST Volume 12, Number 6, NOVEMBER/DECEMBER 2005

(This is from the MDA magazine and says nothing new that we at

haven't already heard on NT-3, but may perhaps be of value to our new

members. You may want to look at our NT-3 File for more information

and Dr. Sahenk's complete paper. Dr. Sahenk announced her news

originally in the Fall of 2003 at AAN in San Francisco. The NT-3

study was funded in part by a grant from The Neuropathy Association,

as well as funding from Regeneron Pharmaceuticals. NT-4 and 5 are

also now in mice research stages, however, not necessarily for CMT.

NT 3,4, and 5 are nerve growth factors. ~ G

Neurotrophin 3 Improved Sensation in CMT1A

A pilot study of eight people with type 1A 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 MDA-

funded study team. Jerry Mendell, a neurologist and MDA clinic co-

director at OSU Hospitals, was also an investigator.

NT3 is a natural neurotrophic (nerve-nourishing) factor.

The investigators, who published their findings online July 6 in

Neurology, studied people with a form of CMT that results from an

abnormally duplicated PMP22 gene on chromosome 17.

In CMT1A, 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 and motor signals.

After establishing that NT3 was apparently effective in mice with

PMP22 defects, the research team gave four adults with CMT1A

injections of NT3 three times a week for six months. The other

subjects received a placebo.

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

scale of 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 didn't 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.