MDA Research News has a short article about the success of NT3 for nerve regeneration in a small study of CMT1A patients. Again, this isn’t specific to Dejerine-Sottas, but may turn out to be applicable for future research.

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.

The same family of chemical signals that attracts developing sensory nerves up the spinal cord toward the brain serves to repel motor nerves, sending them in the opposite direction, down the cord and away from the brain, report researchers at the University of Chicago in the September 2005 issue of Nature Neuroscience (available online August 14). The finding may help physicians restore function to people with paralyzing spinal cord injuries. [Medical News Today]

Zarifa Sahenk, M.D., director of the Neuromuscular Division and the Experimental Neuromuscular Laboratories at Ohio State University, outlined advances in her studies of the role of neurotrophin-3 (NT3) in nerve regeneration in patients with Charcot-Marie-Tooth (CMT) neuropathy. NT3, which encourages nerve growth, is secreted by the Schwann cells that form a protective insulation (myelin sheath) around peripheral nerves. [The Neuropathy Association]

By studying nerves in “pre-tadpole” frogs, researchers at the Johns Hopkins Institute for Cell Engineering have uncovered the first link between two key biological factors that guide growing nerves.
The finding sheds light on how nerves grow in the right direction so they can connect to the right places–critical information to have if damaged nerves are ever to be repaired in people. In particular, the discovery reveals for the first time how the guidance cues that attract or repel the tip of a growing nerve influence the flow of calcium ions into the nerve cell, solving a decades-old mystery.
“For 20 years researchers have known that calcium flow is critical for proper nerve growth, but no one has known how it gets into the nerve in response to a guidance cue,” says Guo-Li Ming, M.D., Ph.D., assistant professor of neurology in the Institute for Cell Engineering’s Neuroregeneration and Repair Program. “Now we have some details about how that happens in frogs. The findings are likely to hold for other animals and people, too, because we have similar versions of these proteins.”

Read the rest of the article at Science Daily.

The University of Pittsburgh is making a big push into drug discovery by opening a laboratory that will focus exclusively on treatments for rare diseases, especially some kinds of cancer….
New treatments for a range of similar diseases, including Parkinson’s, Lou Gehrig’s and Alzheimer’s, also may be discovered in the new lab, according to Dr. Steven DeKosky, director of the Alzheimer’s Disease Research Center at Pitt. In this family of medical problems, protein molecules go bad, setting off a cascade of cellular changes that result in neuromuscular problems, including difficult speech and movement, DeKosky said.
The hope is to find compounds whose molecules block the start of the cascade, kind of like finding a key that fits a specific lock, said DeKosky. “It’s a hugely exciting time.”

Read the rest of the article at MSNBC.

Until recently, Dr. Stephen G. Waxman, director of the Yale University-affiliated Center for Neuroscience and Regeneration Research, refused to use the c-word.
Repairing severed spinal cords, treating multiple sclerosis or relieving the intense pain that lingers after an amputation were far too distant goals.
“Cure” was a false promise.
Now, restoring certain physical functions and mending torn, severed or diseased nerves is almost in reach.

Read the full article.

From HNF-Cure.org: The first major advance comes with the passing of the “FY 2005 Labor, Health and Human Services, and Education Appropriations” bill by the United States House of Representatives. This bill includes a call by Congress to have the National Institute of Health (NIH) investigate mechanisms of increasing federal research on CMT and CMT-related disorders. The NIH is to report back to Congress by March, 2005, paving the way for increased research in the near future. An associated report the notes that [Congress is] “concerned about the prevalence of this disease and its effect on people across the age spectrum and recognizes the value of CMT research for advancing understanding into other neuromuscular disorders. The Committee encourages NIH to identify new research opportunities on CMT that could lead to a relevant program announcement or request for applications.” The legislation must be also be reviewed by the U.S. Senate, but once enacted will direct funds to the NIH to enable the review to be undertaken.

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