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STAR’s Transformation Project 


CMTA’s STAR initiative announces “The Transformation Project” – Adult Human Stem Cell testing to find effective compounds to treat CMT.

STAR Transformation Project Graphic For the first time ever, live adult human stem cells will be used to search for effective compounds to treat CMT.

By using cutting-edge technology developed at the University of Wisconsin’s Waisman Center, live human skin cells can be transformed into cells that can be tested against pharmaceutical compounds for the purpose of finding candidate drugs for use in human clinical trials.

The CMTA has embarked on this groundbreaking STAR research project to rapidly accelerate the drug development process in search of a successful treatment to slow, stop or potentially reverse the progression of CMT. 

In addition to searching for effective CMT treatments, results discovered in STAR “The Transformation Project” will have potential applications in other closely related disease’s such as ALS, MS, Parkinson’s and Alzheimer’s.


The Time is NOW!

The cost to fund STAR “The Transformation Project” is $100,000; we can’t do this alone and need your financial support.  The CMTA humbly invites you to partner with us in this historical journey by making a donation in any amount to STAR “The Transformation Project.”

[An anonymous donor has offered to match donations made to the Transformation Project dollar-for-dollar up to $100,000. This means that with your contribution, we can potentially raise a total of $200,000 to fund our adult human stem cell testing in the search for effective compounds to treat CMT. Learn more…]


Unlike other neuromuscular disorders the causes of CMT are known to be linked to at least 33 specific gene defects. More importantly, these genetic mutations have been replicated and grown as tissue cultures allowing for the screening of 350,000 compounds to test for possible treatments.

The Charcot-Marie-Tooth Association (CMTA) has initaiated the Strategy to Accelerate Research (STAR)™ to fund CMT-related research.  The STAR program is unique in that the prominent, international researchers are sharing information and working together to find a cure.

CMT1A , the most common form of CMT,  is caused by a duplication of a gene leading to an overproduction of a protein named PMP22.  The over-expression of PMP22 causes the deterioration of the myelin sheath that surrounds the nerve fibers, or axons. Ultimately the axons deteriorate as well, so that  nerve impulses no longer transmit efficiently, resulting in the weakness and loss of sensation that characterize CMT1A.

Vitamin C and a progesterone antagonist, onapristone have  been shown to reduce PMP22 levels to a more normal expression in laboratory models of CMT1A. However, neither has been validated in human clinical trials yet.  Ongoing vitamin C trials in the United States are funded by the CMTA in partnership with the MDA. Unfortunately, the progesterone antagonist (onapristone) used to treat CMT1A in laboratory models isn’t suitable because of its toxicity in humans.

Using “high-throughput screening,” a method utilizing robots and cultured Schwann cells, the CMTA, partnering with the NIH and pharmaceutical companies, have tested 350,000 compounds. With this method, potential candidate medicines that lower PMP22 levels have been discovered.

Next these candidate compounds will be validated, through more stringent tests at a level closest to human testing.  Then the next step is to study how the human PMP gene is “turned on” and causes an overexpression of that specific protein in CMT1A.

The CMTA hopes to find potential treatments arising from these trials within 3 to 5 years. Additionally they are studying new strategies for treating other forms of CMT including 1X, and Types 2 and 4.

Resource: Retrieved 12/30/2010


parents with daughterWednesday, April 20, 2011 9:10 AM | CMTA

The CMTA has received an anonymous donation in the amount of $100,000 in support of the STAR program.  The incredibly generous donation has compelled the CMTA’s Board of Directors to issue a new Challenge Grant.  The Board of Directors will match all funds raised between now and May 31, 2011 up to $100,000 — which gives us the chance to turn the initial $100,000 into $300,000!  Please take this great opportunity to help advance the STAR program — your support is greatly appreciated!!

Please click the following link to donate to the STAR program:

Thank you!

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A novel LRSAM1 mutation is associated with autosomal dominant axonal Charcot-Marie-Tooth disease

Charcot-Marie-Tooth (CMT) disease is the most common hereditary neuropathy resulting from mutations in >3 genes expressed in either the Schwann cells or the axon of peripheral nerves. The disease is classified into demyelinating (CMT1), axonal (CMT2) or intermediate (CMTI) based on electrophysiological and pathological findings.




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