Initiative Ignites Call to Action for Support on Worldwide Research Project to Deliver First Treatments for Most Commonly Inherited Peripheral Neuropathy
GLENOLDEN, Pa., Jan. 23, 2013 /PRNewswire-USNewswire/ -- The national non-profit Charcot-Marie-Tooth Association announced today that it has launched STAR Pathways, a research protocol designed to deliver the first treatments for the most commonly inherited peripheral neuropathy in the world, Charcot-Marie-Tooth (CMT). STAR Pathways, is a critical milestone within the CMTA's STAR (Strategy to Accelerate Research) Initiative, which fosters collaboration of an international consortium of leading peripheral neuropathy researchers and clinicians who are working to find treatments for the estimated 2.6 million people worldwide that are affected by CMT.
STAR Pathways will ignite a call to action for support of STAR, which has recently reached major research milestones, by identifying the sequential steps necessary for drug delivery for treatments of CMT. Over the next two years, the CMTA will raise $25 million to sponsor continued scientific research specifically targeting CMT, type 1A, the most common form of CMT. Types 2A and 2E are also being examined by the same research protocol.
The CMTA launched STAR to dramatically speed up the pace of CMT research. The opportunities associated with STAR stem from the identification of 51 specific gene defects opening an extraordinary window of opportunity to develop treatments and cures for CMT in the immediate and foreseeable future. STAR's unique character stems from the willingness of the scientists to come together to advance CMT research as a team, sharing and communicating ideas, discoveries and research findings. Leveraging and promoting this unique expertise in a collaborative manner is what makes STAR unique.
STAR reached noteworthy success in 2012 when potential treatment options, many of which capitalized on scientific breakthroughs in genetics, saw significant progression. This progression led to STAR Pathways, the integration of a directed protocol to determine effective treatments for those with CMT through the following steps: building cell lines for testing purposes, creating an animal model through which drugs may be tested, putting cell lines through intense screening at the National Institute of Health (NIH) to diagnose the impact on the disease, testing compounds that are effective at regulating CMT during a pre-clinical phase, investigating the safety of newly established medication and two phases of human trials.
All of these steps, when combined, will work to provide in-depth findings that can be applied toward treatment, and eventually a cure, for CMT. Currently, the STAR Initiative has successfully delivered scientific results in the first three of the aforementioned research phases. STAR Pathways will be working to deliver the final four steps through the collection goal of $25 million, to come through charitable donations.
For more information, visit www.cmtausa.org, or become a fan of the official "Charcot-Marie-Tooth Association: The Time is Now" Facebook Fan page (www.facebook.com/CMTAssociation) or call 1-800-606-2682.
About the Charcot-Marie-Tooth Association (CMTA):
The CMTA, headquartered in Glenolden, Pennsylvania, is a 501(C)(3) nonprofit organization founded in 1983. The mission of the CMTA is to support the development of new treatment for CMT, to improve the quality of life for people with CMT and, ultimately, to find a cure. The organization is led by a Chief Executive Officer and governed by a voluntary Board of Directors, and an international Medical Advisory Board and The CMTA Scientific Advisory Boards are comprised of senior scientists with extensive research experience related to CMT. The CMTA has more than 20,000 patients and families, supportive friends, and medical professionals in its member database. For more information, visit www.cmtausa.org.
1800-606-2682 x 108
SOURCE Charcot-Marie-Tooth Association