ALS Research Briefs

TDP43 has unexpectedly extensive role

Scientists have found that the protein TDP43 normally influences the synthesis of more than 1,500 other proteins, including itself. Mutations in the gene for TDP43 are a known cause of ALS, and TDP43 is depleted in the nucleus and enriched in the cytoplasm (main cell compartment) in ALS-affected nerve cells, even when no TDP43 mutations exist. Understanding TDP43's many functions is likely to improve understanding of ALS.

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ALS TDI to focus on TDP43 mouse

The MDA-supported ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., announced March 22, 2011, that it has entered into collaboration with MDA and three other nonprofit organizations to study mice with a mutation in the TDP43 gene. These mice may be a good model of human ALS. The availability of a new ALS mouse model to supplement the widely used SOD1 research mouse is expected to expand understanding of the disease and improve treatment development.


Ceftriaxone trial still open

A phase 3 trial of the drug ceftriaxone remains open to people with ALS who have had symptoms for less than three years. The study is taking place at 57 locations in the United States and Canada. Ceftriaxone, an antibiotic, may reduce levels of potentially toxic glutamate around nerve cells.

See Ceftriaxone ALS Clinical Research Trial or Clinical Trial of Ceftriaxone in Subjects With ALS for more information and a list of study sites. Or, contact Sarah Titus at or (617) 726-1398.




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