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This week in therapeutics Indication

Target/marker/ pathway

Summary

Licensing status

Crystallization studies suggest that the small molecule chaperone Alda-1 could activate mutant forms of ALDH2 to help treat alcohol-induced flushing syndrome. Crystal structure analysis showed that Alda-1, which activates both the wild-type and mutant forms of ALDH2, bound at a site distinct from the enzyme’s active site. Once activated, ALDH2 metabolized ethanol, lowering systemic levels of the compound and reducing flush. Next steps include identifying indications for which Alda-1 and similar small molecule chaperones might be effective and testing those compounds in animals.

Patent application filed; available for licensing through Stanford University’s Office of Technology Licensing

Publication and contact information

Other Flushing syndrome

Aldehyde dehydrogenase 2 family mitochondrial (ALDH2)

Perez-Miller, S. et al. Nat. Struct. Mol. Biol.; published online Jan. 10, 2010; doi:10.1038/nsmb.1737 Contact: Thomas D. Hurley, Indiana University School of Medicine, Indianapolis, Ind. e-mail: [email protected]

SciBX 3(4); doi:10.1038/scibx.2010.127 Published online Jan. 28, 2010

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