This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Amyotrophic lateral sclerosis (ALS)

Matrix metalloproteinase 9 (MMP9)

Mouse studies suggest inhibiting MMP9 could help treat ALS. In patients with ALS and in mouse models, slow motor neurons and neurons regulating eye movement and sexual function were resistant to neurodegeneration. In mouse models of ALS, levels of Mmp9 in these neurons were lower than those in fast motor neurons lost during disease progression. In the models, genetic inactivation of Mmp9 or intracerebroventricular administration of a research-grade MMP9 inhibitor delayed the onset of disease symptoms, decreased loss of motor functions and increased survival compared with unmodified Mmp9 expression or vehicle. Ongoing work includes confirming the proneurodegenerative role of MMP9 in patients with ALS.

SciBX 7(6); doi:10.1038/scibx.2014.178
Published online Feb. 13, 2014

Unpatented; unlicensed

Kaplan, A. et al. Neuron;
published online Jan. 22, 2014;
doi:10.1016/j.neuron.2013.12.009
Contact: Christopher E. Henderson, Columbia University Medical Center, New York, N.Y.
e-mail:
ch2331@columbia.edu