Licensing status

Publication and contact information


Spinal muscular atrophy

Calcitonin gene-related peptide (CGRP)

In vitro and mouse studies suggest reducing CGRP expression could help treat spinal and bulbar muscular atrophy (SBMA). In human neuroblastoma cells and motor neurons expressing disease-causing glutamine CAG repeats in androgen receptor, small interfering RNA against or inhibition of CGRP decreased cytotoxicity and increased cell viability compared with control siRNA or no inhibition. In a mouse model of SBMA, reduction of Cgrp expression, using the serotonin (5-HT1D) receptor agonist Amerge naratriptan, increased motor function and lifespan compared with vehicle control. Next steps include Phase II trials of Amerge in the indication.
GlaxoSmithKline plc markets Amerge to treat migraine.
At least four companies have antibodies or antagonists targeting the CGRP receptor in clinical and preclinical testing to treat migraine (see Repurposing naratriptain, page 8).

SciBX 5(40); doi:10.1038/scibx.2012.1056
Published online Oct. 11, 2012

Patent application filed; unavailable for licensing

Minamiyama, M. et al. Nat. Med.; published online Sept. 30, 2012;
Contact: Gen Sobue, Nagoya University, Nagoya, Japan

Contact: Masahisa Katsuno,
same affiliation as above