Approach

Summary

Licensing status

Publication and contact information

Disease models

Induced pluripotent stem (iPS) cell-derived motor neurons from patients with familial amyotrophic lateral sclerosis (ALS) for in vitro therapeutic screening

In vitro studies suggest iPS cell-derived motor neurons from patients with familial ALS could be used to screen for new therapeutics. In cultured iPS cell-derived motor neurons from patients with familial ALS carrying TAR DNA binding protein 43
(TDP-43; TARDBP) mutations, levels of TDP-43 protein and cytosolic aggregates were greater than those in iPS cell-derived motor neurons from healthy controls. In the ALS patient-derived motor neurons, the histone acetyltransferase inhibitor anacardic acid reversed the disease phenotype. Next steps include testing the safety and efficacy of anacardic acid in additional cellular and animal models.

SciBX 5(33); doi:10.1038/scibx.2012.881
Published online Aug. 23, 2012

Patent application filed covering prophylactic and therapeutic applications of anacardic acid; provisional application filed covering screening applications; available for licensing

Egawa, N. et al. Sci. Transl. Med.;

published online Aug. 1, 2012;
doi:10.1126/scitranslmed.3004052
Contact: Haruhisa Inoue, Japan Science and Technology Agency, Tokyo, Japan
e-mail:
haruhisa@cira.kyoto-u.ac.jp