Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Infectious disease

HIV/AIDS

Caspase-1 (CASP1)

Human tissue culture studies suggest CASP1 inhibitors could help treat HIV infection. In cultures of normal human lymphoid tissue, HIV predominantly infected quiescent CD4+ T cells, upregulated CASP1 and led to CASP1-activated pyroptosis, which was not observed in HIV-infected, activated CD4+ T cells. Also in the HIV-infected tissue cultures, CASP1-targeting shRNA or the small molecule CASP1 inhibitor VX-765 decreased CASP1 levels and the number of pyroptotic CD4+ T cells compared with scrambled shRNA or no treatment. Ongoing work in collaboration with Vertex Pharmaceuticals Inc. may include a Phase II trial of the company's VX-765 in combination with antiretroviral therapies in patients with HIV infection.
VX-765 is in Phase II testing to treat epilepsy.

SciBX 7(3); doi:10.1038/scibx.2014.90
Published online Jan. 23, 2014

Patent application filed by the Gladstone Institutes; licensing status undisclosed

Doitsh, G. et al. Nature;
published online Dec. 19, 2013;
doi:10.1038/nature12940
Contact: Warner C. Greene, Gladstone Institute of Virology and Immunology, San Francisco, Calif.
e-mail:
wgreene@gladstone.ucsf.edu