This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Cancer

Neurofibromatosis

Neurofibromin 1 (NF1); MEK

Two studies in mice suggest MEK inhibitors could help treat cancers with mutations in NF1. In a mouse model of neurofibromatosis type 1, a familial cancer in which mutations in NF1 increase the risk of juvenile myelomonocytic leukemia, the MEK inhibitor PD-0325901 decreased blood leukocyte counts and spleen size and increased survival compared with vehicle. In the second study, PD-0325901 decreased the size of neurofibromas and increased survival compared with vehicle in a patient xenograft mouse model of neurofibromatosis type 1. Next steps could include clinical trials of MEK inhibitors in patients with neurofibrosis type 1.
Pfizer Inc.'s combination of PD-0325901 and PF-04691502 is in Phase I testing in cancer.
At least seven other companies have MEK inhibitors in clinical testing for various cancers.

SciBX 6(1); doi:10.1038/scibx.2013.9
Published online Jan. 10, 2013

Patent and licensing status unavailable for both papers

Chang, T. et al. J. Clin. Invest.; published online Dec. 10, 2012;
doi:10.1172/JCI63193
Contact: Kevin Shannon, University of California, San Francisco, Calif.
e-mail:
shannonk@peds.ucsf.edu


Jessen, W.J. et al. J. Clin. Invest.; published online Dec. 10, 2012;
doi:10.1172/JCI60578
Contact: Nancy Ratner, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
e-mail:
nancy.ratner@cchmc.org