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Publication and contact information


Brain cancer

Activin receptor-like kinase 2 (ALK2; ACVR1); SMAD family member 1 (MADH1; SMAD1); inhibitor of DNA binding 1 (ID1)

Three studies in primary tumors and cell culture suggest inhibiting ALK2 could help treat a subset of pediatric brain tumors. Whole-genome sequencing of diffuse intrinsic pontine gliomas and midline high-grade astrocytomas identified ALK2 mutations in 19 of 101 tumors. In human astrocyte cell lines, the mutant ALK2 increased cell growth, levels of activated SMAD1 and expression of ID1 compared with wild-type ALK2. In cultures of primary diffuse intrinsic pontine glioma cells, a research compound that inhibited ALK2 blocked proliferation at low micromolar to high nanomolar GI50 values. Ongoing work includes additional in vitro and in vivo studies of mutant ALK2 function and optimization of the ALK2 inhibitor to treat diffuse intrinsic pontine glioma.

SciBX 7(19); doi:10.1038/scibx.2014.551
Published online May 15, 2014

For findings from first study, ALK2 inhibitor patented by Harvard University; unlicensed

Patent and licensing status unavailable for findings from second study

Findings from third study unpatented; unlicensed

Taylor, K.R. et al. Nat. Genet.; published online April 6, 2014;
Contact: Chris Jones, The Institute of Cancer Research, Sutton, U.K.

Buczkowicz, P. et al. Nat. Genet.; published online April 6, 2014;
Contact: Cynthia Hawkins, The Hospital for Sick Children, Toronto, Ontario, Canada

Fontebasso, A.M. et al. Nat. Genet.; published online April 6, 2014;
Contact: Nada Jabado, McGill University, Montreal, Quebec, Canada