Researchers have found that a newly developed drug, which is aimed at a particular receptor involved in the development of blood vessels that sustain tumour growth, is active in patients with advanced cancers and, in some cases, has halted the progress of the disease. The drug, ACE-041, targets a different molecular pathway to other anti-angiogenesis drugs and may provide a new option to treat cancer.
Results from a phase I clinical study of ACE-041 were presented at the 22nd EORTC-NCI-AACR  Symposium on Molecular Targets and Cancer Therapeutics in Berlin today (Friday). The drug targets a receptor known as activin receptor-like kinase-1 (ALK-1), which regulates the formation of new networks of blood vessels needed for tumour growth a process known as angiogenesis. While existing anti-angiogenic drugs such as bevacizumab, sunitinib and sorafenib target other angiogenesis receptors such as VEGF, ACE-041 is one of the first to target the ALK-1 pathway.
Professor Sunil Sharma, the Jon and Karen Huntsman Presidential Professor of Cancer Research at the Huntsman Cancer Institute, University of Utah, Salt Lake City (USA), told the meeting that the connection of ALK-1 with angiogenesis was made with the discovery that mutations in the ALK-1 gene caused a condition known as hereditary haemorrhagic telangiectasia 2 (HHT2), which is characterised by impaired formation of capillary beds and causes red markings on the skin.
Acceleron Pharma, a biotechnology company in Cambridge, Massachusetts (USA), designed ACE-041 to inhibit ALK-1 signalling and asked Prof Sharma to be one of the investigators to conduct the first-in-man phase I clinical trial of the drug to see if it would inhibit tumour angiogenesis.
"Since ALK-1 is only transiently expressed on proliferating endothelial cells (the cells that line the inner surface of blood vessels), in contrast to the VEGF receptors which are constitutively expressed on endothelial and other c
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ECCO-the European CanCer Organisation