Andy, The implications of the upcoming Science article are huge. Most of Biotechnology's therapeutic products are polypeptide hormones (interferons, interleukins, and growth factors). The products are expensive to manufacture, are taken via injection, and are difficult to modify. These hormones bind to a receptor binding domain (ONTAK sends a toxin to the IL-2 hormone binding domain, Herceptin is an antibody that binds to the HER-2/neu binding domain, etc) that is on the surface of the cell. This initiates a signal transduction pathway that involves various intermediates, culminating with transcription factors that alter gene expression.
LGND's STAT technology screens small molecules that would interact with these transcription factors (or associated proteins) to mimic the effect of the polypeptide hormone.
The upcoming Science paper (all of this now is an assumption on my part based on what I learned about the Lake Tahoe presentation earlier this year) describes a small molecule that interacts with the hormone receptor itself (on the outside of the cell) and mimics the polypeptide hormone with extreme specificity (reacts not only exclusively with the G-CSF receptor, but distinguishes between the mouse G-CSF receptor and the human G-CSF receptor).
In the past, most did not think that this approach was even feasible. LGND's Science paper will show that the approach works, paving the way for screening analogs of G-CSF as well as the myriad of other interleukins (Neupogen, Epogen, Aldesleukin, etc.), interferons (Betaseron, Avonex, etc) and growth factors (Myotropin, etc).
The approach allows for screening of anatgonists as well as agonists and the compounds can be made and modified very cheaply. Thus, the various established Biotech products are fair targets, as are many of the current products under development.
LGND of course has filed EXTREMELY broad patents and I'm sure that LGND and SBH scientist are very happy and excited. |
