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Targeting Molecular ‘Kingpin’ May Offer Promise for Brain Tumors

A new report* shows promising data from a new type of drug for treatment of glioblastoma multiforme (GBM), a type of brain tumor that has a very poor prognosis because it is aggresive and often becomes resistant to treatment. GBM occurs in a small number of cases of NF1 as well as in the general population.  In a new research collaboration with the biotech company NexGenix Pharmaceuticals and headed by a group at Tufts University, the drug NXD30001 proves to be a strong and sustained inhibitor of tumor growth in a mouse model of GBM. NXD30001 is one of a new class of dugs that inhibit heat shock protein 90 (hsp90) an intracellular ‘molecular chaperone’ and now an emerging kingpin in promoting cancer cell growth. Because hsp90 plays such a central role in the function of many cell signaling events,  inhibiting hsp90 also inhibits a cascade of other important cancer-promoting cell targets such as the EGFR-PI3K-Akt axis.  In the mice chronic (continuous long term) treatment seems to be well tolerated.

Though several companies are developing hsp90 inhibitors, the Nexgenix drug is reported to have superior biology that makes it more effective in crossing the blood brain barrier, an important asset for any drug that is to function in the brain. NexGenix was established a few years ago by one of CTF’s founders Dr. Allan Rubenstein and NXD30001 is one of their front runner drugs. It is worth noting that CTF has also funded Drug Discovery Initiative research at the House Ear Institute in Los Angeles to test NXD30001 in mouse models of NF2-related tumors, with some promising results.
This data is of course very early stage, and there is still a long path to bring this to the clinic and assessing if it will have the same positive effects in humans with GBM as it does in the mice. We look forward to further reports on the advancement of hsp90 inhibitors to the clinic.


Zhu HWoolfenden SBronson RTJaffer ZBarluenga SWinssinger NRubenstein AEChen RCharest A. (2010) The novel Hsp90 inhibitor NXD30001 induces tumor regression in a genetically engineered mouse model of glioblastoma multiforme. Mol Cancer Ther. 2010 Jul 19. [Epub ahead of print]