Just to recap: Phylogica was targeting breast cancer but is now...

Just to recap: Phylogica was targeting breast cancer but is now targeting rare blood malignancies; Soucek was targeting lung cancer but has now been awarded a grant to develop a Omomyc treatment for glioblastoma which is a brain tumor. Undoubtedly both parties have made strategic decisions in the choice of targets based upon the commercial and scientific merits of conducting research in rare or orphan diseases.

One can readily understand the commercial merit into research within rare diseases but what about the scientific merit given that MYC is deregulated in most human tumors including the breast, lung, colon, cervix, pancreas, stomach and the brain.

I have suggested that Braiteh and Kurzrock may provide clues within their hypothesis on rare cancers leading to exceptional therapies. If a cancer is driven by a single aberrant mechanism then a therapy which effectively targets this aberration will have a significant effect on the majority of patients. Is this the case for Acute Myeloid Leukaemia, Chronic Myeloid Leukaemia and Multiple Myeloma as well as glioblastoma but not for breast and lung cancers? I suspect so.

In short, it would appear that targeting rare or orphan cancers is a well conceived strategy. If effective MYC therapies can be developed for blood and brain cancers then the door is wide open to assist in the development of therapies for the majority of common cancers in which MYC is deregulated. In the case of common cancers, however, the mutant gene MYC may be only one of a number of distinct molecular defects; precisely the reason why common cancers are not a good place to develop a proof-in-principle.