This Article Full Text Full Text (PDF) Alert me when this article is cited Alert me if a correction is posted Services Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Citing Articles Citing Articles via HighWire Citing Articles via Google Scholar Google Scholar Articles by Braiteh, F. Articles by Kurzrock, R. Search for Related Content PubMed PubMed Citation Articles by Braiteh, F. Articles by Kurzrock, R.

Editorial/Perspectives

Uncommon tumors and exceptional therapies: paradox or paradigm?

Phase I Program, Division of Cancer Medicine, The University of Texas, M. D. Anderson Cancer Center, and the University of Texas Graduate School of Biomedical Sciences at Houston, Houston, Texas

Requests for reprints: Razelle Kurzrock, Phase I Program, Division of Cancer Medicine, M. D. Anderson Cancer Center, Unit 422, 1515 Holcombe Boulevard, Houston, TX 77030-4009. Phone: 713-794-1226; Fax: 713-745-2374. E-mail: rkurzroc{at}mdanderson.org

Abstract

Why does it seem that, repeatedly, when a new treatment with a striking effect is discovered in the cancer field, it is effective for a very rare cancer type? For example, groundbreaking therapeutic discoveries have been made for extremely uncommon malignancies such as hairy cell leukemia, chronic myelogenous leukemia, seminoma, gastrointestinal stromal tumor, (del)5q myelodysplastic syndrome, and acute promyelocytic leukemia. In contrast, progress in the most common and most intensively studied tumors—lung, breast, prostate, and colon cancer—has been slow and incremental. We hypothesize that the reason for this phenomenon is that the pathophysiologic basis for a tumor being rare is one and the same as the reason that it may ultimately be so treatable. That is, if a cancer can be derived only via a single aberrant molecular genetic aberration, then it should be both rare and easily targeted by a molecular cancer therapeutic approach. If, on the other hand, many distinct pathways can lead to the development of a specific tumor type, it should occur much more commonly and be significantly more difficult to treat. The corollary to our hypothesis is the prediction that new therapies will continue to show their most salutary effects in rare cancers. Furthermore, only by stratifying the common tumors, especially when using targeted agents, into the molecular subsets of diseases that compose them are we likely to achieve a substantial effect in these disorders. [Mol Cancer Ther 2007;6(4):1175–9]

¹ http://rarediseases.info.nih.gov/; accessed on 3-1-2006.

² http://www.fda.gov/cder/cancer/approved.htm; accessed on 3-2-2006.

Received 10/31/06; revised 1/17/07; accepted 2/23/07.

This article has been cited by other articles:

				J. Rodon, V. DeSantos, R. J. Ferry Jr., and R. Kurzrock Early drug development of inhibitors of the insulin-like growth factor-I receptor pathway: Lessons from the first clinical trials Mol. Cancer Ther., September 1, 2008; 7(9): 2575 - 2588. [Abstract] [Full Text] [PDF]

				Y. C. Ip, S. T. Cheung, Y. T. Lee, J. C. Ho, and S. T. Fan Inhibition of hepatocellular carcinoma invasion by suppression of claudin-10 in HLE cells Mol. Cancer Ther., November 1, 2007; 6(11): 2858 - 2867. [Abstract] [Full Text] [PDF]