Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Cancer drugs to treat birth defects

Nature Genetics volume 39, pages 1057–1059 (2007)Cite this article

Identical mutations of the same genes can lead either to congenital malformations or to cancer, depending on their cellular and temporal context. The demonstration of activated RAS-ERK signaling in a mouse model of Apert syndrome suggests that drugs designed to inhibit this pathway in cancer may also delay the progression of several serious pediatric syndromes.

Until a few years ago, the title above might have seemed to belong only in the headlines of the tabloid press. However, as the genes mutated in birth defects and cancer have been identified, and the details of how these mutations disturb the regulation of biochemical pathways have been explained, a remarkable convergence in their underlying cellular mechanisms has been uncovered. This is well illustrated in a study by Vivek Shukla, Xavier Coumoul and colleagues1 on page 1145 of this issue. In a mouse model of Apert syndrome in which affected pups normally die within a few weeks with craniofacial malformations, injection of the pregnant mother with a specific signaling inhibitor enables the mutant offspring to survive and even reproduce. Thus, the huge investments into the design of new anticancer drugs might have collateral benefits in the treatment of rare congenital malformations.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

Buy

All prices are NET prices.

Figure 1: FGFR2 activation and the RAS-ERK pathway.

Katie Ris-Vicari

References

  1. Shukla, V., Coumoul, X., Wang, R.H., Kim, H.-S. & Deng, C.-X. Nat. Genet. 39, 1145–1150 (2007).

    Article  CAS  Google Scholar 

  2. Wilkie, A.O.M. et al. Nat. Genet. 9, 165–172 (1995).

    Article  CAS  Google Scholar 

  3. Ibrahimi, O.A. et al. Hum. Mol. Genet. 13, 2313–2324 (2004).

    Article  CAS  Google Scholar 

  4. Pollock, P.M. et al. Oncogene advance online publication 21 May 2007 (doi: 10.1038/sj.onc.1210529).

    Article  CAS  Google Scholar 

  5. Chen, L., Li, D., Li, C., Engel, A. & Deng, C-X. Bone 33, 169–178 (2003).

    Article  CAS  Google Scholar 

  6. Wang, Y. et al. Development 132, 3537–3548 (2005).

    Article  CAS  Google Scholar 

  7. Kim, D.H. & Rossi, J.J. Nat. Rev. Genet. 8, 173–184 (2007).

    Article  CAS  Google Scholar 

  8. Dailey, L., Ambrosetti, D., Mansukhani, A. & Basilico, C. Cytokine Growth Factor Rev. 16, 233–247 (2005).

    Article  CAS  Google Scholar 

  9. Wilkie, A.O.M. Cytokine Growth Factor Rev. 16, 187–203 (2005).

    Article  CAS  Google Scholar 

  10. Eswarakumar, V.P. et al. Proc. Natl. Acad. Sci. USA 103, 18603–18608 (2006).

    Article  CAS  Google Scholar 

  11. Perlyn, C.A., Morriss-Kay, G., Darvann, T., Tenenbaum, M. & Ornitz, D.M. Neurosurgery 59, 210–215 (2006).

    Article  Google Scholar 

  12. Ohren, J.F. et al. Nat. Struct. Mol. Biol. 11, 1192–1197 (2004).

    Article  CAS  Google Scholar 

  13. Kohno, M. & Pouyssegur, J. Ann. Med. 38, 200–211 (2006).

    Article  CAS  Google Scholar 

  14. Schubbert, S., Shannon, K. & Bollag, G. Nat. Rev. Cancer 7, 295–308 (2007).

    Article  CAS  Google Scholar 

  15. Solit, D.B. et al. Nature 439, 358–362 (2006).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Andrew O.M. Wilkie is at the Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK. awilkie@hammer.imm.ox.ac.uk,

    Andrew O M Wilkie

Authors
  1. Andrew O M Wilkie
    View author publications

    You can also search for this author in PubMed Google Scholar

Ethics declarations

Competing interests

The author declares no competing financial interests.

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Wilkie, A. Cancer drugs to treat birth defects. Nat Genet 39, 1057–1059 (2007). https://doi.org/10.1038/ng0907-1057

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/ng0907-1057

This article is cited by

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing