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.

  • Review
  • Published:

New treatments for serious conditions: ethical implications

Gene Therapy volume 24, pages 534–538 (2017)Cite this article

Subjects

Abstract

Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels. The good news that Spinraza brings thus requires additional attention to its ethical and policy implications, to improve counseling and shared decision-making about treatment and research options for patients and all involved in their care.

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

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Similar content being viewed by others

References

  1. Gavrilov DK, Shi X, Das K, Gilliam TC, Wang CH . Differential SMN2 expression associated with SMA severity. Nat Genet 1998; 20: 230–231.

    Article  CAS  Google Scholar 

  2. Russman BS . Spinal muscular atrophy: clinical classification and disease heterogeneity. J Child Neurol 2007; 22: 946–951.

    Article  Google Scholar 

  3. Thomas NH, Dubowitz V . The natural history of type 1 (severe) spinal muscular atrophy. Neuromuscul Disord 1994; 4: 497–502.

    Article  CAS  Google Scholar 

  4. Kolb SJ, Kissle JT . Spinal muscular atrophy. Neurol Clin 2015; 33: 831–846.

    Article  Google Scholar 

  5. Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Hontes J, De Vivo DC et al. Results from a phase 1 study of nusinersen (ISIS-SMN Rx) in children with spinal muscular atrophy. Neurology 2016; 86: 890–897.

    Article  CAS  Google Scholar 

  6. Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet 2016; 388: 3017–3026.

    Article  CAS  Google Scholar 

  7. Biogen Press Release, 2016. US. FDA approves Biogen’s Spinraza (nusinersen), the first treatment for spinal muscular atrophy. Available at: http://media.biogen.com/press-release/neurodegenerative-diseases/us-fda-approves-biogens-spinraza-nusinersen-first-treatment (accessed on 4 February 2017).

  8. FDA News Release, 2016. FDA approves first drug for spinal muscular atrophy. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm (accessed on 4 February 2017).

  9. Hache M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, Xia S et al. Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J Child Neurol 2016; 3: 899–906.

    Article  Google Scholar 

  10. Gawande A. Complications: A Surgeon’s Notes on an Imperfect Science. Picador: New York, NY, USA, 2002..

  11. Arkin LM, Sondhi D, Worgall S, Suh LHK, Hackett NR, Kaminsky SM et al. Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders. Hum Gene Ther 2005; 16: 1028–1036.

    Article  CAS  Google Scholar 

  12. McCoy MS, Carniol M, Chockley K, Urwin JW, Emanuel EJ, Schmidt H . Conflicts of interest for patient-advocacy organizations. N Eng J Med 2017; 376: 880–885.

    Article  Google Scholar 

  13. Tribble SJ. Drugmakers are turning patients with rare diseases into D.C. lobbyists, Kaiser Health News, 10 April 2017. Available at: http://khn.org/news/drugmakers-help-turn-patients-with-rare-diseases-into-d-c-lobbyists/ (accessed on 15 April 2017).

  14. Richtel M. Therapies that can attack cancer, and organs. New York Times 4 December 2016, pp A1..

  15. Shobit S. Biogen, Ionis under fire for Spinraza. Investopedia, 4 January 2017. Available at: http://www.investopedia.com/news/biogen-ionis-under-fire-spinraza-biib-ions/?ad=dirN&qo=investopediaSiteSearch&qsrc=0&o=40186 (accessed on 5 February 2017).

  16. Thomas K. Hefty price set on a new drug that can stunt muscle disease. New York Times 31 December 2016, pp B1..

  17. Biogen, SMA 360. Available at: https://www.spinraza.com/en_us/home/patient-support-services/biogen-support-program.html (accessed on 5 February 2017).

  18. Genetic Information and Nondiscrimination Act, 2008. Pub L No 110-233, 122 Stat 881..

  19. US Department of Health and Human Services, 2009. 'GINA' the Genetic Information Nondiscrimination Act of 2008: information for researchers and health care professionals. Available at: https://www.genome.gov/pages/policyethics/geneticdiscrimination/ginainfodoc.pdf (accessed on 5 February 2017).

  20. Thomas L. The technology of medicine. In: Lives of a Cell: Notes of a Biology Watcher. Viking Press: New York, NY, USA, 1974, pp 31–36..

  21. Lawton S, Hickerton C, Archibald AD, McClaren BJ, Metcalfe SA . A mixed methods exploration of families’ experiences of the diagnosis of childhood spinal muscular atrophy. Eur J Hum Genet 2015; 23: 575–580.

    Article  Google Scholar 

  22. Feudtner C. The want of control: ideas and ideals in the management of diabetes. In: Bittersweet: Diabetes, Insulin, and the Transformation of Illness. UNC Press: Chapel Hill, NC, USA, 2003, pp 121–145..

  23. Baker HB, McQuilling JP, King NMP . Ethical considerations in tissue engineering research: case studies in translation. Methods 2016; 99: 135–144.

    Article  CAS  Google Scholar 

  24. Churchill LR, Collins ML, King NMP, Pemberton S, Wailoo K . Genetic research as therapy: implications of 'gene therapy' for informed consent. J Law Med Ethics 1998; 26: 38–47.

    Article  CAS  Google Scholar 

  25. Grady C . Enduring and emerging challenges of informed consent. N Engl J Med 2015; 372: 855–862.

    Article  CAS  Google Scholar 

Download references

Acknowledgements

This work was supported in part by the National Center for Advancing Translational Sciences of the National Institutes of Health under award number U01TR001792.

Disclaimer

The content is solely the responsibility of the authors.

Author information

Authors and Affiliations

  1. Department of Social Sciences and Health Policy, Wake Forest School of Medicine, Center for Bioethics Health, and Society and Graduate Program in Bioethics, Wake Forest University, Winston-Salem, NC, USA

    N M P King

  2. Department of Pediatrics, Wake Forest School of Medicine, Winston-Salem, NC, USA

    C E Bishop

Authors
  1. N M P King
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. C E Bishop
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to N M P King.

Ethics declarations

Competing interests

The authors declare no conflict of interest.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

King, N., Bishop, C. New treatments for serious conditions: ethical implications. Gene Ther 24, 534–538 (2017). https://doi.org/10.1038/gt.2017.32

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/gt.2017.32

This article is cited by

Search

Advanced search

Quick links