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Possible new approach to treating a life-threatening blood disorder

Date:
January 25, 2011
Source:
Journal of Clinical Investigation
Summary:
Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease of the blood system. Current treatments are protracted and associated with complications. However, new research in mice suggests that the drug NAC, which is FDA approved as a treatment for chronic obstructive lung disease and as an antidote for toxicity due to acetaminophen, might provide a rapid and effective treatment for patients with TTP.

Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease of the blood system. The condition is caused by the presence of ultralarge multimers of the protein von Willebrand factor, which promote the formation of blood clots (thrombi) in small blood vessels throughout the body. Current treatments are protracted and associated with complications.

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However, a team of researchers, led by José López, at the Puget Sound Blood Center, Seattle, has generated data in mice that suggest that the drug N-acetylcysteine (NAC), which is FDA approved as a treatment for chronic obstructive lung disease and as an antidote for toxicity due to acetaminophen (paracetamol), might provide a rapid and effective treatment for patients with TTP through its ability to decrease the size of von Willebrand factor multimers.

In an accompanying commentary, Michael Berndt and Robert Andrews, concur with the conclusions of López and colleagues, although they caution that there are a number of caveats to the view that NAC could be used to treat patents with TTP.

The research appears in the Journal of Clinical Investigation.


Story Source:

The above story is based on materials provided by Journal of Clinical Investigation. Note: Materials may be edited for content and length.


Journal References:

  1. Junmei Chen, Adili Reheman, Francisca C. Gushiken, Leticia Nolasco, Xiaoyun Fu, Joel L. Moake, Heyu Ni, José A. López. N-acetylcysteine reduces the size and activity of von Willebrand factor in human plasma and mice. Journal of Clinical Investigation, 2011; DOI: 10.1172/JCI41062
  2. Michael C. Berndt, Robert K. Andrews. Thrombotic thrombocytopenic purpura: reducing the risk? Journal of Clinical Investigation, 2011; DOI: 10.1172/JCI46091

Cite This Page:

Journal of Clinical Investigation. "Possible new approach to treating a life-threatening blood disorder." ScienceDaily. ScienceDaily, 25 January 2011. <www.sciencedaily.com/releases/2011/01/110125123229.htm>.
Journal of Clinical Investigation. (2011, January 25). Possible new approach to treating a life-threatening blood disorder. ScienceDaily. Retrieved November 28, 2014 from www.sciencedaily.com/releases/2011/01/110125123229.htm
Journal of Clinical Investigation. "Possible new approach to treating a life-threatening blood disorder." ScienceDaily. www.sciencedaily.com/releases/2011/01/110125123229.htm (accessed November 28, 2014).

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