Can the therapeutic efficacy of tocotrienols in neurodegenerative familial dysautonomia patients be measured clinically?

Antioxid Redox Signal. 2008 Apr;10(4):837-41. doi: 10.1089/ars.2007.1874.


Familial dysautonomia (FD) is an inherited, fatal, neurodegenerative disorder manifested by autonomic/hypertensive crises and cardiac instability. Patients produce little IKAP, the gene product of the affected mutated gene, and have low levels of monoamine oxidase A (MAO A), whose reduced presence appears to result in an increased accumulation of biogenic amines, which is a trigger for hypertensive crises. As ingestion of tocotrienols elevates IKAP and MAO A in FD patients, we examined their impact on the frequency of hypertensive crises and cardiac function. After 3 to 4 months of tocotrienol ingestion, approximately 80% of patients reported a significant (> or = 50%) decrease in the number of crises. In a smaller group of patients, a postexercise increase in heart rate and a decrease in the QT interval were observed in the majority of participants. Based on these findings, we hypothesize that tocotrienol therapy will improve the long-term clinical outlook and survival of individuals with FD.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Carrier Proteins / metabolism
  • Dysautonomia, Familial / drug therapy*
  • Dysautonomia, Familial / metabolism
  • Dysautonomia, Familial / physiopathology
  • Heart Rate / drug effects
  • Humans
  • Monoamine Oxidase / metabolism
  • Tocotrienols / pharmacology
  • Tocotrienols / therapeutic use*
  • Transcriptional Elongation Factors
  • Treatment Outcome


  • Carrier Proteins
  • Elp1 protein, human
  • Tocotrienols
  • Transcriptional Elongation Factors
  • Monoamine Oxidase