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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Bezafibrate for X-linked adrenoleukodystrophy.

Marc Engelen1, Luc Tran, Rob Ofman

  • 1Department of Neurology, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.

Plos One
|August 23, 2012
PubMed
Summary
This summary is machine-generated.

Bezafibrate (BF) did not lower very-long-chain fatty acids (VLCFA) in X-linked adrenoleukodystrophy (X-ALD) patients. Further research is needed for effective ELOVL1 inhibitors to treat X-ALD, a condition with no current cure.

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Published on: May 24, 2016

Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • X-linked adrenoleukodystrophy (X-ALD) is a genetic disorder caused by ABCD1 gene mutations, leading to very-long-chain fatty acid (VLCFA) accumulation.
  • Adrenomyeloneuropathy (AMN) is the most common adult manifestation of X-ALD, characterized by progressive myelopathy and lacking curative treatments.

Purpose of the Study:

  • To evaluate the efficacy of bezafibrate (BF) in reducing VLCFA levels in plasma and lymphocytes of X-ALD patients.
  • To assess the safety and tolerability of BF treatment in AMN patients.

Main Methods:

  • A proof-of-principle clinical trial involving ten male AMN patients treated with BF (400 mg/day for 12 weeks, then 800 mg/day for 12 weeks).
  • Regular monitoring of side effects and analysis of plasma and lymphocyte VLCFA levels every four weeks.
  • Assessment of patient adherence through triglyceride level monitoring.

Main Results:

  • Bezafibrate treatment did not result in a significant reduction of VLCFA levels in either plasma or lymphocytes.
  • Patient adherence was confirmed by a notable decrease in triglyceride levels.
  • Plasma bezafibrate levels did not exceed 25 µmol/L, suggesting potential dose-related efficacy issues.

Conclusions:

  • Bezafibrate, at the tested doses, is ineffective in lowering VLCFA levels in X-ALD patients.
  • The limited efficacy may be attributed to the low plasma concentrations of bezafibrate achieved.
  • Future research should focus on developing highly specific ELOVL1 inhibitors that are effective at lower concentrations and well-tolerated.