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Advanced pharmacokinetics and pharmacodynamics for phase Ib/IIa trial of repurposed enoxacin therapy for patients with ALS

Principal Investigator:
  • Angela Genge, McGill University
Team Members:
  • Eran Hornstein, Weizmann Institute of Science
  • Iddo Magen, Weizmann Institute of Science

Project Overview

A Phase 1/2 clinical trial led by Dr. Angela Genge and co-recipient Dr. Eran Hornstein is currently underway to test the safety and tolerability of enoxacin in people living with ALS. Enoxacin is an antibiotic that on a cellular level is known to activate an enzyme called DICER, which in turn can increase the levels of specific substances called microRNA (miRNA), both of which have been shown to be reduced in people living with ALS.

With this grant, the team will conduct more in-depth analyses to further assess the value of enoxacin in ALS. Researchers will measure the ability of enoxacin to reduce miRNA levels in Cerebrospinal fluid and blood. They will also assess the levels of neurofilament light (NF-L), currently considered the best marker of neurodegeneration.

Furthermore, it has been determined that enoxacin inhibits the enzyme that breaks down riluzole in the body. Since participants in the trial will be allowed to take riluzole, this study will do the important work of understanding how riluzole dosing should be modified for participants taking enoxacin to prevent undesired side effects.

Results from this study will not only provide evidence around a potential therapy for patients with ALS but will also leverage the existing fully funded clinical trial by adding value to the study data, to ensure a clear evaluation of the effects of enoxacin in ALS.