Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis

Robert L. Sufit*, Senda Ajroud-Driss, Patricia Casey, John A. Kessler

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

15 Scopus citations


Objective: To assess safety and define efficacy measures of hepatocyte growth factor (HGF) DNA plasmid, VM202, administered by intramuscular injections in patients with amyotrophic lateral sclerosis (ALS). Methods: Eighteen participants were treated with VM202 administered in divided doses by injections alternating between the upper and lower limbs on d 0, 7, 14, and 21. Subjects were followed for nine months to evaluate possible adverse events. Functional outcome was assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R) as well as by serially measuring muscle strength, muscle circumference, and forced vital capacity. Results: Seventeen of 18 participants completed the study. All participants tolerated 64 mg of VM202 well with no serious adverse events (SAE) related to the drug. Twelve participants reported 26 mild or moderate injection site reactions. Three participants experienced five SAEs unrelated to VM202. One subject died from respiratory insufficiency secondary to ALS progression. Conclusions: Multiple intramuscular injection of VM202 into the limbs appears safe in ALS subjects. Future trials with retreatment after three months will determine whether VM202 treatment alters the long-term course of ALS.

Original languageEnglish (US)
Pages (from-to)269-278
Number of pages10
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Issue number3-4
StatePublished - Apr 3 2017


  • Amyotrophic lateral sclerosis
  • VM202 DNA plasmid
  • gene therapy
  • hepatocyte growth factor

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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