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



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.



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.



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.
a Ken & Ruth Davee Department of Neurology , Feinberg School of Medicine, Northwestern University , Chicago , IL , USA.
Robert L
RL
Sufit
a Ken & Ruth Davee Department of Neurology , Feinberg School of Medicine, Northwestern University , Chicago , IL , USA.
Senda
S
Ajroud-Driss
a Ken & Ruth Davee Department of Neurology , Feinberg School of Medicine, Northwestern University , Chicago , IL , USA.
Patricia
P
Casey
a Ken & Ruth Davee Department of Neurology , Feinberg School of Medicine, Northwestern University , Chicago , IL , USA.
John A
JA
Kessler
D000328:Adult; D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D005260:Female; D015316:Genetic Therapy / Q000009:adverse effects*; D017228:Hepatocyte Growth Factor / Q000009:adverse effects* / Q000235:genetics / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006801:Humans; D007273:Injections, Intramuscular; D008297:Male; D008875:Middle Aged; D053580:Muscle Strength; D018482:Muscle, Skeletal / Q000473:pathology; D010957:Plasmids; D016896:Treatment Outcome; D014797:Vital Capacity; D055815:Young Adult
C066855:HGF protein, human; D017228:Hepatocyte Growth Factor
Amyotrophic lateral sclerosis; VM202 DNA plasmid; gene therapy; hepatocyte growth factor