Managing Editor's Note: We are proud to share with you that Lee Silsby Compounding Pharmacy in Cleveland, Ohio, is supplying the Leucovorin for this study below. Alan Israel, lead pharmacist, has been a leader in our community to ensure safe, effective biomedical treatments for our children. On a personal note, Lee Silsby was the first pharmacy I ever used for girls' back in 2001 and I've never forgotten their customer care and attention. Thank you, Alan and the staff at LS. KRS
A Folinic acid intervention for ASD
Dr. Richard Frye
is conducting an exciting study on the impact of a daily treatment of folinic
acid on ASD-related symptoms. The hypothesis is that an folinic acid will
improve folate metabolism in the brain, oxidative stress and mitochondrial
function in children with autism, resulting in specific health improvements for
patients with autism, a discovery that would be very welcome by the autism
Compounding Pharmacy has been selected as the sole supplier of Leucovorin
(folinic acid) for the study. Alan Israel, R. Ph., the lead pharmacist at Lee
Silsby, stated that the biomedical community can benefit greatly from more
research and that he is willing to help out as much as possible. To his credit,
Lee Silsby Compounding Pharmacy has agreed to provide folinic acid for both the
double-blind placebo-controlled portion of the trial as well as the open-label
portion of the trial at no cost to the patients in the trial.
Dr. Frye’s current
proposal can potentially provide strong evidence for a folinic acid
intervention and will provide insight into the subgroups of children with ASD
who would benefit from folinic acid. The
primary goal is to improve language for those with ASD (which is measured by
the receptive and expressive CELF language index). Preliminary studies have
suggested that a folinic acid intervention is associated with receptive and
expressive language improvements. For example, in a case-series of 44 children
with ASD and the folate receptor alpha autoantibody, treatment with high-dose folinic
acid (2 mg/kg/day divided twice a day; maximum dose 50mg / day) improved
communication, attention, and stereotypical behavior in many children with ASD.
Folate is an
essential B vitamin required for normal neurodevelopment. Defects in folate metabolism can cause
secondary physiological abnormalities, some of which have been associated with
ASD. In this study, Dr. Frye and his
team aim to study several physiological mechanisms associated with folate abnormalities
in children with ASD: the folate receptor alpha autoantibody that reduce folate
transport across the blood-brain barrier, low glutathione redox status,
mitochondrial dysfunction and genetic polymorphisms. Most importantly, ASD
patients with these physiological mechanisms have shown improvement in ASD
symptoms with a folinic acid intervention.
The goal of the
study is to extend these preliminary findings by documenting response to a folinic
acid intervention in a double-blind placebo-controlled manner and test whether the
folinic receptor alpha autoantibody titers, glutathione status, mitochondrial
function and genetic polymorphisms predict response to a folinic acid
intervention. If any of these measures of physiological dysfunction predict
response to the intervention, such titers could provide a biomarker to identify
a subset of children with ASD that may benefit from a folinic acid intervention
and may even predict the development of ASD symptoms in high risk siblings
during the presymptomatic period.
biomarkers, it may be possible that children with ASD who optimally respond to
the folinic acid intervention can be readily identified early after diagnosis
or even during the pre-symptomatic period.
The study is
being conducted at the Arkansas Children’s Hospital and it has S. Jill James,
PhD and Stepan Melnky, MD, PhD as co-investigators and in collaboration with Dr
Edward Quadros, Ph.D. at SUNY Downstate in Brooklyn, NY.
Lee Silsby Compounding Pharmacy specializes in autism and
is determined to make a difference in the lives of patients with autism. Please
for more information.
The treatment category is sponsored by Lee Silsby , the leader in quality compounded medications for autism.