Human Umbilical Cord
Mesenchymal Stem Cells
for Autism

 

Clinical Trial

Translational Biosciences DRAFT logo 3Translational Biosciences, a wholly-owned subsidiary of Medistem Panama is currently recruiting patients for this IRB-approved clinical trial. We encourage anyone suffering from autism who meets the inclusion/exclusion criteria below to apply.


Umbilical Cord Tissue-derived Mesenchymal Stem Cells for Autism

Abstract

1 in 150 children develop one of the autism spectrum disorders (ASD), which are characterized by impairment of interest or ability for social interaction, repetitive and stereotypic behavior, impaired ability to communicate, and abnormal response to stimuli. Various immunological and inflammatory abnormalities have been associated with ASD. Umbilical cord tissue derived mesenchymal stem cells (UC-MSC) have been demonstrated to inhibit production of inflammatory mediators, as well as to possess neuro-protective/neuro-regenerative activity.

The proposed study seeks to determine the safety and efficacy of UC-MSC administered to 20 male or female children between the ages of 6 to 16 years old DSM IV diagnosis of autism confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic Interview-Revised (ADIR) as follows: A total of 144 million UC-MSC administered intravenously over the course of one year as series of 4 infusions of 9 million U C-MSC on given on consecutive days once every 12 weeks. The primary objective of safety will be defined as freedom from treatment associated serious adverse events and will be assessed every 12 weeks from the first series of 4 infusions until the final series 3 7 weeks later. Following the last infusion, safety will be assessed at weeks 49 and 89. Secondary efficacy parameters will comprise endpoints of Autism Treatment Evaluation Checklist (A TEC), Childhood Autism Rating Scale (CARS) and changes in serum levels of macrophage-derived chemokine (MDC) and thymus and activation-regulated chemokine (TARC). Efficacy will be assessed at baseline and every 12 weeks from the first series of 4 infusions until the final series 37 weeks later. Following the last infusion, efficacy will be assessed at weeks 49 and 89.

This study will provide support for double-blind placebo controlled investigations. The potential of using cell based therapy for autism will provide a research direction for a condition that currently represents an unmet medical need. This protocol will be conducted under the safety standards and in accordance with the guiding principles of GCP and adequate human subject protection, as well as with local and international policies and regulations that govern the discipline of clinical research and use of stem cells.

Inclusion Criteria

  • Male or Female
  • Ages 6 to 16
  • Diagnostic and Statistical Manual of Mental Disorders (DSM IV) diagnosis of autism confirmed by Autism Diagnostic Observation Schedlie (ADOS) and/or Autism Diagnostic Interview-Revised (ADI-R)
  • No anticipated changes in treatment for the study duration (e.g., diet, nutrients)
  • No additional biomedical treatments started 6 weeks prior to enrollment
  • No changes in dietary management for 3 months prior to enrollment
  • Ambliatory or require minimum support walking, per parent
  • Able to sit still for 5 minutes or longer with a preferred toy item, per parent
  • Adequate vision and hearing for the purposes of test administration, per parent
  • Adequate arm-hand-finger coordination (i.e., able to point) for learning and cognitive tasks used in outcome measurement, per parent
  • Stable and controlled mental disorder
  • Under the care of a caregiver willing to participate by attending regliarly schedlied appointments and completing the necessary measures
  • Normal heavy metals test for lead and mercury levels performed within 30 days of first stem cell infusion
  • Must provide name and specialty of specialist who has made Autism Spectrum Disorder (ASD) diagnosis
  • Adequate financial means to cover $7,200 (US Dollars) plus travel expenses

 

Exclusion Criteria

  • Significant prematurity at birth (less than 32 weeks gestation); or birth weight significantly below normal for gestational age (SGA – small for gestational age)
  • mental retardation
  • seizure disorder
  • auto-immune conditions
  • history of head trauma and other neurological or medical conditions
  • Abnormal heavy metals test for lead and mercury performed within 30 days of first stem cell infusion
    Prior stem cell therapy of any kind

As seen on ClinicalTrials.gov

View this clinical trial on National Institutes of Health ClinicalTrials.gov