Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders (Autism)

The purpose of this study is to determine whether the plasticity of autologous intrathecal hematopoietic cells would improve the neurologic and the social skills of pediatric patients with autism spectrum disorders.

Study Overview

• Status: Completed
• Conditions: Autism; Autism Spectrum
• Intervention / Treatment: Biological: Autologous Intrathecal Hematopoietic Stem Cells; Other: Observation with IDEA and CARS Scales

Detailed Description

There is accumulating evidence that shows that the administration of hematopoietic cells into the brain in the patients with spectrum autism could help in the physiopathology of the illness.

It has been found that after introducing hematopoietic cells in the subarachnoid space of the spinal cord, these cells may be transported through the cerebrospinal fluid and can be delivered more efficiently to the injured area, when compared to the intravenous route.

Patients will be stimulated for 3 consecutive days with granulocyte colony stimulating factor (G-CSF) and then their bone marrow will be harvested according to their weight. Bone marrow will be processed in order to obtain CD34+ cells and minimize the amount of red blood cells. An inoculum of 5 to 10mL of stem cells will be infused intrathecally. Patients will be evaluated with two scales "CARS" and the "IDEA" also we will check the clinical history. On days 0, 30 and 180.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 3

Contacts and Locations

Study Locations

• Mexico
  • Nuevo León
    • Monterrey, Nuevo León, Mexico, 64460
      • Hematology Service, Hospital Universitario Dr. Jose E. Gonzalez

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 11 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria: Children aged 5 to 15 years, Diagnosis of Autism Spectrum Disorder (ASD) confirmed by DSM-5 criteria, Ability to comply with study procedures, including assessments with CARS and IDEA scales, Written informed consent obtained from parents or legal guardians.

Exclusion Criteria: Presence of severe neurological disorders other than ASD, History of immunodeficiency or hematologic disorders, Active infection at the time of enrollment, Previous treatment with intrathecal stem cells, Any medical condition that, in the investigator's judgment, would make participation unsafe.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patients; Children with autism spectrum disorder who will receive intrathecal infusion of autologous CD34+ hematopoietic stem cells after G-CSF stimulation and bone marrow harvest.	Biological: Autologous Intrathecal Hematopoietic Stem Cells; Patients will be stimulated with granulocyte colony-stimulating factor (G-CSF) for 3 consecutive days. Bone marrow will be harvested, processed to isolate CD34+ hematopoietic stem cells and reduce red blood cells. An inoculum of 5-10 mL will then be infused intrathecally into the cerebrospinal fluid.
Other: Control; Children with autism spectrum disorder who will be observed for 6 months and evaluated with CARS and IDEA scales, with option to crossover to the experimental arm afterwards.	Other: Observation with IDEA and CARS Scales; Participants will be observed for 6 months without active intervention. They will be evaluated using the Childhood Autism Rating Scale (CARS) and the IDEA scale at baseline, 30 days, and 180 days. After 6 months, participants may crossover to the experimental arm.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Childhood Autism Rating Scale (CARS) score	CARS scale will be used to evaluate changes in autistic behavior and social interaction after treatment. Participants will be assessed at baseline, 30 days, and 180 days to measure improvement following the intrathecal infusion of autologous CD34+ hematopoietic stem cells.	Baseline, 30 days, and 180 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in IDEA scale score	IDEA scale will assess developmental and social abilities in participants. Measurements will be taken at baseline, 30 days, and 180 days to evaluate the effect of treatment on developmental progress.	Baseline, 30 days, and 180 days

Collaborators and Investigators

• Sponsor: Hospital Universitario Dr. Jose E. Gonzalez

Publications and helpful links

General Publications

• Siniscalco D, Sapone A, Cirillo A, Giordano C, Maione S, Antonucci N. Autism spectrum disorders: is mesenchymal stem cell personalized therapy the future? J Biomed Biotechnol. 2012;2012:480289. doi: 10.1155/2012/480289. Epub 2012 Feb 13.
• Ichim TE, Solano F, Glenn E, Morales F, Smith L, Zabrecky G, Riordan NH. Stem cell therapy for autism. J Transl Med. 2007 Jun 27;5:30. doi: 10.1186/1479-5876-5-30.
• Mehta T, Feroz A, Thakkar U, Vanikar A, Shah V, Trivedi H. Subarachnoid placement of stem cells in neurological disorders. Transplant Proc. 2008 May;40(4):1145-7. doi: 10.1016/j.transproceed.2008.03.026.
• Hayashi T, Iwai M, Ikeda T, Jin G, Deguchi K, Nagotani S, Zhang H, Sehara Y, Nagano I, Shoji M, Ikenoue T, Abe K. Neural precursor cells division and migration in neonatal rat brain after ischemic/hypoxic injury. Brain Res. 2005 Mar 15;1038(1):41-9. doi: 10.1016/j.brainres.2004.12.048.
• Nakatomi H, Kuriu T, Okabe S, Yamamoto S, Hatano O, Kawahara N, Tamura A, Kirino T, Nakafuku M. Regeneration of hippocampal pyramidal neurons after ischemic brain injury by recruitment of endogenous neural progenitors. Cell. 2002 Aug 23;110(4):429-41. doi: 10.1016/s0092-8674(02)00862-0.
• Goldman SA, Schanz S, Windrem MS. Stem cell-based strategies for treating pediatric disorders of myelin. Hum Mol Genet. 2008 Apr 15;17(R1):R76-83. doi: 10.1093/hmg/ddn052.
• Rempe DA, Kent TA. Using bone marrow stromal cells for treatment of stroke. Neurology. 2002 Aug 27;59(4):486-7. doi: 10.1212/wnl.59.4.486. No abstract available.
• Felling RJ, Snyder MJ, Romanko MJ, Rothstein RP, Ziegler AN, Yang Z, Givogri MI, Bongarzone ER, Levison SW. Neural stem/progenitor cells participate in the regenerative response to perinatal hypoxia/ischemia. J Neurosci. 2006 Apr 19;26(16):4359-69. doi: 10.1523/JNEUROSCI.1898-05.2006.
• Gordon PH, Yu Q, Qualls C, Winfield H, Dillon S, Greene PE, Fahn S, Breeze RE, Freed CR, Pullman SL. Reaction time and movement time after embryonic cell implantation in Parkinson disease. Arch Neurol. 2004 Jun;61(6):858-61. doi: 10.1001/archneur.61.6.858.
• Kulbatski I, Mothe AJ, Nomura H, Tator CH. Endogenous and exogenous CNS derived stem/progenitor cell approaches for neurotrauma. Curr Drug Targets. 2005 Feb;6(1):111-26. doi: 10.2174/1389450053345037.

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2012
• Primary Completion (Actual): November 1, 2016
• Study Completion (Actual): October 1, 2017

Study Registration Dates

• First Submitted: November 22, 2012
• First Submitted That Met QC Criteria: November 30, 2012
• First Posted (Estimated): December 4, 2012

Study Record Updates

• Last Update Posted (Estimated): September 16, 2025
• Last Update Submitted That Met QC Criteria: September 10, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: children; autism; stem cells; autism spectrum
• Additional Relevant MeSH Terms: Autism Spectrum Disorder; Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autistic Disorder; Investigative Techniques; Methods; Observation
• Other Study ID Numbers: HE12-021; Autism uanl (Other Identifier: Autism uanl)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• product manufactured in and exported from the U.S.: No