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Autism and Stem Cell Therapy

Autism and Stem Cell Therapy

Mesenchymal stem cells (MSCs) are self-renewing, multipotent progenitor cells with multilineage potential to differentiate into cell types of mesodermal origin. In addition, MSCs can;

  • Migrate to sites of inflammation,
  • Exert anti-inflammatory effects,
  • Modulate and suppress immune system through interactions between lymphocytes associated with both the innate and adaptive immune system,
  • Differentiate, proliferate and repair damaged tissues.


The immune system is thought to play an important role in autism. Interactions between the immune system and the nervous system begin early during the embryonic stage of life, and successful neurodevelopment depends on a balanced immune response.

MSCs participate in both innate and adaptive immunity, and their immunomodulatory functions are exerted mainly via interactions with immune cells through cell-to-cell contact and paracrine activity involving T cells, B cells, natural killer (NK) cells, macrophages, monocytes, dendritic cells (DCs) and neutrophils. Because of their tendency to interact with other cell types and their immunomodulatory abilities, MSCs have become a prospective therapeutic approach. Different inflammatory cytokines control the immunomodulatory capacity of MSCs, and the interaction between immune cells and MSCs may promote both regeneration and the development of various inflammatory disorders. Cell-to-cell contact and paracrine activity primed with cytokines, chemokines, extracellular vesicles, inflammatory stimuli, or co-culture with other cells are the key processes involved in MSC immunomodulation.


Currently, mesenchymal stem cells (MSCs) seem to hold the most promise for cell therapies for autism spectrum disorder (ASD), likely owing to their immunomodulatory capacities. It is hypothesized that they would function in ASD treatment through the secretion of anti-inflammatory cytokines and survival-promoting growth factors and integrating into neural networks and restoring plasticity. Early clinical trials of human umbilical cord-derived mesenchymal stem cells (UCMSCs) transplantation have shown some efficacy in treating ASD.

In autism, stem cells implantation treatment and self stem cells activation treatment to repair the motor neuron damage and would be expected to have a peripheral anti-inflammatory effect, with the potential to heal the gut and quiet autoimmune reactions. The patient will receive treatment to improve blood circulation to increase the blood supply of those damaged nerves and nourish the neurons, together with daily rehabilitation training to promote the recovery of his motor function. This kind of treatment method can make the patient’s spirit and intelligence become well. At the end, the disease could be halted.

However, when discussing improvements, it is important to remember that improvements might greatly differ from one patient to another due to many factors, such as patient’s medical course, physical condition, severity, age and so on. Therefore, improvement cannot be guaranteed.


Since umbilical cord derived mesenchymal stem cells are immune system privileged, cell rejection is not an issue and Human Leukocyte Antigen (HLA) matching is not necessary.


The stem cells with the best anti-inflammatory activity, immune modulating capacity, and ability to stimulate regeneration can be screened and selected.

Allogeneic stem cells can be administered multiple times over the course of days in uniform dosages that contain high cell counts.


Umbilical cord tissue provides an abundant supply of mesenchymal stem cells.

No need to collect stem cells from the patient’s hip bone or fat under anesthesia, which especially for small children and their parents, can be an unpleasant ordeal.


There is a growing body of evidence showing that umbilical cord-derived mesenchymal stem cells are more robust than mesenchymal stem cells from other sources.


No need to administer chemotherapy drugs like granulocyte-colony stimulating factor (G-CSF or GCSF) to stimulate the bone marrow to produce granulocytes and stem cells and release them into the bloodstream.


     Who are Suitable for Stem Cell Therapy?

Patients aged between 6 to 16 with a diagnosis of autism confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic Interview-Revised (ADI-R) according to DSM 5.

No anticipated changes in treatment for the study duration (e.g., diet, nutrients)

No additional biomedical treatments started 6 weeks prior to treatment

No changes in dietary management for 3 months prior to treatment

Ambulatory 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 regularly scheduled appointments and completing the necessary measures

Normal heavy metals test for lead and mercury levels performed within 30 days of first stem cell infusion

Adequate financial means.



Which Stem Cell Types are Used for Cellular Therapy?

Main cellular product used for the treatment of ASD is Mesenchymal Stem Cells (MSCs). MSCs can be produced from a variety of different tissues but Umbilical Cord (UC) derived MSCs are mostly preferred. Donors of the raw tissue are chosen with comprehensive evaluations. As all MSC products, UC-MSCs can also be used allogenically. They are minimum divided young and healthy cells that has been least affected by environmental factors, medicines and other harmful impacts. After manufacturing, MSC products are elaborately analyzed for their cellular, genetic and microbiologic characteristics. Final UC-MSC product is a sterile, apyrogenic, stabile, safe, effective and live product which can be directly injected according to the treatment protocol of the physician.


Although MSCs are considered as the most reliable, safe and efficient cellular therapy products, bone marrow derived mononuclear cells (MNCs), cord blood hematopoietic stem cells (HSCs) and induced pluripotent stem cells (iPSCs) are also alternative cellular products used in the treatment of ASD in different clinical trials.

MSC derived exosomes are also another option that emerged in the last years. Exosomes, with a diameter of about 40–100nm, are biological nanoscale spherical lipid bilayer vesicles secreted by cells. Studies have found that exosomes contain nucleic acids, proteins, lipids, cytokines, transcription factor receptors and other bioactive substances. There are studies suggesting that MSC derived exosomes regulate neuroinflammatory factors in the brain.


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