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Stem Cells Therapy for Autism: Does it Work?

October 12, 2023

Most of us are familiar with the scientific fact that any living, breathing animal, insect etc. is made up of cells. These cells form tissues and organs that support the existence of the host. Many of us have also heard of stem cells therapy for autism but are unsure about its validity.

Stem Cells Therapy for Autism: Does it Work?

Scientists have studied the underlying mechanism of cells, as well as their functioning, and have discovered ways of using the cells to improve the lives of humans and treat diseases. To do so, scientists have discovered stem cells; think of it as the building blocks of a fully differentiated cell.

Stem cells are human cells that can be developed and differentiated into other cell types. These cells can be derived from any part of the body, for example, stem cells from the brain, muscle, bone marrow, etc. Stem cells are versatile in that they can be used to fix damaged tissues. The two essential characteristics of stem cells include: Firstly, the ability to self-renew to create successors identical to the original cell. Secondly, stem cells, unlike cancer cells, are controlled and highly regulated, therefore, stem cells need to be able to give rise to specialized cell types that become part of the healthy body.

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The purpose of stem cell therapy is to regenerate and repair damaged tissues and cells in the body. There are two main classes of stem cells. Pluripotent stem cells have the potential to become any cell in the adult body and multipotent stem cells are much more restricted to a specific population or lineage of cells. Other stem cell types include totipotent and unipotent.

A look at the two main types of stems cells

Let’s look at pluripotent and multipotent stem cells in detail.

Pluripotent stem cells

Pluripotent stem cells are generated from somatic cells. These mainly come from embryos and, as such, they’re often referred to as “embryonic stem cells”.

Let’s discuss three types of embryonic stem cells that are used to generate pluripotent cells. These include true embryonic stem cells (ES), nuclear transfer of somatic cells (ntES), and parthenogenetic embryonic stem cells (these are stem cells from unfertilized eggs).

1. “True” embryonic stem cells (ES)

The true embryonic stem cells are made from unused embryos, such as those that undergo IVF (in vitro fertilization). The process of IVF is such that the eggs and sperm are fertilized in a lab dish. What then happens is that, through this process, more embryos are generated, usually more than the couple actually need. Those that aren’t used can be donated to science.

Pluripotent cells made from these unused embryos are not genetically matched to the original hosts. These are mainly used in science for studies to learn how stem cells regenerate. 

2. Nuclear transfer of somatic cell (ntES)

Every cell contains an organelle called a nucleus. The nucleus contains all the cell’s genetic information essential to its function. The word somatic refers to any cell in the body. 

The process of somatic cell nuclear transfer (SCNT) extracts the nucleus from a somatic cell and transfers it into another cell that has had its own nucleus removed; i.e. the nucleus from the previous cell is being transferred to an egg cell that does not contain a nucleus (unnucleated).

When the nucleus is transferred to another cell, it activates the process of pluripotent cell generation that reprograms the generation of genes in that cell. The egg then becomes a zygote nucleus or a fertilized egg, the cell then replicates and through it embryonic stem cells are created.

3. Parthenogenetic embryonic stem cells (pESCs)

Imagine being able to fertilize an egg without fertilization by sperm. Unusual, but science makes crazy things happen. 

Parthenogenesis is the process whereby an unfertilized egg develops an embryo without fertilization. This can be achieved through chemical, physical or combined activation methods.

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The parthenogenetic embryonic stem cells have the capacity for infinite proliferation and self-renewal, and maintain the ability to differentiate into one or more specialized types of cell or tissue.

pESCs are especially useful for regenerative medicine, and therefore allow the generation of functional cells that could potentially be used as treatment for many incurable diseases in the future.

Advantages of pluripotent cells

  • These stem cells are useful for studying how diseases occur
  • Scientists can track the point at which a disease began developing, or the event at the cellular level that may have led to the formation of the disease
  • Allows the scientist to study the difference between the diseased cell and the normal cell at the genetic and/or cellular level
  • Stem cells made through the ntES, or pECS process have the opportunity to create rejection-proof cells that would benefit individuals going through tissue or organ transplants

Multipotent stem cells

Multipotent stem cells are unspecialized cell types that have the ability to self-renew and differentiate into specialized cell types. However, these cells are specific to the type of tissue or organ. For example, a multipotent adult stem cell from the bone marrow can become specialized to produce all blood cell types; and cells in the stem cells from neural networks in the brain can specialize to glial and neuronal cells. 

When we talk about all blood cell types, we have to get a little scientific, but for the curious mind, “all blood cell types” refers to platelets, B and T lymphocytes, natural killer cells, dendritic cells….the list goes on.

In addition, for the curious mind, various types of stem cells include hematopoietic stem cells (the ones that make blood cell types), mesenchymal stem cells (differentiate into bone, fat, cartilage, muscle, and skin), and neural stem cells (from neural networks).

Now that we’ve covered the types of stem cells, the question remains, can stem cell therapy cure autism? Let’s have a look.

Can autism be “cured” by stem cell therapy?

To answer this question, I refer to the review by Price (2020) as it is the latest up-date data on this subject. It is important to note however, that at the time of reading this article there may be other research data published on this topic.

Several research studies cite immune dysfunction as the cause and effect of autism spectrum disorder (ASD). By virtue of this analogy, it has informed the basis of the stem cell therapy approach for treating autism. This is founded on the properties that regulate the immune system (immuno-regulatory properties).

From the review, it was also found that when exposed to inflammatory stimuli, this may lead to the development of postnatal diagnosis of ASD. Inflammation to the cell describes the process that occurs when the cell is exposed to harmful stimuli such as bacteria, trauma, toxins, heat, and pathogens. The affected cells then release chemicals that cause blood vessels to leak fluid into the tissues, causing swelling. 

Therefore, an inflammatory stimuli is that which influences the occurrence of an inflammatory response.

Other bodies of research found an altered level of proteins called cytokines which are essential for interaction and communication between cells in ASD. These may also be the cause of the development of autism spectrum disorder. Some genetic studies propose an association between a genetic loci (a specific point on the genome of the autistic individual) and ASD whose function is related to immune function. While others suggest a possible anomaly in the neuronal signaling pathway that directs communication and information transfer between neurons

All these are proposed reasons that hypothesize the use of stem cell therapy to treat autism biologically. However, all these propositions do not lead to one voice, there are too many hypotheses that make it difficult to narrow down the target area that would potentially treat autism or autism symptoms. Keeping in mind that autism traits are diverse, therefore, narrowing this information down to one plausible pathology is an even greater challenge.

How effective is stem cell therapy for autism?

So, is stem cell therapy effective? The answer to this is unknown. 

Is ASD caused by genetic, immune dysfunction, or inflammatory stimuli? The answer to this is not clear and there’s a vast number of studies that argue different theories. 

It is even more disturbing to consider these hypotheses because, for example, each person can experience bacterial or viral infections, or stress that can impact immune functioning and/or lead to inflammation but we’re not all on the spectrum. Therefore, we can’t say that factors which alter our immune functioning lead to the development of neurodevelopmental conditions. 

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However, according to Price, the study by Riordan et al. (2019) proposes the influence of cytokines for the treatment of autism.The data proposed could be a point in a positive direction to answering whether stem cell therapy could potentially treat autism symptoms.

What is the success rate of stem cell therapy?

Unfortunately, there is no data to positively state the effectiveness of stem cell therapy for treating autism. As more research is developed in this field, there’s hope that more understanding of autism will arise, and perhaps an alternative form of treatment of autism symptoms can be developed. It is also worth noting the possibility of genetic markers that could help diagnose autism during pregnancy or during the prenatal development stage.

In summary

The studies highlighted in this article are simply preliminary assessments. Further research needs to be conducted in order to understand the potential of cell therapies for treating autism. 

The findings of these studies vary in hypothesis and this makes generalization hard. Science has developed greatly over years, therefore, for those that believe in the potential of science and all that it could offer, there’s a reason to hope that stem cell therapy could potentially be used as treatment for autism in the near future.


Biehl, J. K., & Russell, B. (2009). Introduction to stem cell therapy. The Journal of cardiovascular nursing, 24(2), 98–105. https://doi.org/10.1097/JCN.0b013e318197a6a5 

Price, J.(2020). Cell therapy approaches to autism: a review of clinical trial data. Molecular Autism, 11, 37 . https://doi.org/10.1186/s13229-020-00348-z


Thermo Fisher Scientific. An Overview of Pluripotent and Multipotent Stem Cell Targets. https://www.thermofisher.com/za/en/home/life-science/antibodies/antibodies-learning-center/antibodies-resource-library/antibody-methods/pluripotent-multipotent-stem-cell-targets.html 

Yu, Z., Han, B. (2016). Advantages and limitations of the parthenogenetic embryonic stem cells in cell therapy. Journal of Reproduction and Contraception, 27 (2), Issue 2, 118-124. https://doi.org/10.7669/j.issn.1001-7844.2016.02.0118

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