Stem cell therapy is the latest scientific breakthrough in the medical treatment of autism. Whereas on one hand, stem cell therapy brings a lot of hope to children with autism, there is also a lot of hype. Therefore, the optimism of stem cell therapy needs to be considered with caution before we make any final decision in this connection.

On one hand, many centres all the over the world claim to have treated children with autism, with a good safety record and reasonably good clinical improvements. On the other hand, many members of the medical community still believe that this treatment is in its early stages and is not completely established.

To proceed with our recommendations and views on the subject of stem cell therapy, it is first imperative to understand the various types and sources of stem cells.

Broadly speaking, stem cells are of two types:

I. Autologous stem cells: Autologous stem cells are those which are obtained from the patient’s own body. They may be obtained from 1) Bone Marrow, 2) Abdominal fat and 3) umbilical cord (if preserved at birth)

II. Allogenic stem cells: Allogenic stem cells are those procured from matched related or unrelated donors. These may be subdivided into those of embryonic origin or non-embryonic origin such as the cells procured from another person’s umbilical cord.

In the above categories, there are two sub-classifications:

I. Minimally manipulated: Minimally manipulated stem cells are those which are transplanted without altering their fundamental nature. II. More than minimally manipulated: More than minimally manipulated stem cells are those which are transplanted after significantly altering their fundamental nature.

The following is the view of Autism Connect and is meant to help parents decide:

Based on a review of the literature, along with the information available in the public domain and personal discussions with parents who have already taken stem cell therapy for their children, we believe that stem cell therapy definitely offers a potential medical solution to healing those parts of the dysfunctional brain that cause autism.

1. We are in agreement with the statement made in the white paper published by the International Society for Cellular Therapy with regard to two aspects;

  • The right to seek treatment:

    "Patients seeking medical treatment for cellular therapies have the following rights that must be respected by healthcare providers and all associated with their care. a. The right to seek treatment: patients and their families/partners have the right to seek treatments for their diseases. No entity should withhold this fundamental right unless there is a high probability of harm to the patients. b. The right to information: patients have the right to an accurate representation regarding the safety and efficacy record of the cell treatment. This includes probable side-effects and a truthful record of efficacy. c. The right to informed consent: patients have a right to a true informed consent process that includes all the elements described above."

  • Distinction between legitimate medical stem cell therapy services and fraudulent services:
    "The following guidelines are useful in assessing scientific rigor and for differentiating between legitimate cell therapy medical services (including clinical trials and medical innovation) and fraudulent cell therapies.
    • Peer review and transparency: consumers of cell therapy medical innovation should evaluate evidence from peer-reviewed publications, professional society presentations and scientific recognition. They should be encouraged to seek multiple professional opinions and have all questions answered to their satisfaction.
    • Safety and regulatory history: patients should consider the reputation of the investigator and clinic, as well as the record of disciplinary activities against these entities.
    • Informed consent: patients should expect to be informed fully and accurately of the risks, benefits, costs, safety, compensation for injury, investigator conflicts of interest and alternative therapies, as a minimum."

Bearing this in mind, as a minimum, below are our recommendations to parents looking at the option of stem cell therapy for their children. (Please note that this is not a substitute for a formal medical advice and all parents are required to do their own due diligence before making this important decision.)

All parents should initially try the conventional rehabilitative treatment such as occupational therapy, speech therapy, ABA, etc. They should also take all the medications advised by the pediatric neurologists/psychiatrists/other physicians to control symptoms such as epilepsy, hyperactivity, etc. Complementary treatment such as dietary regulation, supplements, etc. may also be considered. Stem cell therapy should be considered only for those where despite all conventional rehabilitation, the child continues to remain significantly symptomatic.

Once parents have decided to consider stem cell therapy, the next important decision would be which type of stem cells to take and where to undergo the therapy. The following is our view in this connection:

We believe in safety first and therefore recommend that at this stage of evolution of stem cell therapy, it is safer to use:

A. Autologous stem cells

B. Minimally manipulated stem cells

C. Bone marrow derived stem cells (since these are obtained by a needle aspiration only, as compared to abdominal fat stem cells, which require a surgical procedure of laparoscopy to extract the fat from the abdomen).

We would also recommend that parents should consider only those centres that have published their safety and clinical research in scientific medical journals. The reason for this is that when something is published in a medical journal, it is considered as peer-reviewed. This means that someone apart from the treating doctor has reviewed the safety and efficacy of the treatment and considered it worthy and accurate enough to be published in their journal. We believe that information on hospital and clinic websites online is not good enough, since there is no system of checks and balances to ensure that only authentic and accurate information is put up on the website.

We also firmly believe that the therapies should be offered only after an informed consent which provides information on risks, benefits, cost of the therapy, other therapy options available, safety of the therapy, compensation provided by the investigator in case of any adverse event and any conflict of interest of the investigator.

On review of the medical literature through the PubMed scientific database, we have found that the following centers have published their clinical results in medical journals:

  1. NeuroGen Brain and Spine Institute, Mumbai, India

Alok Sharma, Nandini Gokulchandran, Hemangi Sane, Anjana Nagrajan, Amruta Paranjape, Pooja Kulkarni, Akshata Shetty, Priti Mishra, Mrudula Kali, Hema Biju, Prerna Badhe. Autologous bone marrow mononuclear cell therapy for autism - an open label proof of concept study. Stem cell international. 2013 (2013), Article ID 623875, 13 pages.

The above publication, along with 8 more publications on autism from this institute, can be accessed here

  1. Shenzhen Beike Biotechnology Co Ltd Shenzhen China

Yong-Tao Lv, Yun Zhang, Min Liu, Jia-na-ti Qiuwaxi, Paul Ashwood, Sungho Charles Cho et al. Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. Journal of Translational Medicine 2013, 11:196

The above can be accessed here

  1. Emcell, Kyiv, Ukraine.

Bradstreet JJ, Sych N, Antonucci N, Klunnik M, Ivankova O, Matyashchuk I, M Demchuk; Siniscalco D. Efficacy of fetal stem cell transplantation in autism spectrum disorders: an open-labeled pilot study. 2014;23 Suppl 1:S105-12

The above can be accessed here

  1. Duke Centre for Autism and Brain Development, Duke University Medical Centre, Durham, USA

Dawson, Geraldine, Jessica M. Sun, Katherine S. Davlantis, Michael Murias, Lauren Franz, Jesse Troy, Ryan Simmons, Maura Sabatos‐DeVito, Rebecca Durham, and Joanne Kurtzberg. "Autologous cord blood infusions are safe and feasible in young children with autism spectrum disorder: Results of a single‐center phase I open‐label trial." Stem Cells Translational Medicine 6, no. 5 (2017): 1332-1339.

The above can be accessed here

Although many centers all over the world claim to get good clinical results with stem cell therapy, there is no way we can authenticate or validate those claims. Hence, our recommendation to parents is to preferably consider the centers listed above, whose work is published.

What is stem cell therapy?

Recently, stem cell therapy is evolving as a new treatment for various incurable disorders such as autism. This therapy aims at replacing the damaged cells of the body using healthy cells. In autism, selective areas of the brain are damaged and do not function normally. Hence, this therapy helps in repairing and restoring the affected functions and thereby improving the quality of life of the individuals affected with autism. It helps to make these children independent and integrate them into the society.

What are stem cells? 

Stem cells are the fundamental elements forming all the tissues of the human body. They are unique cells with an ability to rapidly multiply and convert into any type of tissue in the body. The main purpose of these cells is to regenerate, repair and replace damaged tissue. They are naturally capable of travelling towards the damaged areas and start the repair process.

What are the different types of stem cells?

There are many types of stem cells, but broadly they are classified into 3 types:

(a) Embryonic stem cells

(b) Umbilical cord stem cells

(c) Adult stem cells 

 (a) Embryonic Stem cells: These cells are obtained from the embryo or an unborn fetus. These are obtained from spare embryos from IVF clinics. These cells are not being used presently due to various medical and ethical controversies associated to them. Tumor formation is recorded as one of the serious side effects of these cells.

(b) Umbilical cord Stem cells: These cells are derived from the umbilical cord which connects the baby and mother at birth. They are safer and less controversial compared to embryonic cells. But, since these cells are derived from outside the patient, the donor cells have to be compatible with the recipient. Various cord blood banking companies store these cells. Storage, availability and expense can be a concern.

(c) Adult Stem cells: These cells are obtained from the adult tissues, either from the hip bone or the fat/adipose tissue, etc. Currently, these are the safest and most popularly used stem cells. They are easily available and safe for use. Recently, another type of stem cell called Induced Pluripotent Stem Cells (iPSCs) has shown a lot of potential. iPSCs are made from cells taken from the patient which are genetically modified to behave like an embryonic stem cells. These cells combine the efficacy of embryonic stem cells with the safety of adult stem cells.

How do stem cells work in autism?

Stem cells repair, regenerate and replace the damaged cells in the brain of the individual with autism by following mechanisms:

  • They release growth factors which help to regulate the immune system of individuals with autism and have a healing and regenerative effect on damaged tissue.
  • They increase the blood supply to the damaged tissue by forming new blood vessels by a process called angiogenesis thereby helping in their repair process.
  • They can convert into any tissue type into which they are injected, thereby replacing non-functioning tissue.

What are the results and improvements after stem cell therapy in autism? 

The outcome of stem cell therapy in autism is remarkably positive. 91% of individuals with autism have shown clinical improvements. A decrease in aggressive behaviour & hyperactivity, improved eye contact & attention span, improvements in communication & social skills are observed. Improvements are also reported in the PET CT scan of the brain as the change in metabolic activity of the brain. These changes correlate to the clinical improvements confirming the positive effect of stem cell therapy in autism.