Acknowledging World Autism Day on the 2nd of April, Next Biosciences aims to bring you a summary of the progress, potential, and spectrum of opinion on stem cell treatment for autism spectrum disorders (ASD).
Where in the world of autism do stem cells fit in?
In the complex arena of ASD, there are many proposed interventions which can be divided into behavioural, nutritional, and medical treatment. However, due to the considerable variation of symptoms among autistic individuals, there is not yet a clear consensus on management, and many clinical trials are underway.
Stem cell therapy is a topical contender within the emerging field of autism research and has been hypothesised to improve symptoms and reduce the challenges of ASD, reducing inflammation and facilitating neural repair.
Understanding the research process
A clinical trial is a study in human patients for an experimental therapy that has not yet been adopted as a standard therapy. Such a trial is designed to evaluate the effectiveness and safety of a specific medical intervention and typically involves the comparison of outcomes between patients who received the intervention and a control group of those who did not.
Notably, autism has been treated with stem cell therapy in many clinical trials, some of which have shown significant symptom improvement. However, to be considered a standard treatment approach, a therapeutic intervention must have undergone numerous randomised placebo-controlled clinical studies that consistently demonstrate its efficacy and superiority over a placebo1.
The big picture: Meta-analysis results of stem cell treatment in autism
For enhanced accuracy and assessment of averages, a meta-analysis study can be performed. These studies combine the data from multiple existing studies on a particular topic to pool the results and gain a better (statistical) understanding of the big picture.
- A 2022 meta-analysis suggested that stem cell treatment for children with autism might be safe and effective. However, the evidence was compromised by limitations in the study size and a lack of standardisation of injection routes and stem cell dosage across the various studies2.
- Another 2022 meta-analysis reported a collective improvement in symptoms on the CARS scale (The Childhood Autism Rating Scale) and found no serious adverse effects from stem cell treatment3.
- A 2023 systematic review concluded that the minimal invasiveness and ease of use of stem cell therapy make it a promising treatment option for autism but also that the inconsistency of treatment plans across numerous studies makes it impossible to consolidate the results4.
Down to the details: What are the stem cells?
Stem cells are undifferentiated cells in the body that have the potential to develop into any cell type in the body. They are involved in the repair and replacement of damaged or diseased cells. Umbilical cord blood contains a large amount of stem cells that are easily accessible and therefore storage of umbilical cord blood at the birth of a baby is recommended. For more information about stem cells, click here.
Stem cells have been used to successfully treat several types of blood-related cancers and diseases in which they replace damaged or diseased cells of the bone marrow, regenerate new cells and reduce inflammation. Considering that ASD is associated with cellular abnormalities, disruptions in neural connectivity and inflammation, it is no wonder that stem cells have been identified as a possible treatment5.
Where we are: The current obstacles of stem cell therapy for ASD
One of the challenges facing health care providers is that the presentation, diagnosis, and treatment of autism is incredibly complex. Each patient will demonstrate a unique profile of symptoms, which means that a one size fits all approach is not possible, and treatment expectations must be managed accordingly.
Navigating the numerous alternatives for autism therapy can be overwhelming for patients and leaning on reputable sources for advice is imperative.
Where we are going: The exciting potential of stem cell therapy for ASD
In summary, stem cell therapy for autism is in the developmental stage and, but we know that stem cells are the key to regenerative medicine. In this regard, Next Biosciences - home to Netcells - looks forward to the future of stem cell science and the day that regulatory authorisation for a standard treatment is obtained.
According to Dr Joanne Kurtzberg, a researcher at Duke University Medical Centre, that day resides within the near future6. From her standpoint - working within two clinical trials extending from 2015 to 2020 - the use of cord blood infusions to treat ASD demonstrated not only administrative safety but also improvement in communication skills, eye tracking and EEG measurements in autistic patients without intellectual disability. These studies offer a positive indication that hope is abound and that a breakthrough may be imminent.
We must remember that stem cell banking only started in the early 1990s and that the breadth of medical research on cord blood and ASD is still in its infancy. However, we can certainly draw inspiration from the numerous case-study success stories and the passionate doctors and researchers in this field.
References:
- Narzisi A, Halladay A, Masi G, Novarino G, Lord C. Tempering expectations: considerations on the current state of stem cells therapy for autism treatment. Frontier – Psychiatry. October 2023.
- Qu J, Liu Z, Li L, Zou Z, He Z, Zhou L, Luo Y, Zhang M, Ye J. Efficacy and Safety of Stem Cell Therapy in Children With Autism Spectrum Disorders: A Systematic Review and Meta-Analysis. Frontiers in Pediatrics. May 2022.
- Villarreal-Martinez L, Gonzalez-Martinez G, Saenz-Flores M, Bautista-Gomez AJ, Gonzalez-Martinez A, Ortiz-Castillo M, Robles-Saenz DA, Garza-Lopez E. Stem Cell Therapy in the Treatment of Patients With Autism Spectrum Disorder: a Systematic Review and Meta-analysis. National Library of Medicine – National Center for Biotechnology Information. January 2022.
- Akat A, Karaoz E. Cell therapies for autism spectrum disorder: a systematic review of clinical applications. Springer Open. December 2023.
- Astorkia M, Lachman HM, Zheng D. Characterization of cell-cell communication in autistic brains with single-cell transcriptomes. BMC - Part of Springer Nature. May 2022.
- Hope for the Treatment of Autism. CryoCell International.