Current Therapies are Symptomatic

Autism spectrum disorder (ASD) is a developmental disorder, characterized by deficits in communication, social interactions and sensory perception and by repetitive behavior, typically manifesting in the first three years of life. No curative therapies are available to treat the condition, whose causes remain poorly understood.

Thus, current therapeutic approaches to ASD are symptomatic. However, symptomatic treatments fail to address core etiological factors. Personalized Medicine will provide therapeutic options that are tailored to address the specific diagnostic findings of each individual. Early intervention is often associated with substantial improvements.

Unfortunately, despite intensive behavioral, psychological and/or educational therapies and pharmaceutical treatment to minimize symptoms, many individuals fail to show significant improvement. Response to stem cell treatment may enable subjects to benefit from psychological/behavioral therapy, which would give them more independence and reduce lifelong support and family stress.

Targeted Medicine: Innovative Stem Cell Therapies

Contemporary researchers and the medical community are exploring the potential use of stem cells (SCs) for many medical conditions due to their unique therapeutic properties, both in tissue regeneration and immune modulation. Given their high safety profile, SCs qualify to be a promising option for the treatment of ASD in terms of potential to restore normal immunological function.

WHAT ARE STEM CELLS

SCs are easily isolatable and self-renewing cells with the ability to differentiate into a broad range of specialized cell types. SCs have been appreciated for their benefits in treating disease, particularly by driving tissue repair and immuno-modulatory responses. SC transplants (from another individual) and re-implants (autologous, from the patient’s own system) are routinely performed as standard treatment for oncological diseases following intense radiotherapy or chemotherapy. In recent years, autologous SCT has been assessed for its impact on a number of degenerative and inflammatory diseases and shown to improve symptoms.

Stem Cell Therapy for ASD

Epidemiological and immunological evidence points to an immune etiology in a subset of children with ASD. Cell-El’s preliminary data from its diagnostic study confirm this observation. Thus, SC infusion may have a curative impact on ASD, and data from the literature supports this concept. Optimal dosage, ideal age for treatment, and number and timing of treatments remain open questions for our clinical research. Cell-El seeks to more comprehensively assess the effect of autologous SCT on young ASD patients. Our clinical work focuses on younger patients, since at this stage brain plasticity remains high and more responsive to immuno-modulation.

Autologous SCT – Description of the Procedure

SCs are collected from the sedated patient with mild and short-acting anesthesia. 3-5 ml/kg body weight of bone marrow are removed from the hipbone, which typically does not cause any side effects. The aspirated bone marrow is processed on-site, then re-injected back into the patient within 30-60 minutes.

bone marow

Bone Marrow aspiration

Lumbar Puncture

Treatment with Bone Marrow-Derived Stem Cells (BM-SC)

1. Evaluation

  • Clinical (ADOS…)
  • Laboratory (serum, stool urine)

<1 month pre-treatment

 

2. Bone Marrow Aspiration (3-5 ml/kg body weight) → separated in Operating Room

3. Administration
IV + IT

4. Evaluation
  • Clinical (ADOS…)
  • Laboratory (serum, stool urine)
1 + 3 + 6 months post-treatment

Steps 2 and 3 are under mild sedation, < 1 hour, ambulatory

Monitoring after SCT

Cell-El’s planned protocol includes monitoring participants at 1, 3, 6 months after the SCT for psychological and laboratory evaluations. Studying these changes may enable Cell-El to develop a biomarker based tool to guide treatment efficacy.

Further reading: Therapeutic properties of mesenchymal stem cells for autism spectrum disorders, Medical Hypotheses

If you are interested in finding out more about our diagnostic and therapeutic studies, please contact us.

FAQ

  • The mechanism by which SCs provide targeted treatment for ASD is modulating the underlying immune dysfunction as well as improving the function and regeneration of normal brain tissue. In young children, brain plasticity, or the capacity to regenerate brain tissue, is particularly high. The synergistic effect of immune modulation and brain tissue regeneration may lead to a significant clinical improvement.

Therapeutic Rationale of MSC for ASD

  • Chronic autoimmune reaction
  • Neurogenesis ↓ (neuro-developmental disorder)

Due to the dual properties of MSC, suppressing immune dysfunction while promoting neurogenesis, 

MSC may be appropriate therapy for ASD,  in particular in young children (“brain plasticity”). Synergistic Effect (1+1>2)

To date, autologous stem cell therapy (SCT) has proven safe and effective. Short-term side effects may include headache and dizziness that resolves quickly.

Further reading: Research has shown that Stem Cells are Safe and Feasible in Young Children with ASD. Read more

Biomarker study based on biological immune dysfunction in children with ASD

Cell-El is the first to study the potential impact on serum proteins of ASD children following stem cell therapy.  Cell-El is investigating the correlation between the immune system biomarkers of the children with ASD before and after stem cell therapy (SCT) vs. their behavior. 

Please note that this page illustrates one therapeutic approach for Autism.