Current Therapies are Symptomatic
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 (allogeneic, 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, both allogeneic and 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 both allogeneic and 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 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
Allogeneic SCT
SCs are collected from donors of umbilical cord blood or tissue and placental tissue. Often the cells are also reproduced in the lab and an injectable product is produced. Sometimes after production into an injectable product they are frozen (cryopreserved) and then thawed before use. Other clinics use freshly obtained cells which were not frozen. Each clinic using this allogeneic SCT procedure has its own protocol for dose (how many cells per kg weight of the patient) dosing schedule (one time dose or multiple doses over several days) and method of delivery (only via IV or combined with intrathecal method – as described above). The patient is usually sedated for the procedure with mild and short-acting anesthesia for comfort and cooperation.
It remains to be determined by the research as to which method – including source of cells, dose, dosing schedule and method of delivery – is most effective. In recent years- a cell product called exosomes were added by some clinics to their protocol. Exosomes are cell material located outside the cell which then pass on information to other cells further along. As opposed to stem cells, they are very small and can pass into the brain. Some practitioners feel this adds to the efficacy of the treatment. They are often administered by a nasal spray device. Again- this has not yet been determined by any clinical trials for autism.
Monitoring after SCT
Cell-El’s protocol includes monitoring participants at 2 and 6 months following stem cell transplantation (SCT), using psychological assessments and laboratory evaluations. As the first to study the potential impact of SCT on serum proteins in children with autism spectrum disorder (ASD), Cell-El is investigating correlations between immune system biomarkers and behavioral outcomes before and after treatment. These findings may support the development of a biomarker-based tool to help guide and evaluate treatment efficacy.
Further reading: Therapeutic properties of mesenchymal stem cells for autism spectrum disorders, Medical Hypotheses
Please note that this page illustrates one therapeutic approach for Autism.
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.
Help Us Help You
Better diagnostics for ASD based on laboratory-measured objective biomarkers can possibly enable researchers and clinicians to provide more precise and personalized treatment. You too can make a difference in the lives of children with ASD and their families by joining the Cell-El study. Cell-El is recruiting mothers and their infants aged 10-18 months who have not been diagnosed with ASD but who have a sibling diagnosed with ASD.
Please contact Leah at [email protected] or fill out the form to find out about eligibility to participate in our diagnostic study and please help spread the word about the Cell-El study. Sharing our study will enable our important biomarker diagnostic tool to be integrated into autism treatment as quickly as possible.