Spinal muscular atrophy or SMA is a group of genetically inherited progressive neuromuscular diseases. SMA is thought to be caused by a missing/mutated gene that attacks voluntary muscles throughout the human body. As a collection of various muscle-related diseases, SMA is the second leading cause of death after Duchenne.
What is Spinal Muscular Atrophy?
It is estimated that spinal muscular atrophy affects one in 7000 babies born and that about one in 50 people are genetic carriers of the disease. SMA is an autosomal recessive genetic disorder which means that most cases of SMA can be attributed to getting defective genes from both parents. It is believed that if both parents are carriers of this genetic disorder to chance of the child developing this disease is 25%. Potential potential parents can use genetic testing to see if they are carriers of this gene though PGD pre-implementation genetic diagnosis testing that is available in most countries around the world including Thailand.
4 Types & Classifications of SMA disease
- SMA type I which is the most severe type is also known as Werdnig-Hoffman disease.
- SMA type II is less severe than type I asked patients typically infants do not show symptoms of SMA during early infancy however over time they become weaker and weaker.
- SMA type III is a less severe form and typically begins in early adulthood and progressively gets worse as the patient’s age.
- SMA type IV is the least severe type of disease where weakness and muscle wasting generally start in adulthood.
Due to its genetic nature, traditional treatments have resulted in inconsistent results. Neural stem cell transplants and gene therapy are currently the most effective treatment options for control and remission of spinal muscular atrophy.
Symptoms of SMA
Muscle weakness in the legs and shoulder muscles are the first signs of the disease. Weakness and muscle atrophy get significantly worse over time and can eventually lead to complete paralysis and death.
Patients with SMA also have significant difficulty with physical activities such as walking, crawling, and I coordination, neck and had control and dysphagia ( trouble swallowing ). Patients with SMA also have a significantly higher risk for developing breathing issues due to the respiratory muscles in the lungs failing.
Other symptoms include:
- Muscle atrophy
- Muscle weakness
- Trouble Breathing
- Thin muscle mass
- Trouble eating or swallowing
- Lack of head and neck control
- Involuntary facial twitching
- Atrophic muscle changes
- Muscle twitching
- Sensory neuropathies
- Difficulty sitting up or walking or crawling (infants )
- Thin muscle mass
- Missing tendon reflexes
- Impotency, ED and/or reduction in sexual potency
Testing and Diagnosis
Testing or diagnosis SMA can be performed before, during or even after pregnancy. Please note we do note a diagnosis from a neurologist in your home country is required for all potential patients. The first 2 options are preventative in nature and can be done using blood, tissue or genetic samples at a prenatal testing lab. The last option is for those that are alive but displaying symptoms of the disease. Blood samples are required for genetic testing to check for the faulty gene that causes SMA. Physical examinations can also be carried out by trained physicians to look for certain signs and symptoms such as:
- Reduction or absence of tendon reflexes
- Involuntary twitching of muscle fibers
- Signs of muscle weakness and/or wastage
Neural Cell Transplants for Spinal Muscular Atrophy
The underlying cause of SMA is a missing or severely mutated gene known as SMN1 or “Survival Motor Neuron 1.” SMN1 produces a protein called SMA or “Survival Motor Neuron.” It is this protein ( or lack thereof ) that is needed for humans to have healthy motor neurons (nerve cells) that act as the wires for the spinal cord signal to muscles throughout our bodies. Without the SMN protein, our nerve cells begin the process of atrophy and will eventually die, causing the symptoms of weak/limp muscles.
TREATMENT PRECAUTIONS & RISKSPlease note that not all cases will qualify for treatment of Spinal Muscular Atrophy using enhanced neural stem cells. Patients with late stage, severe underlying conditions or travel restrictions are also not candidates for the 2-4 week treatment protocol.
Stem Cell Treatment for SMA
The regeneration center offers effective therapeutic solutions for the family of neuromuscular diseases such as ALS, Ataxia, Brain Strokes, Motor Neuron disease, Parkinson’s, MS and Alzheimers. The 2-3 week treatment protocol in Bangkok seeks to target the increased production of the SMN 1 and 2 gene that is responsible for healthy motor-neuron functions.
Using laboratory enhanced allogeneic mesenchymal cells derived from umbilical cord blood, Amniotic fluid or healthy placenta. we are able to provide remission for over 70% of all cases we accept. Our treatment protocol is unique because we focus on engrafting enriched neural cells that help produce neurotrophic factors that are primarily the ones responsible for the overall growth, survival, and flourishing of mature neuron cells. The neurotrophic factors & exosomes also help in the inhibition of HGK kinases and GSK-3 that help prevent premature motor-neuron cell-death. Learn more about stem cells therapies.
Our SMA stem cell treatment helps to slow down the rapid progression of the disease and have been used in regenerative medicine for over 20 years to treat a wide variety of spinal cord accidents/injuries and other neuromuscular diseases that are interrupting the proper flow of neuro-signals that control our nerve and voluntary muscular movement.
SMA Treatment Overview
The protocol used will depend entirely on the patients’ needs and current condition. The Neural stem cells and progenitor cells are GMP certified and administered with all documentation provided. All injections are administered via Intravenous Drips, Intrathecal or lumbar punctures as needed depending on the patients need and age.
Rehabilitation for “SMA”: Physical Rehabilitation therapy is optional but available full-time or part-time after treatment. Physical Rehabilitation can take place in Bangkok or your home country. We offer full rehab facilities with Medical visas and accommodations for extended stay upon request.
SMA Therapy Guidelines
Due to varying types and severity, the costs for treating spinal muscular atrophy using neural progenitor stem cells will require a proper review of patients’ actual medical needs. Our neurologists and medical team will need to conduct an in-person or virtual consultation before being able to offer acceptance with a detailed treatment plan. Our medical review can be done online or via email. To make a determination and provide an accurate and detailed treatment plan our medical review team will require recent medical records including any diagnostic exams such as recent Muscle biopsies, Brain MRI’s or CT Scans. All Results must be less than 3 months old to make an accurate diagnosis.
To request a new medical review for our multi-stage SMA treatment please contact us today.
Published Clinical Citations
 ^ Grunseich, Christopher, Kristen Zukosky, Ilona R Kats, Laboni Ghosh, George G Harmison, Laura C Bott, Carlo Rinaldi, et al. 2014. Stem cell-derived motor neurons from spinal and bulbar muscular atrophy patients. Neurobiology of disease (June 9). doi:10.1016/j.nbd.2014.05.038. https://www.ncbi.nlm.nih.gov/pubmed/24925468
 ^ Wang, Zhi-Bo, Xiaoqing Zhang, and Xue-Jun Li. 2012. Recapitulation of spinal motor neuron-specific disease phenotypes in a human cell model of spinal muscular atrophy. Cell research, no. 3 (December 4). doi:10.1038/cr.2012.166. https://www.ncbi.nlm.nih.gov/pubmed/23208423
 ^ Zanetta, Chiara, Giulietta Riboldi, Monica Nizzardo, Chiara Simone, Irene Faravelli, Nereo Bresolin, Giacomo P Comi, and Stefania Corti. 2014. Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA). Journal of cellular and molecular medicine, no. 2 (January 8). doi:10.1111/jcmm.12224. https://www.ncbi.nlm.nih.gov/pubmed/24400925