Stem Cell Treatment for Spinal Muscular Atrophy (SMA)

A comprehensive protocol combining stem cells and supportive therapies. Giving patients a real chance for improvements and a better quality of life.

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CELLAAX is a clinical-stage biopharmaceutical company engaged in the discovery, research, development and commercialization of Regenerative Medicine.

Spinal Muscular Atrophy (SMA)

Is Stem Cell Treatment for Spinal Muscular Atrophy Effective?
Are you considering Stem Cell Treatment for Spinal Muscular Atrophy?

Spinal muscular atrophy (SMA) is a neurodevelopmental condition affecting the muscle function and mobility of many children that has limited curative treatment options, with most only focusing on alleviating the present symptoms and increasing the lifespan and quality-of-life of such individuals. Stem Cell Treatment however offers hope, with studies showing stem cell therapy can slow or reverse core symptoms of SMA.

Read on to see if Spinal Muscular Atrophy Stem Cell Treatment might be right for you.

How does stem cell treatment for SMA work?

Through the release of substances such as growth factors, cytokines, and extracellular vesicles, mesenchymal stem cells (MSCs) initiate tissue healing, adjust the immune system’s behavior, and facilitate tissue renewal, while also alleviating inflammation.

In the context of treating Spinal Muscular Atrophy (SMA), it has been discovered that these MSCs can spur the revival of injured motor neurons and bolster muscle performance. Beyond this, they have the capacity to subdue inflammation and enhance immune responses, thereby providing further assistance in the recuperation of harmed tissues. All things considered, stem cell therapy emerges as a promising, safe, and efficient therapeutic approach for those suffering from SMA.

How Stem Cell Therapy Improves Symptoms of Spinal Muscular Atrophy

Stem cells are cells that are “pluripotent”, meaning they can differentiate into all other cells due to their self-renewing abilities. They can develop into ectodermal (ex. skin and some neurological structures), mesodermal (ex. bones, cartilages, and blood cells), or endodermal cells (ex. cells of internal body organs). Therefore, injecting stem cells – from a donor with normal SMN gene – should theoretically allow them to differentiate and “replace” the damaged neurons, and neuron proteins, defective due to genetic abnormalities leading to SMA (6). Despite the lower number of patients tested in literature due to the rarity of the disease, stem cell therapy has proved quite promising results in SMA. It has provided a new hope in curing or at least ameliorating and delaying the symptoms of SMA for a better quality of life. Following the testing of stem cell treatment on people with SMA, in addition to their self-renewing abilities, stem cells have proven to have additional benefits other than tissue replacement; including (7-9):

  • Replacing and repairing the damaged neurons: As mentioned, this is their original function; to replace the damaged tissue – i.e. neurons in case of SMA – through the conversion of one cell type to another. These cells can therefore have normal proteins to function normally.
  • Increasing the production of neurotrophic factors that promote nervous cell proliferation and differentiation (ex. glia derived neurotrophic factor (GDNF) and brain-derived neurotrophic factor (BDNF)). These factors can locally enhance cellular recruitment, proliferation and maturation within the damaged or affected spinal neurons.
  • Modulating the immune system and the ongoing inflammatory process: Thereby reducing the neurodestructive and atrophic process causing characteristic symptoms of SMA
  • Promoting vascular supply to the nervous system: By stimulating the generation of new blood vessels (neovascularization/angiogenesis) through stimulating different vascular stimulating growth factors (ex. VEGF)
  • Preventing neuronal death: Through inhibiting the process of apoptosis, or programmed cell death, of the affected neurons until they are adequately repaired
Spinal Muscular Atrophy Patient Outcome Data

The table below presents the findings from a questionnaire completed by 18 patients who underwent stem cell treatment with Beike Cell Therapy for Spinal Muscular Atrophy. This survey aimed to capture insights regarding patient satisfaction, the perceived effectiveness of the stem cell treatment, and any potential areas of enhancement.

The collected responses have been systematically arranged to offer a thorough overview of the patients’ experiences and outcomes.

This data last updated on the 5th of December 2024

% of Patients who noticed Improvement % of Patients who noticed a Small Improvement % of Patients who noticed a Moderate Improvement % of Patients who noticed a Large Improvement
Energy 100% 43% 21% 36%
Trunk control 88% 35% 35% 18%
Movement in general 88% 35% 41% 12%
Balance 88% 41% 35% 12%
Limb muscle strength 94% 65% 24% 6%
Overall strength 88% 44% 31% 13%
Trunk muscle strength 88% 44% 38% 6%
Fine motor control 87% 40% 40% 7%
Hand control 93% 50% 43% 0%
Range of movement 81% 44% 38% 0%
Standing up 73% 53% 13% 7%
Swallowing 78% 22% 33% 22%
Spasticity 60% 50% 10% 0%
Walking 43% 29% 14% 0%
Crawling 38% 23% 15% 0%
Benefits of Stem Cell Therapy in Spinal Muscular Atrophy

Studies testing stem cell therapy in people with SMA have reported that the use of stem cell therapy in affected infants has shown improvement in (9, 10):

  • Muscle tone:
  • Respiration capacity
  • Speech (in older infants/toddlers)
  • Facial muscle control and expressiveness
  • Better muscle control and movement
  • Swallowing and feeding
  • Degree of drooling
  • Improved general condition and weight


Some scientists in these trials have described the improvement seen following stem cell therapy in SMA as “otherwise-impossible”. However, we advise early stem cell intervention in order to allow for the best possible results.