Stem Cell Treatment for Optic Nerve Hypoplasia (ONH)
A blend of stem cells and comprehensive therapies offers patients a genuine opportunity for meaningful improvement.
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CELLAAX is a clinical-stage biopharmaceutical company engaged in the discovery, research, development and commercialization of Regenerative Medicine.
Optic Nerve Hypoplasia
Is Stem Cell Treatment for Optic Nerve Hypoplasia (ONH) Effective?
Are you exploring Stem Cell Therapy for Optic Nerve Hypoplasia (ONH) or similar visual impairments?
Optic Nerve Hypoplasia is a congenital condition where the optic nerve, responsible for transmitting visual information from the eye to the brain, is underdeveloped. This can lead to significant vision loss or blindness. Standard treatments primarily focus on managing symptoms and maintaining quality of life. However, recent studies suggest that stem cell therapy offers the potential to repair damaged or underdeveloped optic nerves, providing new hope for ONH patients.
Read on to discover if stem cell therapy could benefit Optic Nerve Hypoplasia.

Stem Cell Treatment for Optic Nerve Hypoplasia: A Breakthrough in Visual Rehabilitation
Stem cell therapy has emerged as a promising treatment for optic nerve-related disorders. Through targeted injections, stem cells can potentially restore some vision by regenerating optic nerve cells, improving vision and the overall quality of life for ONH patients.
What is Optic Nerve Hypoplasia?
Optic Nerve Hypoplasia (ONH) is a developmental condition where the optic nerve, responsible for relaying visual information from the eye to the brain, is underdeveloped. Unlike optic nerve atrophy, which involves degeneration, ONH results from incomplete development, typically occurring during prenatal development. This underdevelopment limits the number of optic nerve fibers, which can lead to partial or complete vision impairment. ONH can present alone or alongside other neurological or endocrine conditions.
Factors that may contribute to or exacerbate vision loss in ONH include:
- Prenatal Genetic Factors: Genetic or chromosomal abnormalities can play a role in the development of ONH, though the exact genetic causes are not fully understood.
- Maternal Health Conditions: Health conditions during pregnancy, including diabetes, infections, or substance exposure (such as alcohol or certain medications), have been linked to higher ONH incidence.
- Brain Abnormalities: ONH may occur with conditions like septic dysplasia, in which the optic nerve and parts of the brain, such as the pituitary gland, are underdeveloped.
Other Developmental Disorders: ONH can coincide with other conditions that impact vision or neurological function, leading to a complex clinical picture in affected individuals.
Common Symptoms of ONH
- Reduced visual acuity
- Nystagmus (involuntary eye movement)
- Diminished peripheral vision
- Sensitivity to light
- Depth perception difficulties
How Can Stem Cell Treatment help with Optic Nerve Hypoplasia?
The table below presents findings from a survey of 108 patients who received stem cell treatment with Beike Cell Therapy for Optic Nerve Hypoplasia. This questionnaire aimed to capture detailed insights into patient satisfaction, the stem cell treatment’s perceived effectiveness, and potential improvement areas.
The responses have been carefully organized to view patients’ experiences and treatment outcomes comprehensively.
Data Last updated 10th November 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 | |
---|---|---|---|---|
Light perception | 71% | 38% | 12% | 21% |
Ability to see hand movement | 47% | 22% | 8% | 17% |
Visual field | 54% | 34% | 11% | 9% |
Vision in right eye | 47% | 25% | 10% | 10% |
Vision in left eye | 50% | 28% | 8% | 14% |
Blindness | 59% | 35% | 11% | 13% |
Ability to see things clearly | 33% | 17% | 5% | 11% |
Ability to see things at a close distance | 47% | 24% | 10% | 13% |
Colour vision | 39% | 23% | 5% | 11% |
Ability to focus eyes quickly | 48% | 29% | 11% | 8% |
Able to count fingers | 24% | 12% | 5% | 7% |
Night vision | 23% | 15% | 2% | 6% |
Ability to keep eyes focused for a long time | 41% | 25% | 10% | 6% |
Strabismus (side glances) | 45% | 26% | 12% | 7% |
Nystagmus (uncontrolled eye movement) | 70% | 36% | 16% | 18% |
Ability to see things at a far distance | 32% | 16% | 9% | 7% |
Astigmatism | 11% | 8% | 2% | 1% |
Droopy eye lids | 12% | 5% | 2% | 5% |
Do you feel as though your stem cell treatment has improved the quality of life?
No | 20% |
Yes, has slightly improved | 41% |
Yes, has moderately improved | 18% |
Yes, has significantly improved | 21% |
% of patients with some level of positive result | 80% |
Are you currently satisfied with the outcome of the treatment?
No | 7% |
No comment | 13% |
Somewhat satisfied | 33% |
Yes | 55% |
% of patients a positive level of satisfaction | 88% |
Are you currently experiencing any improvements in your general physical condition?
No | 31% |
Yes, small improvements | 33% |
Yes, moderate improvements | 20% |
Yes, significant improvements | 16% |
% of Patients with ongoing improvements | 69% |
How Stem Cell Therapy Improves Symptoms of Retinal or Optic Nerve Disorders
Stem cells 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, cartilage, and blood cells), or endodermal cells (ex., cells of internal body organs).
Therefore, injecting stem cells – from a donor – should theoretically allow them to differentiate and “replace” the damaged retina or optic nerve seen in different ophthalmological disorders. Stem cell therapy has provided new hope in curing or at least improving the sight-related symptoms associated with retinal or optic degeneration in order to provide patients with a better quality of life. Following the testing of stem cell treatment on people with retinal and/or optic nerve atrophy, in addition to their self-renewing abilities, stem cells have proven to have additional benefits other than tissue replacement.
- Replacing and repairing the damaged optic cells: As mentioned, this is their original function; to replace the damaged tissue – i.e. retinal cells in case of retinal damage and optic neurons in case of optic nerve atrophy – through the conversion of one cell type to another.
- 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 retinal/optic nerve neurons.
- Modulating the immune system and the ongoing inflammatory process: Stem cells produce different antioxidants; thereby reducing the neurodestructive and atrophic process characterizing retinal degeneration and optic nerve atrophy.
- Preventing cell death: Through releasing substances that can inhibit the process of apoptosis, or programmed cell death, of the damaged cells until they are adequately repaired by stem cells.
Benefits of Stem Cell Therapy in Optic Nerve Hypoplasia (ONH) / Septooptic Dysplasia
Studies testing stem cell therapy in people with retinal and/or optic nerve disorders have reported that the use of stem cell therapy in affected infants has shown improvement in (8):
- Visual Acuity
- Color perception
- Visual field
- Light perception
- Depth perception
- Night vision
Improvement rates following stem cell transplantation reach as high as 83%; with improvement being seen in both eyes simultaneously (8). These results could be seen as miraculous in such optic disorders with otherwise irreversible neurological damage.