Stem Cell Treatment for Ataxia: What It Involves, Who It Is For, and What the Results Show

Is Stem Cell Treatment for Ataxia Effective?

Are you considering Stem Cell Treatment for Ataxia? Ataxia is a debilitating condition affecting the nervous system that has limited curative treatment options, with most focusing on symptom alleviation. Stem cell treatment is being studied as a way to change that, with early clinical studies in spinocerebellar ataxia reporting possible slowing of disease progression and improvements in neurological function in some patients (1, 2, 3, 6).

Read on to see if Ataxia Stem Cell Treatment might be right for you.

Is Stem Cell Therapy a Cure for Ataxia?

No. Stem cell therapy does not cure ataxia.

What the published studies and our own follow-up data describe is possible improvement in specific symptoms, such as balance, coordination, speech and fatigue, in some patients, though not in all. Stem cells do not correct the gene behind a hereditary ataxia, so where improvement occurs it may not be permanent, and periodic treatment may be considered in selected cases. Clinical benefit is not guaranteed, the degree of improvement differs considerably from one person to another, and the treatment remains experimental (1, 2, 3, 7, 8, 9).

What the most recent research shows

Several clinical studies have investigated stem cell treatment for ataxia and related neurological conditions, with some reporting encouraging improvements in balance, coordination and neurological function.

In a 2013 study, 16 patients with hereditary spinocerebellar ataxia received umbilical cord-derived mesenchymal stem cells through intravenous and intrathecal administration. Most patients showed improvements in balance and ataxia scores lasting at least six months, with no serious treatment-related adverse events reported during the 12-month follow-up (1). An earlier study published in 2011, involving 24 patients with spinocerebellar ataxia or multiple system atrophy-cerebellar type, also reported improvements in neurological function and daily activities following intrathecal umbilical cord MSC treatment, although more than half of the patients (14 of 24) returned to their pre-treatment condition within 1 to 14 months, on average after about three months (2). Both studies were small and had no placebo control group, but their findings suggested that umbilical cord MSC therapy might help alleviate certain symptoms of ataxia.

A recently published clinical trial in hereditary ataxia is a 2026 phase I/IIa study of ten people with hereditary ataxia (eight with Friedreich’s ataxia, one with SCA1 and one with SCA2). Each received a single intrathecal dose of their own bone marrow mesenchymal stem cells. The average score on the SARA ataxia scale fell from 14.9 to 10.36 over six months, and the reported treatment-related side effects were a mild headache in three patients and a low fever in one. The trial had no control group (the planned one was dropped because of funding limits and international sanctions), and the authors themselves state that no conclusion about efficacy can be drawn from it (report summary) (3).

One important placebo-controlled study was conducted in multiple system atrophy, a related condition that causes cerebellar ataxia. A 2012 randomized trial involving 33 patients reported that disability still worsened in both groups, but neurological deterioration was slower than in the placebo group over one year. The gap between the groups was widest at about eight months and narrowed afterwards (4). More recently, a 2026 systematic review of seven studies involving 123 patients, including this trial, found that several studies reported slower disease progression following cell-based treatment, with mostly mild and temporary side effects. Every non-randomized study in the review was rated at serious or critical risk of bias, and the studies used different cell types and treatment methods (report summary) (5).

A 2026 systematic review of 17 clinical reports, including nine involving mesenchymal stem cells, found that cell-based therapies showed encouraging short-term safety and possible improvements in neurological function in some patients. However, most studies were small and uncontrolled, and improvements were sometimes temporary. Where benefits were seen they were generally modest and often short-lived, and most studies followed patients for only one to six months (6).

Another 2026 review of the field highlighted the potential neuroprotective and anti-inflammatory effects of mesenchymal stem cells, including their ability to support existing nerve cells. These findings provide further scientific reasons to investigate stem cell therapy for ataxia, although much of the evidence for these mechanisms comes from laboratory and animal studies. The review reports that in preclinical studies, injected cells can be detected for one to four weeks, with few or no cells remaining after four to eight weeks. These findings come from experimental research and do not establish how long transplanted cells survive in patients with ataxia. However, their potential effects may last longer than the cells themselves, and the need for repeat treatment has not yet been established. It also notes that two pooled analyses of the same three small, uncontrolled clinical studies found no statistically significant overall improvement, with limited long-term safety data beyond twelve months in patients with ataxia (report summary) (7, 8, 9).

The studies so far are generally small and many remain uncontrolled. Their results are encouraging, and some controlled studies have reported possible slowing of deterioration but no reversal, although consistent long-term effectiveness has not yet been established. All of our summaries are listed on the ataxia medical reports index.

Who Is a Candidate for Stem Cell Treatment for Ataxia?

Not every form of ataxia is likely to benefit from stem cell therapy, and the first step is always an accurate diagnosis.

• Primary ataxias are the main group we treat: These are the hereditary forms, such as spinocerebellar ataxia and Friedreich’s ataxia, and idiopathic late-onset cerebellar ataxia. They have no curative conventional treatment. However, certain types of hereditary ataxia now have approved disease-specific medications (15, 21, 22, 23, 24).
• Acquired ataxias: Where ataxia is caused by something treatable (a vitamin B1 or B12 deficiency, hypothyroidism, excessive alcohol intake, a tumour or a stroke), treating that cause comes first, and the ataxia may improve depending on the cause and the extent of neurological damage.
• Confirmed diagnosis: Anyone with a progressive gait disorder or imbalance should be evaluated by a neurologist. An MRI is commonly recommended as part of the evaluation, and genetic testing should be considered when hereditary ataxia is suspected or no treatable cause is found. A recent systematic review found a median genetic diagnostic rate of 43 percent in patients with suspected hereditary ataxia, although the percentage varies depending on the patients and testing methods used (10, 11). We ask for these records as part of the medical review.
• Repeat treatment: In hereditary ataxias, improvements may not be permanent because stem cells do not influence the genetic origin of the disease. Periodic treatments may be considered following medical reassessment, although whether repeat treatment helps has not yet been tested in clinical studies (7).

Suitability is decided case by case. Every enquiry goes to our medical department, which reviews the patient’s medical condition through our online medical evaluation system and only then recommends a specific treatment location and protocol. We recommend speaking to our specialists before you decide, so that you know what can realistically be expected in your case.

Send us the medical records and our team will come back to you.

How Stem Cell Therapy Works in Ataxia

Stem cell treatment involves the administration of specialized cells that have the ability to support tissue repair and communicate with other cells in the body. The MSCs used in our treatment are multipotent cells, meaning they can develop into several specialized cell types and, importantly, release biological signals that can influence surrounding tissues. In neurological disorders, their potential benefits are thought to come mainly from these biological signals rather than from directly replacing damaged nerve cells (7, 12).

In the case of ataxias and other neurological disorders, they may offer additional neuroprotection, anti-inflammatory and regenerative-supporting effects, and potentially help to reduce some of the processes involved in neurological degeneration.

Research suggests that MSCs may support neurological function through several mechanisms (7):

• Release of neurotrophic factors: Release or stimulate the production of factors such as BDNF and GDNF that support nerve cell survival, function and communication.
• Immune modulation: Reduce some inflammatory signals and change the activity of immune cells around injured neural tissue.
• Promoting vascular and trophic support: Promoting the release of factors that support vascular function and tissue repair.
• Antioxidant effects: Potentially reducing free radicals and reactive oxygen species production that could have detrimental effects on the nervous system.
• Protection of existing neurons: MSC treatment may help protect Purkinje cells, which play a major role in balance and coordination, as demonstrated in preclinical studies (12).
• Supporting cellular communication: MSCs release signaling molecules that may help maintain a healthier environment for surviving neurons.

Given the potential benefits reported for stem cell therapy in multiple conditions, including many genetic disorders, many studies have aimed to test the efficacy of stem cell treatment in several common forms of hereditary ataxia that lack a curative therapy – namely Spinocerebellar ataxia and Friedreich’s ataxia.

In patients with hereditary ataxias, studies of stem cell therapy have reported or are investigating the possibility that it may (1, 2, 3, 7, 12):

• Help slow disease progression in some patients
• Help protect cerebellar neurons from further damage
• Support the function and survival of existing nerve cells
• Improve motor skills, balance, and coordination
• Support nervous tissue functioning and daily activity, with possible improvements in alertness and fatigue reported by some patients
• Possibly reduce pain in certain patients, although evidence for this particular benefit remains limited
• Modulate immune and inflammatory responses that may contribute to neurological damage

In general, stem cell therapy has been shown to be well-tolerated in people with ataxia, with minimal side effects that are easily managed and rarely cause significant problems. Some patients report noticing an improvement within around two weeks of stem cell transplantation, although this differs from person to person. The degree of improvement varies considerably between patients. Some may notice changes in specific symptoms, while others may experience little or no improvement (1, 2, 3, 6).

Additionally, stem cell therapy acts through several mechanisms at once and may provide a range of potential benefits that are difficult to obtain through conventional treatment methods that require multiple modalities because each drug usually targets one aspect of the disease. This doesn’t in any way say that stem cell therapy can be used as a single treatment modality since we still recommend physiotherapy and other forms of treatments in our center, but it simply states the possible advantages of stem cell therapy over conventional modalities.

Few conventional treatment options are available for patients diagnosed with ataxia, which mostly focus on alleviating the symptoms. Although some disease-specific treatments are now available, many progressive forms of ataxia still lack effective treatments capable of preventing neurological degeneration. Stem cells are being investigated for their potential neuroprotective and regenerative-supporting effects, which may help improve certain symptoms and neurological functions in some patients (6, 7, 8, 9).

Possible improvements after Stem Cell Therapy for Ataxia

The purpose of our stem cell treatment is to support neurological function and potentially improve the symptoms associated with ataxia through the possible neuroprotective and immunomodulatory effects of stem cells. Thus, various kinds of improvement are possible after our treatment and our past patients have reported the following*:

Safety, Side Effects and Cell Quality Control

Like any medical treatment, stem cell therapy can have side effects. Many of the short-term reactions reported are similar to those that can occur after an IV infusion or injection, such as fever, headache, nausea, temporary discomfort, or an allergic or immune reaction. Intrathecal administration also involves a lumbar puncture and may cause temporary procedure-related symptoms such as headache, back discomfort, nausea or vomiting. In general, stem cell therapy has been reasonably well tolerated in early clinical studies involving patients with ataxia, with side effects that are usually mild and can be managed medically (3, 6, 13, 14).

In the 2026 hereditary ataxia trial described above, three of ten patients had a mild headache and one a low fever after the intrathecal injection, with no severe events over six months (report summary) (3). The 2026 review of multiple system atrophy studies reported fever after MSC treatment, and mild headache and fever in studies using cord blood cells. It also reported small ischaemic lesions on MRI after delivery of cells through an artery, a route we do not use, although these occurred at a similar rate in patients given placebo and were attributed to the procedure rather than the cells. In one study using intrathecal stem cell injections, MRI scans showed thickening or mild enhancement of the nerve roots in the lower spine in every patient who received the medium or high dose. About half of these patients had low back or leg discomfort, which persisted in two and led the investigators to stop the highest dose, but none developed neurological deficits (report summary) (5, 14). Long-term safety data remain limited.

Our protocol delivers cells both intravenously and by intrathecal injection, which is performed after a lumbar puncture, a procedure with its own risks (most commonly headache and back pain afterwards), which the treating physicians go through with you before you consent.

On the cells themselves: mesenchymal stem cells and umbilical cord blood stem cells have been studied for safety, and unlike embryonic stem cells, they have not shown the same tendency to form tumours in clinical studies to date (study). In randomised trials of intravenous umbilical cord MSCs, no increase in cancer has been seen so far, although most trials followed patients for a year or less and longer follow-up is still needed (13).

Every batch of cells is screened, tested twice and traceable by code before it is released for use. The full process is described on our standards and certifications page.

Where Treatment Happens, and Under Which Regulations

Treatment is delivered at affiliated hospitals in Bangkok, Thailand and in Dongguan, China. It is not approved by the US Food and Drug Administration and is not offered in the United States. It is given under Thai and Chinese regulations for cell therapy, and the cells are prepared in laboratories with CNAS, GMP and ISO accreditation. The accreditation details and the certificates are on our standards and certifications page.

Our Treatment Program in Details

Beike is unlike any other stem cell treatment provider in the world, the reason? Since 2005, we have been developing and optimizing our stem cell treatment protocols with the concept that a comprehensive treatment approach may provide more individualized support for our patients.

We believe that stimulation through various therapies is necessary to enhance stem cell regenerative response, therefore our protocols include daily therapies to support the stem cells. Finally, we provide a wide variety and large quantities of stem cells in order to adapt to each patient specific condition and deliver maximized regenerative potential.

Our ataxia treatment consist in 6 to 8 simple and minimally invasive injections of umbilical cord derived stem cells. The stem cells are transplanted using two separate methods: by intravenous way using a standard IV drip system, and through intrathecal injection performed after lumbar puncture. These two delivery methods allow for increased efficacy while ensuring safety and minimum inconvenience for the patient.

Patient case Study - Ataxia Stem Cell Treatment

Robin and his family were in shock when he was diagnosed with Ataxia at only 16 years old of age. The first symptoms were shaking, walking imbalance, and difficulties in speaking. After researching online his mom came across our Youtube videos and decided that Beike could be the hope to improve Robin’s quality of life. After a successful first stem cell treatment in 2018, Robin and his mom agreed to sit with us and share the dramatic improvements he has experienced and what made them decide to come back in March 2022.

Watch more videos

Frequently Asked Questions

Q.Can stem cell therapy cure ataxia?

A.

No. Stem cell therapy is not a cure for ataxia. It does not correct the gene behind a hereditary ataxia. What studies and our own follow-up data describe is possible improvement in specific symptoms, such as balance, coordination and speech, for some, but not all, patients, and in hereditary forms repeat treatment may be considered individually to maintain it, although its effectiveness in maintaining improvement remains uncertain. Improvement cannot be guaranteed and differs considerably from one patient to another (1, 2, 3, 7, 8, 9).

Q.Is stem cell therapy for ataxia approved by the FDA?

A.

No. Stem cell therapy for ataxia is not approved by the US Food and Drug Administration, and we do not offer it in the United States. Treatment is delivered at affiliated hospitals in Thailand and China under those countries’ regulations for cell therapy. The laboratories that prepare the cells hold CNAS, ISO and GMP accreditation, which is not the same thing as FDA approval of the treatment. More detail is on our standards and certifications page.

Q.What exactly is Ataxia?

A.

In order to delve into this treatment modality in ataxia, we first need to explain what exactly we mean when we say “ataxia”. Ataxia describes a loss of coordination and balance caused by problems affecting the nervous system. It can make activities such as walking, speaking, writing and using the hands more difficult. You might see yourself as clumsy or ungraceful, but this might actually be one symptom of ataxia. It happens when the parts of your nervous system responsible for coordinating movements, especially the cerebellum (a part of the brain), are not working properly. Ataxia is not a single disease but a symptom that can occur in several neurological conditions. It can result from inherited neurological conditions or from acquired causes, including stroke, autoimmune disease, vitamin deficiencies or certain medications (15, 16).

Q.What causes Ataxia?

A.

Ataxia can have several causes, including inherited genetic conditions, damage to the nervous system, or other medical problems. Depending on the cause, ataxia can generally be divided into three main groups (15, 16):

1. Hereditary Ataxia (Genetic Ataxia) This type of ataxia is caused by changes in certain genes that affect how the nervous system works. It can be passed down from parents to their children, although some patients may have no known family history of the condition. There are several types of hereditary ataxia, depending on how the genetic condition is inherited:

• Autosomal Dominant: This type can develop when a person inherits one affected copy of a gene from either parent. Each child of an affected parent generally has a 50% chance of inheriting the genetic change. Common examples include Spinocerebellar Ataxia (SCA), such as SCA1, SCA2, SCA3, and SCA6. Symptoms often begin in adulthood and gradually worsen over time, affecting balance, walking, speech, and coordination. Some types may also cause muscle stiffness, abnormal eye movements, or difficulty swallowing.
• Autosomal Recessive: This type usually occurs when a person inherits an affected copy of a gene from both parents, who may be healthy carriers. Common examples include Friedreich’s Ataxia (FRDA) and Ataxia-Telangiectasia. Symptoms often begin during childhood or adolescence, although some forms can appear later in life. In addition to balance and coordination problems, certain types may affect the heart, nerves, immune system, or other parts of the body. Progression varies depending on the specific condition.
• Mitochondrial Ataxia: This type is associated with genetic changes affecting the mitochondria, which are responsible for producing energy in our cells. Some forms are inherited through the mother, while others follow different inheritance patterns. These conditions may affect balance and coordination, along with other parts of the body. Symptoms may begin in childhood or adulthood and can vary considerably. Besides coordination problems, patients may experience muscle weakness, fatigue, hearing or vision difficulties, and sometimes seizures. Disease progression also varies depending on the underlying condition.
• X-linked Ataxia: This is a less common form caused by changes in genes located on the X chromosome. It may affect males more frequently, although females can also develop symptoms. One example is Fragile X-associated Tremor/Ataxia Syndrome (FXTAS). Symptoms of FXTAS usually appear after the age of 50, often beginning with hand tremors, followed by increasing balance difficulties. Some patients may also experience memory or concentration problems, with symptoms gradually worsening over time.

2. Acquired Ataxia This type develops as a result of another medical condition or damage to the nervous system. Some common causes include:

• Stroke or brain injury
• Vitamin deficiencies, especially vitamins B1, B12, and E
• Multiple sclerosis and other autoimmune diseases
• Brain tumors or infections
• Excessive alcohol consumption
• Certain medications or toxic substances

Unlike many hereditary ataxias, acquired ataxia may develop suddenly, as in a stroke, or gradually, depending on the cause. In some forms, treating the underlying condition can improve ataxia symptoms or prevent further deterioration, especially if treatment begins early.

3. Idiopathic Ataxia (Unknown Cause) In some patients, ataxia develops without an identifiable cause, even after medical investigations. This is known as idiopathic ataxia and may also be described as sporadic ataxia when there is no known family history.

Idiopathic late-onset cerebellar ataxia usually develops during adulthood and gradually worsens over time. Symptoms commonly begin with balance and walking difficulties, followed by possible problems with coordination and speech.

In some cases, further investigations or newer genetic tests may eventually identify the underlying cause. Because ataxia can have different causes, an accurate diagnosis is important to determine the most appropriate treatment and management plan.

Q.What are the Conventional Treatments for Ataxia?

A.

When it comes to treating ataxia, treatment mainly depends on the exact cause of ataxia. For example, if ataxia is caused by a stroke, then we manage the stroke and the ataxia may subsequently improve depending on the extent of the injury. If it is caused by nutritional deficiency, then we supplement the deficient nutrient, and we may see improvement in ataxia, particularly when the deficiency is treated early. Treating the underlying cause may help prevent further deterioration and, in some cases, improve neurological function.

We still don’t have a single “curative” treatment for hereditary and progressive ataxias. Most conventional treatments focus on alleviating symptoms, improving mobility and coordination, and helping patients maintain their independence and quality of life. In hereditary ataxias, doctors usually recommend physiotherapy and occupational therapy for better muscle control and in coping with the condition. A recent meta-analysis found that physiotherapy, especially programmes combining coordination, balance and gait training, modestly reduced ataxia severity and slightly improved daily functioning, although the quality of the evidence is low (17). Symptomatic treatments are usually used as well to alleviate symptoms depending on the main complaint. For example, if you are complaining of urinary incontinence, bladder relaxants are prescribed, and so on. Sometimes, medications are used for their potential to improve neurological symptoms or slow progression, similar to their use in other neurological disorders, such as:

• Baclofen: This is a muscle relaxant that could help in muscle spasticity and pain associated with ataxia.
• Acetazolamide and 4-Aminopyridine: In a randomised trial, both medications reduced the number of attacks in episodic ataxia type 2 (18).
• Riluzole: This drug is also commonly used in amyotrophic lateral sclerosis (ALS). It works by modulating glutamate-related neurological activity and has been tested in hereditary ataxias with mixed results: one trial in spinocerebellar and Friedreich’s ataxia reported improvement, but a later trial in spinocerebellar ataxia type 2 found no benefit (19, 20).
• Varenicline: Commonly used for smoking cessation, this drug has also been investigated for certain symptoms of ataxia, although supporting evidence remains limited.
• Amantadine: Also used in Parkinson’s disease, it has been studied for neurological symptoms in some forms of ataxia, with limited and inconsistent evidence.

Their effectiveness varies, and they are not routinely recommended for all patients.

Recent medical advancements have introduced new treatment options for certain hereditary ataxias. For example, omaveloxolone (Skyclarys) is approved for patients aged 16 years and older with Friedreich’s ataxia. It has shown benefits in neurological function, although it is not a cure (15, 21, 23). More recently, in 2026, levacetylleucine (Aqneursa) was approved to treat ataxia in adults and children with ataxia-telangiectasia weighing at least 15 kg. These treatments represent important progress, although they are only suitable for specific types of ataxia and do not provide a cure (22, 24).

However, even with all the current medical advancements, there is still not a single curative treatment for hereditary ataxias, and many available treatments remain supportive or disease-specific, with limitations in their efficacy and possible side effects.

Many are now probably thinking “So where does stem cell therapy come in all of this?” Well, to be honest, not all forms of ataxia could probably benefit from this form of therapy; however stem cell therapy has shown encouraging early results in some patients with primary forms of ataxia – i.e. those not due to a secondary/acquired cause. Primary forms of ataxia usually result from progressive degeneration of the brain areas concerned with balance, in which inflammation plays a secondary part, and stem cell therapy may help support the surviving neurons through neuroprotective and anti-inflammatory mechanisms, as shown in animal studies (7, 12).

Q.What is the difference between Autosomal Dominant and Autosomal Recessive Ataxias?

A.

Autosomal dominant and autosomal recessive ataxias are hereditary ataxias, and spinocerebellar ataxia (SCA) and Friedreich’s ataxia (FRDA) are among the most recognized forms of hereditary ataxia. Autosomal dominant ataxia can develop when only one copy of a gene is affected, while autosomal recessive ataxia usually requires both copies of the gene to be affected. Thus, people can carry a recessive genetic change without developing symptoms, while dominant genetic changes are more likely to cause symptoms, although these may not appear until later in life. The severity and progression of ataxia depend on the specific genetic condition rather than simply whether its inheritance is dominant or recessive (15).

Q.What is the role of Purkinje cells, where can we find those cells ?

A.

Purkinje cells are a type of neuron found in the cerebellar cortex, at the base of the brain. They are among the largest neurons and are responsible for important signaling in the cerebellum involved in coordinating movements. The Purkinje cells and the cerebellum are essential to the body’s motor function. Disorders involving the Purkinje cells usually negatively affect the patient’s movements. Experimental studies have shown that MSC treatment may help protect Purkinje cells and preserve motor coordination in animal models of ataxia (7, 12).

Q.How does ataxia affect the patient?

A.

Ataxia might be a solitary presentation or might be part of a larger set of symptom presentations. Therefore, good awareness of different etiologies and syndromes of ataxia is quite important for proper diagnosis and management. Most types of ataxia have these symptoms in common (15, 16):

• Poor movement coordination
• Regression of fine motor skills (such as buttoning your shirt or using cutlery)
• Loss of balance while walking and adopting a wide-based gait to compensate for this lack of balance and due to fear of falling
• Speech and swallowing problems
• Eye muscle coordination problem (ex. Nystagmus which is involuntary eye movement)
• Fatigue and reduced ability to perform daily activities in some patients

Q.How is Cerebellar Ataxia Diagnosed?

A.

Most people don’t know what cerebellar ataxia is and may overlook the early symptoms. Anyone with progressive gait disorder or imbalance should be evaluated by a neurologist. MRI is commonly recommended as part of the evaluation. If a treatable cause is not discovered, genetic testing should be considered, particularly when hereditary ataxia is suspected. A recent systematic review found a median genetic diagnostic rate of 43 percent in patients with suspected hereditary ataxia, although the percentage varies depending on the patients and testing methods used (10, 11).

Q.What are the Types of Ataxia?

A.

As previously mentioned, there are multiple classifications for different forms of ataxias according to how exactly we look at them. For example, ataxia can be classified according to (15, 16):

• Etiology/Cause: As we discussed, it can be hereditary, acquired, or idiopathic (unknown cause).
• How Symptoms Develop: Acute (developing over hours to days), sub-acute (over weeks), chronic (over months to years), or episodic (occurring in repeated attacks).
• Part of the Nervous System Affected: Ataxia may involve the cerebellum (cerebellar ataxia), the sensory nerves responsible for body position (sensory ataxia), or the inner ear and balance system (vestibular ataxia).

There are many diseases manifesting with ataxia, either as a primary symptom or as part of a larger set of symptoms. However for the sake of being concise, we will briefly mention some of the most commonly encountered hereditary types of ataxia.

Spinocerebellar Ataxia (SCA): This type is the most common dominantly inherited ataxia. In this type, an affected family member is usually present on taking family history and people with this form usually present during adulthood, although symptoms can also begin earlier. There are many genetically different types of spinocerebellar ataxias, each presenting with different symptoms depending on the affected gene. For example, in addition to the previously mentioned symptoms, people with spinocerebellar ataxia could have additional muscle stiffness and pain, loss of hand and feet sensation, reduced bladder control, and problems with memory and language (15, 16).

Friedreich’s Ataxia (FRDA): This is one of the most common autosomal recessive hereditary ataxias, usually occurring when a child inherits an affected copy of the gene from each parent. It typically manifests during childhood or adolescence, although some patients develop symptoms later in adulthood. People with this form of ataxia may have additional findings including slurred speech, diabetes, reduced hearing or vision, abnormal spine curvature (scoliosis), heart problems, and progressive leg weakness. As the disease progresses, some patients may eventually require walking aids or wheelchair assistance, although progression varies considerably between individuals (15).

Ataxia-telangiectasia: This is a rare autosomal recessive hereditary condition that usually begins in early childhood. It commonly causes progressive problems with balance and coordination. People with this condition often have characteristic telangiectasias – i.e. visible clusters of small blood vessels especially in the eyes and skin – and may also develop immune system problems, frequent infections, and an increased risk of cancer. The severity and rate of progression vary, with some patients experiencing milder forms of the disease (25).

Medically reviewed by

Dr. Mohammad Alzogool

Medical Director

Beike's Medical Director, Dr. Mohammad Alzogool, is a physician-researcher with a background in ophthalmology and regenerative medicine. His research contributions span ophthalmology, regenerative medicine, artificial intelligence, medical imaging, and evidence-based clinical guidelines. He brings a strong scientific and clinical perspective to the evaluation of emerging regenerative therapies.

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Published March 23, 2017 Last updated October 19, 2026

Published research

Research on ataxia

Plain-language summaries of independent, peer-reviewed studies. Each one links to the original publication.

• Mesenchymal Stem Cell-Based Therapy for Cerebellar Ataxia: From Bench to Bedside 2026
• Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression 2026
• Safety and Preliminary Efficacy of Autologous Bone Marrow-Derived Mesenchymal Stem Cell Transplantation in Hereditary Cerebellar Ataxia: Phase I/IIa Clinical Trial 2026
• Safety and Efficacy of Mesenchymal Stem Cell Therapy in Multiple System Atrophy: Systematic Review 2026
• Post-Symptomatic Administration of hMSCs Exerts Therapeutic Effects in SCA2 Mice 2024
• Effect of stem cell treatment on functional recovery of spinocerebellar Ataxia 2021
• Pre-emptive Allogeneic Hematopoietic Stem Cell Transplantation in Ataxia Telangiectasia 2018
• Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells 2017

All 14 studies on ataxia

Medical References

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2. 2. Han D, Liu J, Xue M, et al. Clinical analysis of the treatment of spinocerebellar ataxia and multiple system atrophy-cerebellar type with umbilical cord mesenchymal stromal cells. Cytotherapy. 2011;13(8):913–917. https://doi.org/10.3109/14653249.2011.579958
3. 3. Shapouri Moghaddam A, Tavakol-Afshari J, Kaffash Farkhad N, et al. Safety and preliminary efficacy of autologous bone marrow-derived mesenchymal stem cell transplantation in hereditary cerebellar ataxia: phase I/IIa clinical trial. Stem Cell Research & Therapy. 2026;17:249. https://doi.org/10.1186/s13287-026-05163-6
4. 4. Lee PH, Lee JE, Kim HS, et al. A randomized trial of mesenchymal stem cells in multiple system atrophy. Annals of Neurology. 2012;72(1):32–40. https://doi.org/10.1002/ana.23612
5. 5. Elimam N, Albarari SS, Shaalan Y, et al. Safety and efficacy of mesenchymal stem cell therapy in multiple system atrophy: systematic review. Biologics: Targets and Therapy. 2026;20:593367. https://doi.org/10.2147/BTT.S593367
6. 6. Khalesi H, Beiraghi Toosi M, Khalesi A, et al. Cell-based therapies for spinocerebellar degenerations: a systematic review of human clinical evidence. The Cerebellum. 2026;25(3):52. https://doi.org/10.1007/s12311-026-01994-4
7. 7. Suk K, Lee HW, Kim SR. Mesenchymal stem cell-based therapy for cerebellar ataxia: from bench to bedside. CNS Neuroscience & Therapeutics. 2026;32(7):e71038. https://doi.org/10.1002/cns.71038
8. 8. Appelt PA, Comella K, de Souza LAPS, Luvizutto GJ. Effect of stem cell treatment on functional recovery of spinocerebellar ataxia: systematic review and meta-analysis. Cerebellum & Ataxias. 2021;8:8. https://doi.org/10.1186/s40673-021-00130-8
9. 9. Bhartiya M, Kumar A, Singh RK, et al. Mesenchymal stem cell therapy in the treatment of neurodegenerative cerebellar ataxias: a systematic review and meta-analysis. The Cerebellum. 2023;22(3):363–369. https://doi.org/10.1007/s12311-022-01403-6
10. 10. Tenorio RB, Camargo CHF, Donis KC, Almeida CCB, Teive HAG. Diagnostic yield of NGS tests for hereditary ataxia: a systematic review. The Cerebellum. 2024;23(4):1552–1565. https://doi.org/10.1007/s12311-023-01629-y
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